StemCell Therapy

Its uncontroversial among biologists that many species have two, distinct biological sexes. Theyre distinguished by the way that they package their DNA into gametes, the sex cells that merge to make a new organism. Males produce small gametes, and females produce large gametes. Male and female gametes are very different in structure, as well as in size. This is familiar from human sperm and eggs, and the same is true in worms, flies, fish, molluscs, trees, grasses and so forth.

Different species, though, manifest the two sexes in different ways. The nematode worm Caenorhabditis elegans, a common laboratory organism, has two forms not male and female, but male and hermaphrodite. Hermaphroditic individuals are male as larvae, when they make and store sperm. Later they become female, losing the ability to make sperm but acquiring the ability to make eggs, which they can fertilise with the stored sperm.

This biological definition of sex has been swept up into debates over the status of transgender people in society. Some philosophers and gender theorists define a woman as a biologically female human being. Others strongly disagree. Im addressing those who reject the very idea that there are two biological sexes. Instead, they argue, there are many biological sexes, or a continuum of biological sexes.

Theres no need to reject how biologists define the sexes to defend the view that trans women are women. When we look across the diversity of life, sex takes stranger forms than anyone has dreamt of for humans. The biological definition of sex takes all this in its stride. It does so despite the fact that there are no more than two biological sexes in any species youre likely to have heard of. To many people, that might seem to have conservative implications, or to fly in the face of the diversity we see in actual human beings. I will make clear why it does not.

I call this the biological definition of sex because its the one biologists use when studying sex that is, the process by which organisms use their DNA to make offspring. Many philosophers and gender theorists will protest at making the creation of offspring foundational to how we define sex or distinguish different sexes. Theyre surely right that sex as a social phenomenon is much richer than that. But the use of DNA to make offspring is a central topic in biology, and understanding and explaining the diversity of reproductive systems is an important scientific task. Gender theorists are understandably worried about how the biology of sex will be applied or misapplied to humans. What they might not appreciate is why biologists use this definition when classifying the mind-stretching forms of reproduction observed in limpets, worms, fish, lizards, voles and other organisms and they might not understand the difficulties that arise if you try to use another definition.

Many people assume that if there are only two sexes, that means everyone must fall into one of them. But the biological definition of sex doesnt imply that at all. As well as simultaneous hermaphrodites, which are both male and female, sequential hermaphrodites are first one sex and then the other. There are also individual organisms that are neither male nor female. The biological definition of sex is not based on an essential quality that every organism is born with, but on two distinct strategies that organisms use to propagate their genes. They are not born with the ability to use these strategies they acquire that ability as they grow up, a process which produces endless variation between individuals. The biology of sex tries to classify and explain these many systems for combining DNA to make new organisms. That can be done without assigning every individual to a sex, and we will see that trying to do so quickly leads to asking questions that have no biological meaning.

While the biological definition of sex is needed to understand the diversity of life, that doesnt mean its the best definition for ensuring fair competition in sport or adequate access to healthcare. We cant expect sporting codes, medical systems and family law to adopt a definition simply because biologists find it useful. Conversely, most institutional definitions of sex break down immediately in biology, because other species contradict human assumptions about sex. The United States National Institutes of Health (NIH) uses a chromosomal definition of sex XY for males and XX for females. Many reptiles, such as the terrifying saltwater crocodiles of northern Australia, dont have any sex chromosomes, but a male saltie has no trouble telling if the crocodile that has entered his territory is a male. Even among mammals, at least five species are known that dont have male sex chromosomes, but they develop into males just fine. Gender theorists have extensively criticised the chromosomal definition of human sexes. But however well or badly that definition works for humans, its an abject failure when you look at sex across the diversity of life.

The same is true of phenotypic sex, the familiar idea that sex is defined by the typical physical characteristics (phenotypes) of males and females. Obviously, this approach will produce completely different definitions of male and female for humans, for worms, for trees and so forth. Incubating eggs inside your body, for example, is a female characteristic in humans but a male one in seahorses. That doesnt mean that human institutions cant use the phenotypic definition. But it isnt useful when studying the common patterns in the genetics, evolution and so forth of female humans, female seahorses and female worms.

Understanding the complex ways in which chromosomes and phenotypes relate to biological sex will make clear why the biological definition of sex shouldnt be the battleground for philosophers and gender theorists who disagree about the definition of woman. There might be very good reasons not to define woman in this way, but not because the definition itself is poor biology.

Why did sexes evolve in the first place? Biologists define sex as a step towards answering this question. Not all species have biological sexes, and biology seeks to explain why some do and others dont. The fact that no species has evolved more than two biological sexes is also a puzzle. It would be quite straightforward to engineer a species that has three, but none has evolved naturally.

Many species reproduce asexually, with each individual using its own DNA to create offspring. But other species, including our own, combine DNA from more than one organism. Thats sexual reproduction, where two sex cells gametes merge to make a new individual. In some species, these two gametes are identical; many species of yeast, for example, make new individuals from two, identical gametes. They reproduce sexually, but they have no sexes, or, if you prefer, they have only one sex. But in species that make two different kinds of gamete and where one gamete of each kind is needed to make a new organism there are two sexes. Each sex makes one of the two kinds of gamete.

In complex multicellular organisms, such as plants and animals, these two kinds of gamete are very different. One is a large, complex cell, what wed typically call an egg. Its similar to the eggs produced by asexual species, which can develop into a new organism all on their own. Many species of insect and some lizards, snakes and sharks can reproduce using just an egg cell. The other kind of gamete is a much smaller cell that contains very little beyond some DNA and some machinery to get that DNA to the larger gamete. We are familiar with these two kinds of gametes from human eggs and sperm.

Theres no obvious reason why complex multicellular organisms need to have two kinds of gamete, or why these two kinds are so different in size and structure. Its perfectly possible to make three or more different kinds of gamete, or gametes that vary continuously, just as people vary continuously in height. One question that biologists seek to answer, then, is why those forms of sexual reproduction arent observed in complex organisms such as animals and plants.

Earthworms are hermaphrodites: one part of the worm produces sperm and another part produces eggs

When a species produces two different kinds of gamete, biologists call this anisogamy, meaning not-equal-gametes. Some anisogamic species have separate sexes, like humans do, where each individual can produce only one kind of gamete. Other anisogamic species are hermaphrodites, where each individual produces both kinds of gamete. Because they produce two kinds of gametes, hermaphroditic species still have two biological sexes they simply combine them in one organism. When a biologist tells you that earthworms are hermaphrodites, they mean that one part of the worm produces sperm and another part produces eggs.

Some single-celled and very simple multicellular organisms have evolved something called mating types. These are gametes that are identical in size and structure, but in which the genome of each gamete contains genetic markers that affect which other gametes it can combine with. Typically, gametes with the same genetic marker cant recombine with one another. Some species have many hundreds of these mating types, and newspapers often report research into this phenomenon under headlines such as: Scientists discover species with hundreds of sexes! But, formally, biologists refer to these as mating types, and reserve the term sexes for gametes that are different in size and structure.

Why distinguish between these two phenomena? One reason is that the evolution of anisogamy gametes that differ in size and structure explains the later evolution of sex chromosomes, sex-associated physical characteristics and much more. But the existence of mating types doesnt have these dramatic knock-on evolutionary effects. Another reason to keep the distinction is that anisogamy and mating types are thought to have evolved via different evolutionary processes. One theory is that anisogamy appeared when mating-type genome markers somehow became linked to genes that controlled the size of the gamete, or mutated in some way that affected gamete size. These differences in gamete size would then kickstart the evolution of sexes.

The evolution of sex seems to be strongly associated with multicellularity, so the obvious place to look for a shift from mating types to sexes is in organisms that sit at the multicellular boundary such as algae, which sometimes exist as single-celled organisms, and sometimes as colonies of cells. And indeed, there are species of algae where gametes are just a little bit anisogamous, blurring the distinction between mating types and sexes. Theres much we dont know about how sex evolved, and how it might have evolved differently across species. But the point is that sexes and mating types are very different phenomena, with different causes and consequences.

The fact that sex evolved in some species but not others tells us something important about how biologists think about sex. Many cultures take the difference between male and female to be something fundamental, and label other natural phenomena such as the Sun and the Moon as male or female. But for biologists, the separation between male and female is no more fundamental or universal than photosynthesis or being warm-blooded. Some species have evolved these things, and some havent. They exist when they do only because of the local advantages they afforded in evolutionary competition.

So why did some species evolve two, distinct sexes? To answer this question, we need to forget about creatures with complex sex organs and mating behaviours. These evolved later. Instead, think of an organism that releases its gametes into the sea, such as coral, or into the air, such as fungal spores. Next, consider that there are two goals that any gamete must achieve if its to reproduce sexually. The first is finding and recombining with another gamete. The second is producing a new individual with enough resources to survive. One widely accepted idea, then, is that the evolution of sexes reflects a trade-off between these goals. Because no organism has infinite resources, organisms can either produce many small gametes, making it more likely that some of them will find a partner, or produce fewer but larger gametes, making it more likely that the resulting individual will have what it needs to survive and thrive.

Since the 1970s, this idea has been used to model how anisogamic species might have evolved from species with only one kind of gamete. As mutations introduce differences in gamete size, two winning strategies emerge. One is to produce a large number of small gametes too small to create viable offspring unless they recombine with a larger, well-provisioned gamete. The other winning strategy is to produce a few, large, well-resourced gametes that can create viable offspring, no matter how small the recombinant they end up merging with. Intermediate approaches, such as producing a moderate number of moderately well-provisioned gametes, dont do well. Organisms that try to follow the middle way end up with gametes less likely to find a partner than smaller gametes, and more likely to have insufficient resources than larger gametes. When the two successful complementary strategies have evolved, fresh evolutionary pressures make the gametes even more distinct from one another. For example, it can be advantageous for the small gametes to become more mobile, or for the large, immobile gametes to send signals to the mobile ones.

Once anisogamy has evolved, it shapes many other aspects of reproductive biology. Most species of limpet shellfish that you see on rocks at the beach are sequential hermaphrodites. When young and small they are male, and when mature and large they become female. This is believed to be because small limpets dont have sufficient resources to produce large female gametes, but theyre capable of producing the smaller male ones. In some other species, successful males can arrest their growth and remain small (and male) for their entire life.

Chromosomes arent male or female because these bits of DNA define biological sex. Its the other way around

Sequential hermaphroditism occurs in the opposite direction too. Australian snorkellers love to spot the large blue males of the eastern blue groper, but its rare to see more than one. Most groper are smaller, brown females. They are all born female and become sexually mature after a few years, when 20 or 30 cm in length. At around 50 cm, they change sex and acquire other male characteristics, such as being blue. Unlike the limpet, the main problem facing a male groper is controlling a territory on the reef, so becoming male when youre small is a losing strategy.

Biology aims to understand the extraordinary diversity of ways in which organisms reproduce themselves, as well as to identify common patterns, and to explain why they occur. In general, organisms become sexually mature when they reach an optimal size for reproduction. This optimal size is often different for the two sexes, because the two sexes represent divergent strategies for reproduction. The limpet and the groper are two of many examples. In constructing these explanations, biological sex is defined as the production of one type of viable anisogamous gamete. If we defined sex in some other way, it would be hard to see the common patterns across the diversity of life, and hard to explain them.

So-called sex chromosomes, such as the XX and XY chromosome pairs seen in humans, are often brandished as something thats fundamental to sex. Its partly the inadequacy of this definition that drives scepticism about the existence of two, discrete biological sexes. Molecular genetics is likely to require a shift from binary sex to quantum sex, with a dozen or more genes each conferring a small percentage likelihood of male or female sex that is still further dependent on micro- and macroenvironmental interactions, writes the gender scholar Vernon Rosario.

But any biologist already knows that theres more to sex determination than chromosomes and genes, and that male and female sex chromosomes are neither necessary nor sufficient to make organisms male and female. Several species of mammal, all rodents of one kind or another, have completely lost the male Y chromosome, but these rats and voles all produce perfectly normal, fertile males. Other groups of species, such as crocodiles and many fish, have neither sex chromosomes nor any other genes that determine sex. Yet they still have two, discrete biological sexes. The Australian saltwater crocodile, whom we met before, lays eggs that are very likely to develop into gigantic, highly territorial males if incubated between 30 and 33 degrees Celsius. At other temperatures, genetically identical eggs develop into much smaller females.

The reality is that chromosomes arent called male or female because these bits of DNA define biological sex. Its the other way around in some species that reproduce using two discrete sexes, those sexes are associated with different bits of DNA. But in other species this association is either absent or unreliable. Medical institutions use a chromosomal definition of sex because they judge, rightly or wrongly, that this is a reliable way of categorising humans. But humans really arent the best place to start when trying to understand sex across the diversity of life.

So much for genes. But perhaps sex could be defined by the physical characteristics that organisms develop, which then add up to constitute an organisms sex? An organism with more female than male characteristics would be more female than male and vice-versa. Thats a reasonable way to think about sex, and this idea of phenotypic sex is widely used. But if we apply the biological definition of sex, some of the individuals who are in the middle as far as sex-associated characteristics go are bona fide members of one biological sex. Others are not clearly members of either biological sex.

Nothing in the biological definition of sex requires that every organism be a member of one sex or the other. That might seem surprising, but it follows naturally from defining each sex by the ability to do one thing: to make eggs or to make sperm. Some organisms can do both, while some cant do either. Consider the sex-switching species described above: what sex are they when theyre halfway through switching? What sex are they if something goes wrong, perhaps due to hormone-mimicking chemicals from decaying plastic waste? Once we see the development of sex as a process and one that can be disrupted it is inevitable that there will be many individual organisms that arent clearly of either sex. But that doesnt mean that there are many biological sexes, or that biological sex is a continuum. There remain just two, distinct ways in which organisms contribute genetic material to their offspring.

Whats more, the physical characteristics of an organism can be labelled as male or female only if there is already a definition of sex. Whats so male about a groper being blue as opposed to brown? Many male organisms are brown. Whats so female about incubating eggs in a womb? After all, in many pipefish and seahorse species the male incubates the eggs in his brood pouch. What makes this part of the hermaphroditic earthworm male and that part female? Gender studies scholars have noticed this logical discrepancy, and some have gone on to argue that the sexes must therefore be defined in terms of gender. But from a biological perspective, what makes an observable physical characteristic male or female is not its association with gender, but its association with something more tangible: the production of one or other of the two kinds of gamete.

This explains why the existence of individuals with combinations of male and female characteristics doesnt show that biological sex is a continuum. These organisms have a combination of characteristics associated with one biological sex and characteristics associated with the other biological sex. They do not have some part of the ability to make small gametes combined with some part of the ability to make large gametes. Their phenotypic sex might be intermediate, but their biological sex is not. Being fully biologically male and fully biologically female hermaphroditic can be an effective evolutionary strategy, and we have encountered several hermaphroditic species already. But making both kinds of gametes incompletely would be an evolutionary dead-end.

Like phenotypic characteristics, sex chromosomes can be more or less reliably associated with biological sex. The eastern three-lined skink, an Australian lizard, has sex chromosomes, and under some circumstances XY skinks become male and XX skinks become female, just as in humans. But in cold nests, every skink becomes male, whatever their chromosomes. By becomes male, biologists mean that they grow up to produce small gametes sperm.

No animal is conceived with the ability to make sperm or eggs (or both). This ability has to grow

This effect of temperature on sex is not surprising, as many reptile species produce genetically identical offspring whose sex is determined by incubation temperature. Whats more surprising is that varying the size of the egg yolk in this species of skink can produce both sexes with the wrong sex chromosomes: XX males and XY females. The skink seems to have three mechanisms for determining sex chromosomes, temperature and hormones in the yolk. This is not a mere quirk of nature. The skink is one of many species that actively control the sex of their offspring, responding to environmental cues that predict whether male or female offspring have better chances of surviving and reproducing.

If all species were like the skink, we probably wouldnt label sex chromosomes as male or female. After all, we dont think of extreme nest temperatures as female and intermediate temperatures as male, merely because they produce male and female crocodiles or male and female geckos. We think of sex chromosomes as male or female because we focus on species where they are reliably associated with the production of male or female gametes.

Sex chromosomes play much the same role in sex determination as nest temperatures and hormones. Theyre simply mechanisms that organisms use to turn genes on and off in offspring so that they develop a biological sex. No animal is conceived with the ability to make sperm or eggs (or both). This ability has to grow, through a cascade of interactions between genes and environments. In some species, once an individual acquires a sex, it remains that sex for the rest of its life. In others, individuals can switch sex one or more times. But in every case, the underlying mechanisms are designed to grow organisms that make either male or female gametes (or both). The other changes the body undergoes as it becomes male, female or hermaphroditic are designed to fit the reproductive strategies that this species has evolved.

These mechanisms by which organisms develop or switch biological sex are complex, and many factors can interfere with them. So they produce a lot of phenotypic diversity. Sometimes, organisms grow up able to make fertile gametes, but otherwise atypical for their biological sex. Sometimes, they grow up unable to make fertile gametes of either kind. This is usually an accident, but sometimes by design. In bees, eggs that arent fertilised develop into males, so male bees have half as many chromosomes as female bees. Meanwhile, all fertilised eggs start to develop into females, but most of them never complete their sexual development. The queen sends chemical signals that block the development of the worker bees ovaries at an early stage. So worker bees are female in the extended sense that they would develop into fertile females if they werent actively prevented from doing so. Occasionally, worker bees manage to evade these controls and lay their own eggs. They are not popular with beekeepers, who select against these mutant strains.

The diversity of outcomes in individual sexual development doesnt mean that there are many biological sexes or that biological sex is a continuum. Whatever the merits of those views for chromosomal sex or phenotypic sex, they are not true of biological sex. A good way to grasp this is to imagine a species that really does have three biological sexes. Biotechnologists have proposed curing mitochondrial diseases by removing the nucleus from an egg with healthy mitochondrial DNA, and inserting a new nucleus containing the nuclear DNA from an unhealthy egg and the nuclear DNA from a sperm. The resulting child would have three genetic parents.

Now imagine if there was a whole species like this, where three different kinds of gametes combined to make a new individual a sperm, an egg and a third, mitochondrial gamete. This species would have three biological sexes. Something like this has actually been observed in slime moulds, an amoeba that can, but need not, get its mitochondria from a third parent. The novelist Kurt Vonnegut imagined an even more complex system in Slaughterhouse-Five (1969): There were five sexes on Tralfamadore, each of them performing a step necessary in the creation of a new individual. But the first question a biologist would ask is: why havent these organisms been replaced by mutants that dispense with some of the sexes? Having even two sexes imposes many extra costs the simplest is just finding a mate and these costs increase as the number of sexes required for mating rises. Mutants with fewer sexes would leave more offspring and would rapidly replace the existing Tralfamadorians. Something like this likely explains why two-sex systems predominate on Earth.

We can also imagine a species where biological sex really does form a continuum. Recall that some algae have slightly anisogamous gametes, much closer together than sperm and eggs. We can imagine a more complex organism using this system, with some slightly smaller gametes and some slightly larger ones. Successful reproduction might require two gametes that, when added together, are big enough but not too big. But the sexually reproducing plants and animals that actually exist all have just two, very different kinds of gamete male and female. Theyre not merely different in size, theyre fundamentally different in structure. This is the result of competition between organisms to leave the greatest number of genetic descendants. In complex multicellular organisms such as plants and animals, we know of only three successful reproductive strategies: two biological sexes in different individuals, two biological sexes combined in hermaphroditic individuals, and asexual reproduction. Some species use one of these strategies, some use more than one.

Human beings have come up with many ways to classify the diversity of individual outcomes from human sexual development. People who want to apply the biological definition of sex to humans should recognise that its ill-suited to do what many human institutions want, which is to sort every individual into one category or another. What sex are worker bees? They are sterile workers whose genome was designed by natural selection to terminate ovary development on receipt of a signal from the queen bee. They share much of the biology of fertile female bees but if someone wants to know Are worker bees really female?, theyre asking a question that biology simply cant answer.

Nor is being a sterile worker a third biological sex alongside male and female. This is easier to see in ants, where there is more than one sterile caste. Workers, soldiers, queens and male flying ants each have specialised bodies and behaviour, but there are not four biological sexes of ant. Workers and soldiers are both female in an extended sense, but not in the full-blown sense that queen ants are female. There is a human imperative to give everything a sex, as mentioned above, but biology doesnt share it.

The biological definition of sex wasnt designed to ensure fair sporting competition, or settle healthcare disputes

Juvenile organisms and postmenopausal human females also cant produce either kind of gamete. Juveniles are assigned to the sex they have started to grow into. But once again, this is more complicated than it seems when we focus only on humans. In almost all mammals, sexual differentiation is initiated by a region of the Y chromosome, so a mammalian egg can become either male or female. In birds, its the other way around the egg carries the sex-determining W chromosome, so sperm can become either male or female. After fertilisation, therefore, we can say that an individual mammal or bird has a sex in the sense that it has started to grow the ability to produce either male or female gametes. With a crocodile or a turtle, though, wed have to wait until nest temperature had its sex-determining effect. But that doesnt mean that we need to create a third biological sex for crocodile eggs!

More importantly, nothing guarantees that any of these organisms, including those with sex chromosomes, will continue to grow to the point where they can actually produce male or female gametes. Any number of things can interfere. From a biological point of view, there is nothing mysterious about the fact that organisms have to grow into a biological sex, that it takes them a while to get there, and that some individuals develop in unusual or idiosyncratic ways. This is a problem only if a definition of sex must sort every individual organism into one sex or another. Biology doesnt need to do that.

In human populations, there are plenty of individuals whose sex is hard to determine. Biologists arent blind to this. The definition of biological sex is designed to classify the human reproductive system and all the others in a way that helps us to understand and explain the diversity of life. Its not designed to exhaustively classify every human being, or every living thing. Trying to do so quickly leads to questions that have no biological meaning.

Human societies cant delegate to biology the job of defining sex as a social institution. The biological definition of sex wasnt designed to ensure fair sporting competition, or to settle disputes about access to healthcare. Theorists who want to use the biological definition of sex in those ways need to show that it will do a good job at the Olympics or in Medicare. The fact that its needed in biology isnt good enough. On the other hand, whatever its shortcomings as an institutional definition, the concept of biological sex remains essential to understand the diversity of life. It shouldnt be discarded or distorted because of arguments about its use in law, sport or medicine. That would be a tragic mistake.

The authors research is supported by the Australian Research Council and the John Templeton Foundation. He would also like to thank Nicole Vincent, Jussi Lehtonen, Stefan Gawronski and Joshua Christie for their feedback on earlier drafts.

See original here:
Sex is real - aeon.co

A little more than a decade ago, seven patients with hemophilia Ba disease caused by a mutation on the F9 gene that prevents patients from forming crucial clotting proteinsvolunteered to be the first humans to receive a gene therapy delivered using an adeno-associated virus as a vector. This particular treatment didnt move past the Phase 1/2 trial because, while it was deemed safe, the patients did not sustain expression of the gene. But two other gene therapies based on an adeno-associated virus (AAV), Luxturna for rare forms of blindness and Zolgensma for spinal muscular atrophy, have since been approved by the US Food and Drug Administration (FDA), and several pharmaceutical companies are now pursuing regulatory approval of AAV-carried gene therapies for hemophilia B.

Recently, scientists followed up with four of those original patients. In a study published in Molecular Therapy in September, they report that the men are still free of any worrisome toxicities related to the treatment. The study wasnt all good news, though. The team also found that after all these years, the men still had elevated levels of AAV-neutralizing antibodies. That means that if an AAV gene therapy is approved to treat their illness, they likely wont be able to benefit from itthe antibodies would chew up the vector before it could insert the corrective gene.

Administration of an AAV gene therapy is essentially a vaccine against AAV, says Lindsey George, a hematologist at the Childrens Hospital of Philadelphia who led the research. Hers was not the first study to identify antibodies as a problem for those receiving AAV gene therapies, but it is the first to show that elevated titers can last this long. This role of AAV neutralizing antibodies is huge, says George, as it stands to undermine the effectiveness of gene therapies.

Because AAVs are viruses, the human immune system creates antibodies upon exposure that recognize and neutralize them in subsequent encounters. Sometimes patients have neutralizing antibodies in their blood before ever having received a gene therapy because theyre exposed to AAVs in the environment.

The ability to effectively modulate the antibody-mediated immune response could make AAV gene therapies far more effective for far more patients than they are now.

Along with high levels of antibodies to the specific AAV vector that theyd receivedAAV2the patients Georges team evaluated also had neutralizing antibodies to several other commonly used AAV vectors, namely, AAV5 and AAV8, she tells The Scientist.

Andrew Davidoff, a pediatric surgeon at St. Jude Childrens Research Hospital who studies AAV gene therapies but was not involved in the study, says, This paper suggests that not only will they not be able to receive a second dose of vector of the same [type of AAV], but potentially even other [types].

If scientists can prevent antibodies from neutralizing the AAV, they would not only give patients like these another opportunity to receive a more effective dose of gene therapy, but it will expand the patients that we can treat with the therapy to include the 3050 percent of patients who have already been exposed to AAVs in the environment, says Giuseppe Ronzitti, who heads a lab focused on gene therapy research at Genethon.

But, Davidoff says, nobody has found a suitable solution yet that is likely to be accepted by patients. The body has evolved over millions of years, this immune system that helps fight off infections. So to overcome that, even temporarily, is not an easy task.

Some immunosuppressant drugs wont work if the body has already developed specific antibodies to a particular pathogen, such as AAV. Scientists are therefore testing combinations of different types of immunosuppressants they hope will prevent the body from attacking AAVs, but these are likely to come with major risks, chiefly, susceptibility to infection.

Another option is plasmapheresisa process in which a persons blood is removed from the body and the cells separated from the plasma so that they can be reinfused without the antibodies found in the plasmabut, like immunosuppressant drugs, the technique is nonspecific and comes with similar risks. Its a matter of risk-benefit with the continued immunosuppression, says Ronzitti.

So scientists have been looking for other ways to control the bodys response to these gene therapy vectors.

Ronzitti and his team recently proposed a solution in Nature Medicine. The scientists used the imlifidase (IdeS) protein, conditionally approved by the European Commission, to degrade immunoglobulin G (IgG) antibodies that are developed after the body encounters a specific antigen so that it can remember and target that antigen in the future, and thus might cause a patient to reject a transplanted kidney. IgG antibodies are responsible for the immune systems response to AAVs. Its a newer, less invasive alternative to plasmapheresis, Ronzitti tells The Scientist in an email.

The team injected monkeys with the IdeS protein before administering a dose of gene therapy targeting the liver. The treatment appeared safe, the monkeys levels of preexisting AAV antibodies went down, and the AAV vector successfully made its way to the liver. To model a scenario in which a patient would need more than one dose of gene therapy, the scientists administered an AAV gene therapy to another group of monkeys before giving them the IdeS protein to degrade the antibodies theyd developed in response, then readministered the gene therapy. Again, AAV antibodies diminished after the IdeS treatment and the second gene therapy dose was successfully delivered.

One drawback to the approach is that IgGs are the most prevalent type of antibody found in the blood, and destroying all of them may have undesirable side effects. In an attempt to develop a more targeted therapy, one group published a study in January demonstrating that a specialized version of plasmapheresis could reduce the levels of antibodies against human AAVs in mice to the point where a new gene therapy should be effective, without depleting all other immunoglobulins that formed in response to infections.

More recently, a team of researchers at the University of Pittsburgh Medical Center made use of CRISPR-Cas9 to increase the efficacy of AAV gene therapy in mice. Pathologist Samira Kiani and her team werent looking for ways to improve gene therapy, but instead were seeking to temporarily modulate immunity in hopes of changing the course of diseases such as septicemia, a precursor to sepsis that occurs when an infection makes its way to the blood. The researchers aimed to temporarily downregulate the Myeloid differentiation primary response 88(Myd88) gene, which would briefly dampen the immune response, and then remove the brakes.

The gene that we chose to target is known to a be a central gene for innate and adaptive immunity, says Kiani. It controls the production of IgG antibodies in response to AAV exposure, which provided a simple way to measure whether the strategy was effective. If the team administered an AAV to an animal shortly after it had received the CRISPR-Cas9 treatment, it should have a substantially lower antibody response to the virus.

First, they administered the CRISPR to tamp down Myd88 activity and measured a reduction in the expression of the Myd88 gene, as theyd expected. Then, the team used the technique to treat mice just before giving them a dose of AAV-based gene therapy that was designed to lower their cholesterol.

Weeks later, the researchers administered a second dose of the same AAV vector to determine if the temporary immunosuppression during the first dose had prevented the mice from making enough antibodies to thwart a second dose. The mice that were pretreated with the immune-modulating CRISPR showed lower levels of AAV-neutralizing antibodies and more dramatic responses to the cholesterol-lowering AAV treatment. The study was published in NatureCell Biologyin September.

If given prior to the administration of an AAV gene therapy, this approach would prevent the formation of new antibodies, so the patient could receive a second dose later, if needed, says Kiani. Given that the CRISPR treatment only prevents the development of antibodies temporarily, it shouldnt cause any long-term suppression of the rest of the immune system. On the flip side, because it doesnt clear existing antibodies, if the patients have already pre-existing antibodies [from natural exposure] this approach might not be the best approach.

All of the potential solutions have a long way to go, including still needing to be tested in human patients, but the ability to effectively modulate the antibody-mediated immune response could make AAV gene therapies far more effective for far more patients than they are now, says Ronzitti. The immune response to these vectors is quite a complex story, he says. But we are solving the issues one by one.

L. George et al., Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B,Molecular Therapy,doi:10.1016/j.ymthe.2020.06.001, 2020.

F. Moghadam et al., Synthetic immunomodulation with a CRISPR super-repressor in vivo,Nature Cell Biology,doi:10.1038/s41556-020-0563-3, 2020.

Read more here:
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy - The Scientist

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Gene therapies have graduated from the laboratory bench to doctor's offices. The first wave of agents, Roche's Luxturna and Novartis' Zolgensma, are already altering the course of disease in two conditions, respectively an inherited form of blindness and the degenerative, often fatal disease spinal muscular atrophy.

In both cases those treatments were developed by companies that had relatively smaller pipelines, Spark Therapeutics for Luxturna and AveXis for Zolgensma.

Enter Taysha, which is headed by a former AveXis business development vice president, R.A. Session II. The company has highly ambitious hopes to launch a new product every two to three years, with the goal of building a durable business around adapting its technology across many diseases driven by defects in single genes.

The company also plans to build a commercial-scale manufacturing plant from the start, aiming to avoid some of the setbacks that can occur when production moves from facilities built to supply clinical trials.

The linchpin of the company's business is an agreement with UT-Southwestern, under which Taysha funds research and can obtain exclusive rights to experimental therapies for central nervous system disorders, through the end of 2021. Neurodegenerative disorders have proven challenging for some gene therapies because of the difficulties in delivering the viral vectors that carry gene replacements to brain tissue.

In spinning out Taysha, UT-Southwestern took an ownership stake in Taysha, amounting to 2.2 million shares, which is now worth more than $40 million. These ownership stakes have become more common with gene therapies in particular, as big pharma companies have been reluctant to license intellectual property straight out of university laboratories.

Taysha's lead project is called TSHA-101, which seeks to treat a condition called GM2 gangliosidosis, a disorder in which lipid accumulation destroys nerves in the brain and spinal cord. The first clinical trial is scheduled to begin in Canada by the end of 2020.

The company's shares rose following their first trades on the NASDAQ exchange, gaining 20% to close the day at $24.06.

Editor's note: This story was updated to reflect the share price at the close of trading.

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Gene therapy company Taysha completes sprint from first funding to IPO - BioPharma Dive

DetailsCategory: DNA RNA and CellsPublished on Friday, 25 September 2020 11:15Hits: 398

BioMarin Builds Upon 15+ Year Commitment to PKU Community with Potential 3rd Therapy in PKU Franchise

Company Leverages Gene Therapy Manufacturing Expertise Using Commercial-Ready Process

SAN RAFAEL, CA, USA I September 24, 2020 I BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

BioMarin will conduct this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

"More than 70 years ago, the first child was treated for PKU in the United Kingdom at Birmingham Women's and Children's Hospital. Today, we continue to make strides in PKU treatment through the clinical study of a gene therapy for PKU," said Tarekegn G. Hiwot at University Hospitals Birmingham NHS Foundation Trust and principal investigator for the PHEARLESS study. "There is a tremendous unmet need for PKU patients. As a treating physician, it is important to me to be involved in clinical research to evaluate innovative therapies that have the potential to change the treatment paradigm in PKU for good."

"BioMarin has been committed to the PKU community for more than 15 years and remains dedicated to the research and development of innovative therapies to advance the standard of care for people with PKU," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "Building upon our experience of delivering two approved PKU therapies to the PKU community, BMN 307 gene therapy combines BioMarin's leadership in the development of PKU therapies with our expertise in gene therapy development and manufacturing."

"PKU is a serious condition and many individuals struggle to manage their disorder on a daily basis. BioMarin is a pioneer in PKU treatments delivering the first two drug therapies to individuals with PKU. We applaud their unwavering commitment to drive research to bring a third treatment to the PKU community and for their substantial contributions to the overall body of scientific knowledge in PKU that they continue to make," said Christine S. Brown, MS, Executive Director, National PKU Alliance. "We are encouraged by BioMarin's efforts to develop a gene therapy that brings together their experience in PKU drug development, gene therapy development and gene therapy manufacturing. "

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation. The Company is actively preparing regulatory submissions to open additional clinical sites in other countries.

BMN 307 Clinical Program

BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, PHENOM, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California that was cGMP certified by the EMA in Q2 2020. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

SOURCE: BioMarin Pharmaceutical

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy | DNA...

Dive Brief:

Zolgensma's approval was a notable milestone, making it both the second gene therapy approved in the U.S., and the second treatment for SMA, a potentially deadly disease for which no medicines existed until four years ago.

But it's been a bumpy ride for Novartis since that 2019 approval. The FDA charged the company of manipulating preclinical data supporting its original application, a controversy that led Novartis to fire some top scientists and shake up its quality control operations. (The agency opted against sanctioning Novartis, however.)

And the drugmaker has now faced multiple delays in its effort to broaden the reach of Zolgensma beyond newborns and young infants.

That effort is crucial to the commercial prospects of Zolgensma. While SMA diagnosed in infancy, known as Type 1, is the most deadly, more people are living with the less severe forms of the disease. The nonprofit SMA foundation, for instance, estimates 88% of patients living with the disease have either Type 2 or Type 3, which can either stop people from walking or rob them of that ability later on.

Younger SMA patients need the muscle-boosting protein Zolgensma helps create to be expressed throughout the body, while older patients require a more targeted approach, which is why Novartis is developing the intrathecal version for them.

Currently, intravenous Zolgensma is approved in the U.S. and Japan for patients under two, and in Europe for children who weigh up to 21 kilograms. By comparison, Biogen's Spinraza and recently, Roche's Evrysdi are approved for much wider groups of SMA patients.

The intrathecal dose of Zolgensma is meant to even that playing field, though it may now be a few years until Novartis can bring that version to market.

The FDA initially halted testing of the intrathecal formulation in October, citing safety concerns from an animal study. That test is still on hold, but even when it restarts, Novartis will need more time to complete the new trial requested by the FDA. The company said the two developments are unrelated.

Novartis is discussing the details of the trial with the agency and said it will give a "comprehensive update" on its SMA program in the future. The pharma had been planning an approval submission next year, but Jefferies analyst Peter Welford now predicts that won't occur until at least 2023.

The delay boosts Roche's and Biogen's drugs, delaying would-be competition in older SMA patients. Novartis noted it's developing a similar type of drug to Roche's Evrysdi that's taken once a week orally.

Some 600 patients have been treated with Zolgensma through clinical trials, expanded access programs and commercially.

News of the FDA's request sent shares of gene therapy developer Regenxbio, which holds royalty rights to Zolgensma, down by as much as 9% Wednesday morning.

Excerpt from:
FDA sets back Novartis plans to expand use of SMA gene therapy - BioPharma Dive

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in two upcoming virtual investor conferences.

Jefferies Virtual Gene Therapy/Editing SummitDate: Thursday, Oct. 1, 2020Time: 12:30 p.m. ET

Chardan Virtual 4th Annual Genetic Medicines ConferenceDate: Tuesday, Oct. 6, 2020Time: 2:00 p.m. ET

Live webcasts of the presentations will be available on the investors section of the AVROBIO website at avrobio.com. After the live webcasts, the events will remain archived on the AVROBIO website for 90 days.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO to Present at Two Upcoming Investor Conferences - Business Wire

Some AAV manufacturing processes use adherent cells grown in stacked trays. AGTC sees that model as a barrier to scaling, pointing to the inability to scale processes up by adding volume. Scaling of adherent cell processes requires additional surface area.

AGTC overcame that limitation by designing a process that makes use of bioreactors, enabling it to scale up from 50 L to 500 L and beyond without significantly increasing its footprint. AGTC enhanced the approach with changes it says improved yield by more than 10 fold.

We adjusted the upstream process which uses two HSV helper viruses to increase AAV production in a number of ways, one of which was optimizing the ratio and amount of the two viruses. Further, we optimized the bioreactor conditions to increase cell density. Both of these changes led to substantial increases in volumetric productivity, said AGTC CEO Sue Washer.

Adjusting the ratios and conditions of the process enabled AGTC to make more vectors and increase the percentage of them that are full. AGTC is now achieving finished product specifications that demonstrate nearly 90% full capsids.

Washer went on to say that AGTC also optimized the column chromatography purification process to more completely separate residuals from vectors. In doing so, AGTC was able to collect a higher quality product from the elution peak, according to Washer.

Using the process, AGTC estimates it could generate 2,000 ophthalmology gene therapy doses from a 50-L manufacturing run. That would enable AGTC to support its move into late-phase clinical trials using a relatively small manufacturing footprint.

The improvements could prove particularly valuable to AGTC as it expands beyond diseases affecting the eye. The gene therapy doses needed to treat ocular diseases are far smaller than those required to address conditions affecting other organs, which typically need to be given systemically.

The industry-wide move from rare diseases treated via local delivery to more common conditions that require systemic administration is putting a strain on manufacturing capacity. If AGTC is right, its process will equip it to manage the manufacturing side of the shift in therapeutic focus better than some other gene therapy companies.

AGTC is now advancing programs that will enable it to start putting that hypothesis to the test. While AGTCs clinical pipeline is focused on rare genetic eye diseases, the biotech is working to get gene therapies for central nervous system disorders into human testing. Washer is alert to the implications of AGTCs manufacturing process.

Improved yields makes use of gene therapy for larger patient populations or systemic diseases much more achievable, said Washer.

AGTC is working to further improve the system. Ongoing areas of activity relate to purification, where Washer sees opportunities to eliminate all process residuals, and the improvement of yields and the percentage of full capsids.

The nature of the process means manufacturing runs could become more productive even in the absence of further technological advances. Simply running the same process at a larger scale should confer some benefits.

Continuing to scale to larger bioreactors could continue to increase the number of doses from a single run and drive down costs as bioreactor scale-up does not put a burden on factory space or FTE needed like the use of plastic flat-stock does, said Washer.

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Using HSV helper viruses to increase AAV production - BioPharma-Reporter.com

NEW YORK, Sept. 23, 2020 /PRNewswire/ --Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today the signing of a Sponsored Research Agreement (SRA) with Duke University to use the MPTP-induced Parkinson's Disease (PD) mouse model to establish in vivo proof-of-concept study to demonstrate that administration of LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the efficacy and safety of SLS-004. Subsequently, other pre-clinical models would be utilized to further validate the investigational product.

"We are extremely pleased to begin the in vivo target engagement study with SLS-004 at Duke as it builds onto the work we began in the spring focused on designing a vector capable of inducing and suppressing Parkinson's related phenotypes," said Raj Mehra Ph.D., Chairman and CEO of Seelos. "Initiating this next portion of the studies at Duke should help us further validate this approach in Parkinson's."

Seelos has also begun work on designing a vector capable of inducing and suppressing PD-related phenotypes by carrying a unit to overexpress the alpha-synuclein (-synuclein) protein and a unit to mediate inducible suppression of -synuclein. The process aims to create a next-generation suppressive unit, carrying a more effective effector molecule than previously used, in the form of DNA methyltransferase 3A and 3L (DNMT3A & L). This research may help to advance a potential novel, effective and precise tool for reversing SNCA pathologies that can provide a valuable new therapeutic strategy for treating PD.

About SLS-004

SLS-004 is a novel epigenome-editing approach to modulate expression of SNCA gene mediated by modification of DNA-methylation. SLS-004 utilizes an all-in-one lentiviral vector harboring dCas9-DNA methyltransferase 3A (DNMT3A) to enrich DNA-methylation within CpGs island at the SNCA intron 1 region. The system resulted in a precise and fine-tuned downregulation (30%) of SNCA overexpression in hiPSC-derived dopaminergic neurons from a PD patient with the triplication of the SNCA locus (SNCA-Tri). Most importantly, the reduction of SNCA expression mediated by the developed system was sufficient to ameliorate disease related cellular phenotypes. The in vitro studies achieved several key millstones including the establishment that DNA hypermethylation at SNCA intron 1 allows an effective and sufficient tight downregulation of SNCA expression levels and suggests the potential of this target sequence combined with the CRISPR-dCas9 technology as a novel epigenetic-based therapeutic approach for PD.

Forward Looking Statements

Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding the initiation of a proof-of-concept study to demonstrate whether administration of the LV-dCas9-DNMT3A virus can prevent and/or delay PD, the potential for the LV-dCas9-DNMT3A virus to prevent and/or delay PD, the efficacy and safety of SLS-004, the potential use of other pre-clinical models to validate SLS-004, the potential for the in vivo proof-of-concept study to validate the design of a vector capable of inducing and suppressing PD-related phenotypes, and the potential for Seelos' research to advance a novel, effective and precise tool for reversing SNCA pathologies. These statements are based on Seelos' current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies and not gaining marketing approvals for its product candidates, the risk that prior test results may not be replicated in future studies and trials, the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval, the risks associated with the implementation of a new business strategy, the risks related to raising capital to fund its development plans and ongoing operations, risks related to Seelos' current stock price, risks related to the global impact of COVID-19, as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.

Contact Information:Anthony MarcianoHead of Corporate CommunicationsSeelos Therapeutics, Inc. (Nasdaq: SEEL)300 Park Ave., 12th FlNew York, NY 10022(646) 293-2136[emailprotected]www.seelostherapeutics.comhttps://twitter.com/seelostxhttps://www.linkedin.com/company/seelos

SOURCE Seelos Therapeutics, Inc.

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Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson's Disease -...

The global Cancer Gene Therapy Market is subject to the arrival of numerous trends in the industry, which in turn is influencing the overall growth of the market. The Cancer Gene Therapy Market survey report is a product of an exhaustive analysis concerning the latest trends in the Cancer Gene Therapy Market. The report contains the market definition, fundamental applications in the market, as well as the different manufacturing methods employed. Our market survey report estimates the current market valuation to be at Cancer Gene Therapy Market, and with the help of the industry variable, we have predicted the market valuation to be at Cancer Gene Therapy Market by the end of the forecast period 2020-2027. We also predict the CAGR growth that will be reached by the end of the forecast period.

The major vendors covered: Bluebird bio, Inc., Merck, Adaptimmune, GlaxoSmithKline, Anchiano Therapeutics, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Celgene, Shanghai Sunway Biotech, OncoGenex Pharmaceuticals, and more

Get a free sample copy @ https://www.datalibraryresearch.com/sample-request/cancer-gene-therapy-market-2470?utm_source=thedailychronicle&utm_medium=39

Through our research, we aim to first introduce the reader to the market and inform them about the different areas of growth within the industry. This will include the different segments by which the Cancer Gene Therapy Market is divided, as well as a thorough regional analysis of the market. The market is segmented based on the product type, application, distribution channel, and region. Based on these different factors, we seek to understand the ability of the market to grow over the forecast period. We take into consideration the different socio-economic, political, and environmental fluctuations that could impact the overall growth of the market. Our Cancer Gene Therapy Market survey report provides a detailed and in-depth understanding of the industry and will help in understanding the business climate in a much better way.

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Through our report, we provide the readers with an understanding of the main drivers of the Cancer Gene Therapy Market. This includes a detailed analysis of the factors that create demand and the reasons for the same. We also delve into the many risks that are faced by the market, and how these play an important role in inhibiting the growth of the market.

Regional Description

After a general global overview of the Cancer Gene Therapy Market, we segment the market based on regions in order to better understand the different factors that drive the growth in these areas. Our Cancer Gene Therapy Market survey report includes North America, South America, Europe, Asia Pacific, Middle East, and Africa. We study the different trends in these regions and also analyse the reason why some regions are able to assert more dominance with regards to market share. We can also ascertain the different opportunities that could benefit the market in the long run.

Method of Research

Our Cancer Gene Therapy Market research methodology involves an analysis of the market during the forecast period with the help of the various parameters provided by Porters Five Force Model. A SWOT based analysis helps to further examine the main strengths, weaknesses, risks, and opportunities that are prevalent in the market, in order to give an in-depth understanding of the markets growth trajectory.

Key Players

Throughout our report, we give attention to the key players involved in the Cancer Gene Therapy Market. We discuss the market shares held by these companies and also try to understand their business operations influence on the Cancer Gene Therapy Market. We also keep the readers updated with the latest newsfrom the industry, including important partnerships and acquisitions.

If you have any special requirements about Cancer Gene Therapy Market report, please let us know and we can provide custom report.

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TipRanks

Markets are volatile, there can be no doubt. So far this month, the S&P 500 has fallen 9% from its peak. The tech-heavy NASDAQ, which had led the gainers all summer, is now leading the on the fall, having lost 11% since September 2. The three-week tumble has investors worried that we may be on the brink of another bear market.The headwinds are strong. The usual September swoon, the upcoming election, doubts about another round of economic stimulus all are putting downward pressure on the stock markets.Which doesnt mean that there are no opportunities. As the old saw goes, Bulls and bears can both make money, while the pigs get slaughtered. A falling market may worry investors, but a smart strategy can prevent the portfolio from losing too much long-term value while maintaining a steady income. Dividend stocks, which feed into the income stream, can be a key part of such a strategy.Using the data available in the TipRanks database, weve pulled up three stocks with high yields from 7% to 11%, or up to 6 times the average dividend found on the S&P 500 index. Even better, these stocks are seen as Strong Buys by Wall Streets analysts. Lets find out why.Williams Companies (WMB)We start with Williams Companies, an Oklahoma-based energy company. Williams controls pipelines connecting Rocky Mountain natural gas fields with the Pacific Northwest region, and Appalachian and Texan fields with users in the Northeast and transport terminals on the Gulf Coast. The companys primary operations are the processing and transport of natural gas, with additional ops in crude oil and energy generation. Williams handles nearly one-third of all US commercial and residential natural gas use.The essential nature of Williams business really, modern society simply cannot get along without reliable energy sources has insulated the company from some of the economic turndown in 1H20. Quarterly revenues slid from $2.1 billion at the end of last year to $1.9 billion in Q1 and $1.7 billion in Q2. EPS in the first half was 26 cents for Q1 and 25 cents for Q2 but this was consistent with EPS results for the previous three quarters. The generally sound financial base supported the companys reliable dividend. Williams has been raising that payment for the past four years, and even the corona crisis could not derail it. At 40 cents per common share, the dividend annualizes to $1.60 and yields an impressive 7.7%. The next payment is scheduled for September 28.Truist analyst Tristan Richardson sees Williams as one of the midstream sectors best positioned companies.We continue to look to WMB as a defensive component of midstream and favor its 2H prospects as broader midstream grasps at recovery Beyond 2020 we see the value proposition as a stable footprint with free cash flow generation even in the current environment. We also see room for incremental leverage reduction throughout our forecast period on scaled back capital plans and even with the stable dividend. We look for modestly lower capex in 2021, however unlike more G&P oriented midstream firms, we see a project backlog in downstream that should support very modest growth, Richardson noted.Accordingly, Richardson rates WMB shares as a Buy, and his $26 price target implies a 30% upside potential from current levels. (To watch Richardsons track record, click here)Overall, the Strong Buy analyst consensus rating on WMB is based on 11 Buy reviews against just a single Hold. The stocks current share price is $19.91 and the average price target is $24.58, making the one-year upside potential 23%. (See WMB stock analysis on TipRanks)Magellan Midstream (MMP)The second stock on our list is another midstream energy company, Magellan. This is another Oklahoma-based firm, with a network of assets across much of the US from the Rocky Mountains to the Mississippi Valley, and into the Southeast. Magellans network transports crude oil and refined products, and includes Gulf Coast export shipping terminals.Magellan's total revenues rose sequentially to $782.8 in Q1, and EPS came in at $1.28, well above the forecast. These numbers turned down drastically in Q2, as revenue fell to $460.4 million and EPS collapsed to 65 cents. The outlook for Q3 predicts a modest recovery, with EPS forecast at 85 cents. The company strengthened its position in the second quarter with an issue of 10-year senior notes, totaling $500 million, at 3.25%. This reduced the companys debt service payments, and shored up liquidity, making possible the maintenance of the dividend.The dividend was kept steady at $1.0275 per common share quarterly. Annualized, this comes to $4.11, a good absolute return, and gives a yield of 11.1%, giving MMP a far higher return than Treasury bonds or the average S&P-listed stock.Well Fargo analyst Praneeth Satish believes that MMP has strong prospects for recovery. [We] view near-term weakness in refined products demand as temporary and recovering. In the interim, MMP remains well positioned given its strong balance sheet and liquidity position, and ratable cash flow stream Satish goes on to note that the dividend appears secure for the near-term: The company plans to maintain the current quarterly distribution for the rest of the year.In line with this generally upbeat outlook, Satish gives MMP an Overweight (i.e. Buy) rating, and a $54 price target that implies 57% growth in the coming year. (To watch Satishs track record, click here)Net net, MMP shares have a unanimous Strong Buy analyst consensus rating, a show of confidence by Wall Streets analyst corps. The stock is selling for $33.44, and the average price target of $51.13 implies 53% growth in the year ahead. (See MMP stock analysis on TipRanks)Ready Capital Corporation (RC)The second stock on our list is a real estate investment trust. No surprise finding one of these in a list of strong dividend payers REITs have long been known for their high dividend payments. Ready Capital, which focuses on the commercial mortgage niche of the REIT sector, has a portfolio of loans in real estate securities and multi-family dwellings. RC has provided more than $3 billion in capital to its loan customers.In the first quarter of this year, when the coronavirus hit, the economy turned south, and business came to a standstill, Ready Capital took a heavy blow. Revenues fell by 58%, and Q1 EPS came in at just one penny. Things turned around in Q2, however, after the company took measures including increasing liquidity, reducing liabilities, and increasing involvement in government-sponsored lending to shore up business. Revenues rose to $87 million and EPS rebounded to 70 cents.In the wake of the strong Q2 results, RC also started restoring its dividend. In Q1 the company had slashed the payment from 40 cents to 25 cents; in the most recent declaration, for an October 30 payment, the new dividend is set at 30 cents per share. This annualizes to $1.20 and gives a strong yield of 9.9%.Crispin Love, writing from Piper Sandler, notes the companys success in getting back on track.Given low interest rates, Ready Capital had a record $1.2B in residential mortgage originations versus our $1.1B estimate. Gain on sale margins were also at record levels. We are calculating gain on sale margins of 3.7%, up from 2.4% in 1Q20, Love wrote.In a separate note, written after the dividend declaration, Love added, We believe that the Board's actions show an increased confidence for the company to get back to its pre-pandemic $0.40 dividend. In recent earnings calls, management has commented that its goal is to get back to stabilized earnings above $0.40, which would support a dividend more in-line with pre-pandemic levels.To this end, Love rates RC an Overweight (i.e. Buy) along with a $12 price target, suggesting an upside of 14%. (To watch Loves track record, click here)All in all, Ready Capital has a unanimous Strong Buy analyst consensus rating, based on 4 recent positive reviews. The stock has an average price target of $11.50, which gives a 9% upside from the current share price of $10.51. (See RC stock analysis on TipRanks)To find good ideas for dividend stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

The rest is here:
PTC Therapeutics to Present at the Jefferies Virtual Gene Therapy/Editing Summit - Yahoo Finance

This press release was orginally distributed by SBWire

Albany, NY -- (SBWIRE) -- 09/25/2020 -- Transparency Market Research (TMR) has published a new report titled, "Gene Therapy Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026". According to the report, the global gene therapy market was valued at US$ 17.0 Mn in 2017 and is projected to expand at a CAGR of 40.0% from 2018 to 2026. New product approvals, promising therapeutic outcomes of gene therapy, and high prevalence of non-Hodgkin Lymphoma are anticipated to drive the global market in the next few years. Europe is projected to dominate the global gene therapy market, followed by U.S., by the end of 2026.

Potential unmet needs in the fields of oncology, rare genetic disorders in the U.S. and Europe, new product approvals and commercialization, and high clinical R&D budgets are likely to drive the gene therapy market in these regions during the forecast period. The gene therapy market in Rest of World is projected to expand at a significant CAGR during the forecast period. The high growth rate is attributed to the anticipated approval and commercialization of gene therapy products in developed countries such as Japan, Australia & New Zealand, GCC countries, and China, and high prevalence of non-Hodgkin Lymphoma and head and neck cancers.

View Report : https://www.transparencymarketresearch.com/gene-therapy-market.html

gene therapy marketNew product approvals & commercialization drives market

2016, 2017, and 2018 were key milestones in the history of the gene therapy market in the U.S. and Europe, as around four gene therapy products have been approved and commercialized. These products are currently in the infancy stage of commercialization, and have exhibited highly positive therapeutic outcomes.

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For instance, in May 2016, GlaxoSmithKline (GSK) gene therapy product, Strimvelis, received marketing approval for the treatment of patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency, due to Adenosine Deaminase deficiency). Strimvelis is the first ex-vivo stem cell gene therapy to be approved in Europe for the treatment of ADA-SCID. Furthermore, in August 2017, the USFDA approved Novartis AG's flagship gene therapy product, Kymriah, for the treatment of children and adults up to the age of 25 years affected with B-cell precursor acute lymphoblastic leukemia (ALL). Thus, recent approvals of gene therapy products in the U.S. and Europe for the treatment of various life threatening disorders is projected to fuel the gene therapy market during the forecast period.

Yescarta to be highest revenue generating gene therapy product

The report offers a detailed segmentation of the global gene therapy market based on different gene therapy products approved and commercialized. Based on product, the global gene therapy market has been segmented into Yescarta, Kymriah, Luxturna, Strimvelis, and Gendicine. Yescarta (Axicabtagene Ciloleucel) is a genetically modified autologous Chimeric Antigen Receptor T (CAR T) cell immunotherapy developed by Gilead Sciences, Inc. for the treatment of adult patients with relapsed or refractory large B-cell lymphoma including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL). It is the first CAR T therapy approved by the US FDA for the treatment of DLBCL. The Yescarta segment is projected to dominate the global gene therapy market by the end of 2026. Anticipated commercialization of Yescarta in Europe and other developed countries and increasing number of treatment centers are key factors that are likely to lead to the dominant share held by Yescarta by the end of 2026.

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Oncology segment to account for high market share

In terms of application, the global gene therapy market has been segmented into ophthalmology, oncology, and adenosine deaminase ?deficient severe combined immunodeficiency (ADA-SCID). The oncology segment is likely to account for a significant share of the market by the end of 2026. Oncology is a highly studied medical field in the clinical pipeline studies of gene therapy candidates. More than 60% of gene therapy clinical research studies are focused on oncology. The large share held by the oncology segment is attributed to the approval and commercialization of Yescarta and Kymriah, in the last one to two years, for the treatment of certain types of non-Hodgkin lymphoma in the U.S. and Europe. Furthermore, increase in demand for Gendicine in China for the treatment of head and neck cancers is projected to drive the segment during the forecast period.

Europe offers high incremental opportunity

The gene therapy market in Europe is projected to expand at a significant CAGR of 30.6% during the forecast period. Large number of patient population with refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), promising therapeutic outcomes, rising demand for gene therapy treatment, and increasing number of gene therapy treatment centers in Europe are key factors that are likely to fuel the gene therapy market in Europe. Moreover, different pricing models are being evaluated by payers and governments to enable access to high priced gene therapy products. This is likely to drive the demand for gene therapy products in Europe during the forecast period.

Read our Case study at : https://www.transparencymarketresearch.com/casestudies/innovative-medical-device-manufacturing-start-up

Large number of clinical pipeline studies and significant investments in gene therapy to gain the first mover advantage

The global gene therapy market is highly consolidated, with very few global players accounting for a major share. Currently, only five companies; Gilead life Sciences Inc. Spark Therapeutic Inc., Novartis AG, Sibiono GeneTech Co. Ltd, and Orchard Therapeutics Limited offer gene therapy products in the market. Most biopharmaceutical companies have invested significantly in clinical R&D for the development of gene therapy products for different chronic and genetic disorders. Large number of gene therapy products are under different stages of clinical pipeline studies, and the number of gene therapy candidates is projected to rise consistently during the forecast period. For instance, according to the Journal of Gene Medicine, there were around 2,597 gene therapy candidates under clinical trials, as of 2017. Of the total clinical studies, around 65% of studies were focused on oncology, 11% of studies were focused on monogenetic field, 7% on infectious diseases and cardiovascular disease, each.

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Gene Therapy Market Trends by Manufacturers, States, Type and Application - Press Release - Digital Journal

Overview Of Gene Therapy Industry 2020-2025:

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The Gene Therapy Market analysis summary by Reports Insights is a thorough study of the current trends leading to this vertical trend in various regions. Research summarizes important details related to market share, market size, applications, statistics and sales. In addition, this study emphasizes thorough competition analysis on market prospects, especially growth strategies that market experts claim.

Gene Therapy Market competition by top manufacturers as follow:Kite PharmaBioVexNovartisSpark Therapeutics

Get a Sample PDF copy of the report @ https://reportsinsights.com/sample/7583

The global Gene Therapy market has been segmented on the basis of technology, product type, application, distribution channel, end-user, and industry vertical, along with the geography, delivering valuable insights.

The Type Coverage in the Market are: Ex VivoIn Vivo

Market Segment by Applications, covers:Cancer DiseasesHematological DiseaseHereditary DiseaseOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

Major factors covered in the report:

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The analysis objectives of the report are:

Access full Report Description, TOC, Table of Figure, Chart, [emailprotected] https://reportsinsights.com/industry-forecast/Gene-Therapy-Market-7583

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Gene Therapy Market: Technological Advancement & Growth Analysis with Forecast to 2025 - The Daily Chronicle

LEXINGTON, Mass., Sept. 25, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced CEO Fred Chereau will present at the Jefferies Virtual Gene Editing/Therapy Summit on Friday, October 2, 2020 at 11:30 AM, ET.

A live audio webcast of the presentation will be available under the Events and Presentations section of LogicBios website. A replay of the presentation will become available approximately one hour after the event and will be archived for 30 days.

About LogicBio Therapeutics

LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Lexington, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families. For more information, please visit http://www.logicbio.com

Contacts:

Investors:Matthew Lane

Gilmartin Investor Relation

matt@gilmartinir.com

Media:Stephanie Simon

TenBridge Communications

Stephanie@tenbridgecommunications.com

617-581-9333

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LogicBio Therapeutics to Present at the Jefferies Virtual Gene Editing/Therapy Summit October 2, 2020 - GlobeNewswire

DetailsCategory: DNA RNA and CellsPublished on Friday, 25 September 2020 11:52Hits: 367

Focus on effect of SVecTM on downregulation of pathological immune responses underlying the destruction of own cells in DM1 and in MS patients

Investment of 0.6 million (US$0.7 million) in animal proof-of-principle studies

LEIDEN, The Netherlands and SEVILLE, Spain I September 25, 2020 I Amarna Therapeutics, a Dutch privately held biotechnology company developing the next-generation SV40-based gene delivery vector platform transforming gene-replacement and immunotherapy across many disease areas. The company today announced it has entered into a collaboration with scientists from the Progreso y Salud Foundation (FPS) at Cabimer in Seville, to jointly examine the efficacy of Amarnas SVecTM gene delivery vector to develop effective immunotherapies for diabetes mellitus type 1 (DM1) and multiple sclerosis (MS).

The collaboration is a joint effort between the research group of the FPS at research institute Cabimer, led by Dr. Benoit Gauthier, and Amarna Therapeutics, represented by Dr. Peter de Haan (CSO) and Miguel Garca Toscano (Head of Laboratory in Spain).

To date, the symptoms of DM1 and MS can be managed, but patients cannot be cured from both autoimmune diseases. The aim of this joint effort is to study the efficacy of Amarnas SV40-based gene delivery vector platform, denoted SVecTM, for downregulation of pathological immune responses that underlie the destruction of own cells in DM1 and in MS patients.

The research will focus on the induction of SVecTM-mediated immune tolerance to the primary self-antigens of both diseases. The studies will use advanced animal models of both autoimmune diseases, that have been established by the collaboration partners. Amarna will invest some 0.6 million over the next two years in the Gauthier research group to conduct the animal proof-of-principle studies for these two indications, for which at present there are no cures available.

Benoit Gauthier, Staff Scientist at Junta de Andalucia-Consejeria de Salud y Familias, comments:

We are thrilled to start this new venture with Amarna Therapeutics, a world leader in viral gene therapy and we anticipate the studies to generate exciting results

Peter de Haan, Amarna Therapeutics Chief Scientific Officer, adds:

We are delighted entering this collaboration with such a renowned academic partner like FPS and we look very much forward to initiate the planned studies. Since the quality of life for patients with DM1 and MS is so severely impaired given the lack of cures for these invalidating diseases, the more efficiently we can develop our groundbreaking SV40-based gene delivery vector based therapies, the sooner patients will experience the positive impact of our solution on their lives.

About FPS

For more than ten years, Benoit Gauthier's research group has focused on the field of diabetes and recently other autoimmune diseases. Its basic quality research has generated important new knowledge which enable the development of new therapies for this disease cluster. An important finding of the group was the mandatory association of immune responses to pancreatic beta cells with their capacity to regenerate in patients with type 1 diabetes. In addition, the discovery of the PAX8 gene, and the relationship between type 2 diabetes (T2D) and increased risk of pancreatic cancer, led to international recognition and generated numerous publications in peer-reviewed scientific journals.

The group is funded by different national and international public and private institutions, as well as from diabetes patient associations and supported by the Andalusian Government.

Amarna Therapeutics

Amarna Therapeutics is a privately held Biotech company founded in 2008. Its head office is located in Leiden (The Netherlands), and it also holds a research facility in Seville (Spain). The company has developed a proprietary production and gene therapy delivery platform in its SuperVeroTM cell line and SVecTM vector for the development of safe and efficient therapies. The companys pipeline targets several major indications as well as orphan diseases within the field of degenerative, inflammatory and autoimmune diseases. The company plans to take the first candidate from its pipeline into clinical development in 2021.

In October 2019, Amarna secured 10 million in new equity, with the aim of bringing the first product into clinical studies. The financing round was led by the Swedish Flerie Invest AB, together with existing shareholders and an innovation credit from the Netherlands Enterprise Agency (RVO.nl).

SOURCE: Amarna Therapeutics

Link:
Dutch Amarna Therapeutics enters research collaboration with Spanish FPS, examining the efficacy of its SV40-based SVecTM gene delivery vector...

Diabetes is a chronic condition that affects how your body uses insulin. This hormone controls how much blood sugar, also known as glucose, is released into your cells to be used as energy.

Over 34 million people in the US have diabetes, according to the Centers for Disease Control and Prevention (CDC). While there is no cure for diabetes, it can be managed with lifestyle and dietary changes, or medication like insulin.

Here's what you need to know to manage diabetes and lower blood sugar levels.

With all types of diabetes, your body either doesn't produce enough insulin, or isn't able to use insulin effectively.

Insulin is necessary to move blood sugar into your cells, where it is stored and used for energy. Without insulin, a condition called hyperglycemia can occur, where blood sugar builds up in your bloodstream instead of traveling into your cells.

Type 1 diabetes makes up just 10% of all diagnosed diabetes cases in the US, according to the CDC. It is most commonly diagnosed in children, teenagers, and young adults.

Although the cause is unknown, type 1 diabetes may be due to an autoimmune response caused by an infection or other trigger. Your body mistakenly attacks and damages the beta cells in your pancreas that make insulin, so little or no insulin is produced.

There are not many risk factors for type 1 diabetes, though genetics is believed to play a role. The odds of the children of men with type 1 diabetes developing the condition is 1 in 17, according to the American Diabetes Association (ADA). For the children of women with type 1 diabetes, the odds are 1 in 25 if the woman is under the age of 25, or 1 in 100 after the age of 25.

A type 1 diabetes diagnosis requires some important lifestyle changes. You must take insulin every day in order to survive. Your blood sugar level needs to be frequently monitored. It's essential to carefully plan your meals and count carbohydrates.

"This can be a frustrating and tiresome adjustment, but it is crucial that patients educate themselves on how certain foods impact glucose levels," says endocrinologist Rocio Salas-Whalen, MD, of New York Endocrinology.

Type 2 diabetes makes up about 90% of all diagnosed diabetes cases in the US. It is most often diagnosed in adults, but the CDC notes that it is becoming increasingly diagnosed in children and teenagers.

With type 2, your body can produce insulin, but it is not able to use it effectively. This is called insulin resistance, which happens when your liver, muscle, and fat cells don't effectively take in the blood sugar from your blood to use it for energy. As a result, your blood sugar level increases, which can eventually lead to type 2 diabetes.

You are more at risk for type 2 diabetes if you:

In addition to eating a healthy diet, it's very important for people with type 2 diabetes to maintain a healthy weight, Salas-Whalen says, because this can also help them control blood sugar levels.

Pregnant people may develop gestational diabetes, which is caused by the body's inability to produce the extra insulin needed during your pregnancy. Gestational diabetes can put your baby at risk for health problems later in life, such as obesity or type 2 diabetes.

About 7% of pregnant people in the US are diagnosed with gestational diabetes. It usually begins in the middle of your pregnancy, without any symptoms. You should be tested for it between your 24th and 28th weeks of pregnancy. It typically goes away after your baby is born, but you will have a higher risk of developing type 2 diabetes later in life.

If you have gestational diabetes, you'll need to work with your doctor to develop a healthy eating plan, and you should also remain physically active to help keep your blood sugar levels low. If a healthy diet and exercise don't lower your blood sugar levels, you may need to take insulin.

Prediabetes is a condition where your blood sugar levels are elevated, but not yet high enough for a diabetes diagnosis. However, if left untreated, prediabetes can develop into type 2 diabetes.

More than a third of all US adults over 88 million have prediabetes, yet 84% of them don't know they have it, the CDC notes.

With lifestyle changes like a healthy diet, losing weight, and getting regular exercise, it's possible for prediabetes to be reversed or delayed. Your doctor may also prescribe medication to help lower your blood sugar level.

"A prediabetic still has the potential to avoid diabetes, which should be avoided in every possible way," Salas-Whalen says.

The signs of all types of diabetes can include the following:

However, these symptoms develop slowly over time, and it may be difficult to recognize them, especially if you have type 2 diabetes. The signs of type 1 diabetes may be more severe, and can also include nausea or vomiting.

Target blood sugar levels are different for those with diabetes. The follow chart depicts normal blood sugar levels for diabetics and non-diabetics:

Yuqing Liu/Insider

Many people with diabetes with need to learn how to check their blood sugar multiple times a day using a glucose meter or a continuous glucose meter.

"Try not to think of blood sugars as 'good' or 'bad' or as a reflection of how well or bad you are doing," says Shelley Nicholls, DNP, APRN, CDCES, director of patient education at the Diabetes Research Institute. "Having a good understanding of what affects blood sugars and which of them a person can control or influence is the best tool a person with diabetes can have."

To treat diabetes, it is important to lower your blood sugar level and make sure it stays in a healthy range.

Doing this will not only increase your energy, but according to the ADA, each percentage point of A1C lowered reduces the possibility of long-term health complications which could include serious heart, kidney, brain, eye, or foot problems by 40%.

These are some of the best natural ways to lower and manage your blood sugar levels over time:

It's important for people with diabetes to be careful about the foods they eat because they can impact your blood sugar levels."Some foods can worsen diabetes, while other foods can actually improve diabetes control," Salas-Whalen says.

Carbohydrates and fiber especially affect your blood sugar levels in the following ways:

It can be helpful to follow a diet to manage your diabetes, as planning out your meals and snacks will help you control blood sugar levels effectively.

"Every person has different needs, so there is no one diet that is recommended for people with diabetes," Nicholls says. "The best option is to meet with a dietitian to determine individual needs and goals."

Here are some of the best diets for diabetics:

The Mediterranean diet includes plant-based foods, lean meats, and healthy fats.

According to a 2009 study published in Diabetic Medicine, people who strictly followed a Mediterranean diet for three months had lower A1C percentages and lower blood sugar levels after meals than those who followed it less strictly.

The DASH diet, which stands for Dietary Approaches to Stop Hypertension, is mainly used to lower blood pressure, but it can also help lower blood sugar.

A 2017 study published in the ADA journal Diabetics Spectrum suggests that the DASH diet can lower insulin resistance and help you lose weight. A 2016 study published in the journal Nutrition found that a DASH diet can also help lower the risk for gestational diabetes by as much as 71%.

This high-fat, low-carb diet limits carbs to 20 to 50 grams daily in an effort to put your body in the metabolic state of ketosis, where you burn fat instead of carbs for fuel.

A 2017 study published in Nutrition & Diabetes found that overweight adults with type 2 diabetes or prediabetes who followed a keto diet had lower A1C levels and lost over 4% more weight after one year than those who followed a moderate-carbohydrate/low-calorie/low-fat diet.

There are also some health risks associated with the keto diet. If you have type 1 diabetes, your lowered blood sugar level may lead to hypoglycemia and serious brain, kidney, or liver complications.

Another issue associated with this diet are "keto flu" symptoms that may include headache, nausea, and vomiting. It's important to consult with your doctor or a registered dietitian before starting a keto diet.

People with type 1 diabetes need to take insulin every day in order to survive. If people with type 2 diabetes are unable to reach their blood sugar target levels with diet and exercise, they may also need medication like insulin or metformin.

People with type 1 diabetes generally need to take three to four doses of insulin every day, according to the ADA. Women with gestational diabetes may need to take insulin daily during their pregnancy if their bodies aren't producing enough of it naturally. Many people with type 2 diabetes may need one dose each day with or without other medications.

Insulin is injected in the fat under your skin using a syringe, insulin pen, or pump. It should be injected in the same area of the body, but not the same place each day. It's best to inject insulin at mealtime so it is more effectively processed in your body.

There are many different types of insulin, and your doctor may even prescribe two or more of the following types:

"The challenge with taking insulin is that it's tough to know precisely how much to take," Nicholls says. The amount is based on factors that may change throughout the day, such as food, exercise, and stress. "So, deciding on what dose of insulin to take is a complicated balancing act."

Taking an extra dose of insulin can also help you lower blood sugar fast if it's an emergency, though you may want to check in with your doctor beforehand.

If you have type 2 diabetes, your doctor may prescribe metformin, a medication that lowers blood sugar by slowing your liver's production of glucose. It is the drug most commonly prescribed to treat type 2 diabetes.

Metformin is available in a liquid, pill, or extended-release tablet. You take it orally at mealtime two to three times a day. The extended-release tablet only needs to be taken once daily.

According to a 2012 scientific review published in Diabetes Care, metformin can effectively reduce A1C levels for people with type 2 diabetes by an average of 1.12%.

Although it's possible to control your diabetes and lower blood sugar levels, there is no specific cure.

"Because of this reality, lifestyle changes must be permanent and not temporary in order to avoid the potential long-term complications of diabetes," Salas-Whalen says.

To develop the best plan of treatment for diabetes, it's important to meet with your doctor for individualized recommendations.

Link:
What is diabetes? A comprehensive guide to lower blood sugar and manage the condition - Insider - INSIDER

September 25, 2020 -A group of Senators has introduced a bill that would expand access to the Medicare Diabetes Prevention Program through telehealth.

S 4709 was introduced this week by Senators Tim Scott (R-SC), Mark warner (D-VA), Kevin Cramer (R-ND), Kyrsten Sinema (D-AZ), Tom Cotton (R-AR) and Tina Smith (D-MN). Titled the Prevent Diabetes Act, it addresses a long-standing issue with a Medicare program designed to help members at increased risk of developing type 2 diabetes.

Its no secret that diabetes is a disease that has disproportionately affected minority communities across the country, Warner said in a press release. To ensure that all individuals have the tools needed to combat this preventable disease, the Prevent Diabetes Act would help expand access to virtual classes under the existing Medicare Diabetes Prevention Program. This commonsense and cost-saving expansion will ensure that more Americans at-risk of developing diabetes who are living in either rural or medically underserved communities, can participate in this critical program that has been proven to delay the full onset of this preventable disease.

The original Diabetes Prevention Programwas developed by the National Institutes of Healths National Institute of Diabetes and Digestive and Kidney Disease (NIDDK), and focused on in-person classes and one-on-one coaching. Based on that model, which is administered by the Centers for Disease Control and Prevention, the Centers for Medicare & Medicaid Services created the National Diabetes Prevention Program for Medicare beneficiaries and launched that program in 2018.

But the Medicare Diabetes Prevention Program Expanded Model conducted by the Center for Medicare and Medicaid Innovation doesnt reimburse care providers for using connected health platforms. Telehealth and mHealth advocates have been lobbying for years to add those services, saying a virtual platform would reach far more people at risk and enable providers to make better use of limited resources.

More than 70 healthcare providers are now listed on the CDCs DPP website, though only a handful have been recognized as offering proof that their online programs reach recognized benchmarks for activity and weight loss.A growing number of programs are using virtual careas a means of expanding the programs reach and making the most of limited resources, and theyre asking CMS to cover those services.

Last year, a group of Senators including those sponsoring the Prevent Diabetes Act wrote a letter to Health and Human Services Secretary Alex Azar and CMS Administrator Seema Verma asking that the program be expanded to include CDC-recognized virtual DPP providers.

Virtual delivery of MDPP has the ability to empower beneficiaries to access MDPP regardless of where they live, and in the format of their choosing, the Senators wrote. Because of the outcome-focused reimbursement structure, CMS has insulated from reimbursing for ineffective treatment. Medicare Advantage plans have also been vocal in their desire to deploy virtual DPP for their beneficiaries. Given this, we also encourage CMS to consider ways for Medicare Advantage plans to use virtual providers to ensure that all Medicare beneficiaries have access to a CDC fully-recognized DPP.

In April, that same group lobbied again for the inclusion of virtual care providers, saying the coronavirus pandemic has created further barriers to in-person care.

The new bill, which as of September 25 contains no text or summary, is supported by several organizations, including the American Diabetes Association, American Medical Association, Connected Health Initiative and National Kidney Foundation, along with digital health companies Livongo, Noom and Omada Health.

More:
New Bill Would Add Telehealth to Medicare Diabetes Prevention Program - mHealthIntelligence.com

Frequent hot tub bathinghada positive impact on glycemia, blood pressure, and body weight in patients with type 2 diabetes, in the first real-world study to analyze the effect of this type of heat therapy in such individuals.

"The data from our analysis showed that the frequency of hot tub bathing could have beneficial influences on diabetic control, hypertension, and obesity even after adjusting for confounding factors," HisayukiKatsuyama, MD, told Medscape Medical News.

Katsuyama presented the findings as a poster at the virtual European Association for the Study of Diabetes (EASD) Annual Meeting 2020. The study aimed to explore the real-world influence of habitual hot tub bathing on the control of type 2 diabetes and other cardiovascular risk factors.

"Heat therapy, shown here with hot tub bathing, can be one effective therapeutic option for type 2 diabetes in daily life. An alternative form of heat exposure might be nutrition therapy and exercise," noted Katsuyama, from Kohnodai Hospital, Ichikawa, Chiba, Japan.

But Lucy Chambers, PhD, head of research communications at Diabetes UK, was not so enthusiastic about the results.

"While this research suggests there might a link between taking regular hot baths and better health in people with type 2 diabetes, it raises more far more questions than it answers," she said.

"It could be that people who bathe more frequently have a healthier lifestyle in general perhaps they are more physically active we just don't know from the limited data collected."

"It isnot possible to say from this research whether bathingcan benefit yourphysicalhealth," she noted in a statement from Diabetes UK.

Prior to the current study, there were no large studies looking at the effects of hot tub bathing on metabolic parameters in patients with diabetes.

One cohort study in Finland revealed that frequency of sauna bathing was inversely associated with fatal cardiovascular events in middle-aged adults (BMC Med. 2018;16:219). And a prior small before-and-after study in patients with diabetes showed a significant reduction in fasting glucose and A1c (N Engl J Med. 1999;341:924-925), Katsuyama noted.

Most homes in Japan, where bathing is a traditional and common practice, have hot tubs, which prompted the researchers' idea for a real-world study, he explained.

Katsuyama and colleagues studied the frequency of hot-tub bathing using a self-reported questionnaire completed by 1297 patients with type 2 diabetes who regularly visited Kohnodai Hospital over 6 months.

They took anthropometric measurements and used blood test results to analyze associations between hot tub use and different variables. Patients were divided into three groups according to frequency of bathing: group 1, 4 baths/week; group 2, 1-<4 baths/week; and group 3, < 1 bath/week.

Mean age was 67 years, weight was 67 kg, BMI was 25.9 kg/m2, and A1c was 7.2%. There were more men than women (713/584).

Most participants, 693, were in group 1 ( 4 baths/week), 415 were in group 2 (1-< 4 baths/week); and 189 were in group 3 (< 1 bath/week).

The mean frequency of bathing was 4.2 times/week and mean duration of bathing was 16 minutes.

Body weight, BMI, waist circumference, diastolic blood pressure, and A1c were all significantly better in group 1 (most frequent bathing) compared with group 3 (least frequent bathing) (Table).

Table. Effects of Frequency of Hot Tub Bathing on Metabolic Parameters

Group 1 4 baths/week

BP = blood pressure

Katsuyama pointed out that animal studies have suggested heat stimulation might improve insulin sensitivity and enhance energy expenditure, an effect also observed during exercise.

"I expect that patients can benefit in a similar way with heat therapy," he added, noting that hot tub bathing might be particularly beneficial for patients who cannot exercise.

"It would probably [also] be beneficial for the prevention of diabetes," and potentially, diabetes complications, he said. Indeed, "cohort studies have shown the possibilities that heat therapy could prevent cardiovascular diseases."

Katsuyama pointed out that a key strength of the study was the relatively large number of participants compared with previous studies.

But there were also limitations due to the nature of the cross-sectional study, which "means we cannot guarantee causality, and secondly, various confounding factors, such as diet and other life habits, could influence the results."

"A well-designed prospective study will be needed to confirm the beneficial effects of the heat therapy," he concluded.

EASD 2020. Presented September 22, 2020. Abstract 342.

Katsuyama has reported no relevant financial relationships.

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Hot Tubs Improve A1c, BMI, and Blood Pressure in Type 2 Diabetes - Medscape

Dublin, Sept. 25, 2020 (GLOBE NEWSWIRE) -- The "Worldwide Diabetes Reusable Insulin Delivery Pen Market: Demand, Insights, Trends, Analysis, Opportunities, Growth Potential and Forecast to 2026" report has been added to ResearchAndMarkets.com's offering.

The Worldwide Diabetes Reusable Insulin Delivery Pen Market size is expected to touch US$ 6 billion by 2026.

The report offers the most up-to-date industry data on the actual market situation and future outlook for the worldwide diabetes reusable insulin delivery pen market. The report provides historical market data for 2013 - 2019, and forecasts from 2020 until 2026.

The report contains a granular analysis of the present industry situations, market demands, reveal facts on the market size, reusable insulin pen volume, revenues for reusable insulin delivery pen, and illustrative forecast to 2026. It also provides 16 countries with an all-round analysis of an overall number of patients with diabetes and insulin users. A comprehensive analysis has been done on the market share of the countries-based market.

The report explores essential insights into worldwide diabetes reusable insulin delivery pen market for the top 16 countries, comprising the United States, the United Kingdom, Canada France, Italy, Spain, Germany, Netherlands, Poland, Sweden, Turkey, Australia, Japan, China, India, and Brazil until 2026. The report also provides a detailed description of growth drivers and inhibitors of the worldwide diabetes reusable insulin delivery pen market.

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The Major Companies Dominating this Market for its Products, Services and Continuous Product Developments are:

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7. Diabetes Reusable (Cartridge) Insulin Delivery Pen Market & Forecast (2013 - 2026) - Major 16 Countries Data Analysis7.1 United States7.1.1 Overall Diabetes Population & Forecast (Volume) 7.1.2 Insulin Users & Forecast (Volume) 7.1.3 Diabetes Reusable (Cartridge) Insulin Delivery Pen Users (Volume) 7.1.4 Diabetes Reusable (Cartridge) Insulin Delivery Pen Market & Forecast (Value) 7.2 Canada7.3 Germany7.4 France7.5 Italy7.6 Spain7.7 United Kingdom7.8 Netherlands7.9 Poland7.10 Sweden7.11 Turkey7.12 Australia7.13 Japan7.14 China7.15 India7.16 Brazil

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Worldwide Diabetes Reusable Insulin Delivery Pen Market Report 2020: Demand, Insights, Trends, Analysis, Opportunities, Growth Potential and Forecast,...

A leading manufacturer of automated insulin delivery systems, Dexcom (NASDAQ:DXCM) signed a five-year collaboration agreement with the University of Virginia on Thursday . The company will fund research at the university that could expand its addressable patient population.

Dexcom already has clinical trial evidence that shows its constant glucose monitoring (CGM) technology reduces the amount of time Type 1 diabetes patients spend with blood sugar levels that are too high or too low. Through its collaboration with the university, the company will test its CGM technology for use among people with Type 2 and gestational diabetes, as well as for hospitalized patients.

Image source: Getty Images.

The University of Virginia's Center for Diabetes Technology will lead the research efforts, but the collaboration will employ experts from multiple disciplines across the University of Virginia System.

This isn't the first time these two have conducted research as partners. In 2019, the collaboration partners presented successful results of a trial with Type 1 diabetes patients that used Dexcom's CGM technology to control their blood sugar levels.

In 2020, Dexcom expects revenue to grow by about 25% to around $1.85 billion. While there has been some uptake of Dexcom's CGM systems among insulin-dependent Type 2 patients, the relative lack of evidence of a benefit is severely limiting the company's total revenue.

In 2017, the American Diabetes Association estimated the number of Type 1 diabetes cases in the U.S. at 1.3 million. In 2012, it estimated the number of Type 2 diabetes patients at 27.8 million.

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Dexcom and University of Virginia to Advance Diabetes Research Together - Motley Fool

My name is Dr. Vishwanath Pattan and I am the Medical Director of Endocrinology at Wyoming Medical Center in Casper. Endocrinology is the study of hormones, and as an endocrinologist I treat patients for a wide variety of diseases related to hormonal deficiencies and imbalances. That includes many patients with diabetes.

At my clinic, Wyoming Endocrine and Diabetes, I treat patients from across Wyoming, and I have noticed an alarming trend for my diabetic patients in relation to the COVID-19 pandemic: an inability to monitor and control glucose or maintain weight in the summer months.

In a typical year, diabetic patients tend to lose weight and achieve better glucose control in the summer because they are able to live a much more active outdoor lifestyle. During the winter, I often see the opposite trend of added weight, less stable glucose levels, seasonal depression, and an increase in overall stress.

2020, however, has not been a typical year, and I have noticed a deviation from the typical summer pattern in my diabetic patients. Many of these patients have actually gained weight, exhibited less than stable glucose control, and had an increase in their overall stress levels.

I have a few theories on why this might be. When COVID-19 was first acknowledged as a public safety concern, people were quick to stock up on everything they could. As we know, the shelf life of heavily processed foods is what makes them some of the first to go amidst a global crisis.

These foods are built to stand the test of time, but for a diabetic patient, they can easily contribute to an unsafe fluctuation of glucose. There was also a lot of uncertainty, fear, and confusion that caused millions to be left without a job and the added stress of strict isolation measures. People were forced to live a much more sedentary lifestyle, whether they wanted to or not, and eat food that does not promote a healthy glucose level. I also saw a major decrease in correspondence with many of my patients with uncontrolled diabetes, further contributing to an atypical summer for the diabetic population.

So, why does this raise a red flag?

Although patients with diabetes are not at any further risk of contracting COVID-19, they are much more likely to suffer greater complications because of it. These complications could lead to the need for ventilator support, further intensive care, and even higher death rates by several folds. This leads me to my main concern with so many of my diabetic patients experiencing poor glucose control prior to a season in which it is already difficult to manage: A person with uncontrolled diabetes in the summer is more likely to have uncontrolled diabetes in the winter, especially during a global pandemic. With the dual-threat of COVID-19 and this upcoming flu season, it is paramount that people with diabetes put their health and safety at the forefront.

I strongly urge people with diabetes and their families to safely support one another through the winter months with the helpful information discussed below.

People with diabetes should:

Monitor glucose regularly, per your healthcare providers recommendation

Make sure to follow up with your healthcare providers, either in person or by utilizing virtual visits. (Healthcare facilities take utmost care and precautions, and put your health as a top priority, so in-person visits should be safe). In the coming months, it is essential to keep your providers up-to-date on your progress, and you should discuss individualized glucose goals with your doctor

Contact your healthcare provider immediately if your blood glucose is above target

Remain compliant with medication regimens and dietary treatment plans

Aim to eat a balanced diet, exercise regularly and get adequate sleep at least between 7 and 8 hours per night

Maintain a healthy immune system by prioritizing glucose control, managing stress levels and taking a Vitamin D supplement. In Wyoming, most people are naturally deficient during the winter months and are encouraged to seek their healthcare providers recommendation for proper supplementation.

Family members of diabetic patients should:

Encourage your loved one to keep appointments with healthcare providers

Assist them with technology for virtual visits

Avoid social gatherings, practice proper hand hygiene, and always wear a mask in public spaces to keep your loved one safe

Help with cooking balanced and healthy meals

Ensure that your loved one has at least 4 to 6 weeks worth of diabetic supplies on hand in case of supply issues later on. These include testing strips, insulin, and necessary insulin administration equipment

Remind patients to take their medication on time and encourage compliance with glucose monitoring

Help to maintain a stress-free environment at home

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Concerning trend found in patients with diabetes - Green River Star