Cell and Gene Therapy Manufacturing Quality Control Market Growing Trends and Technology Forecast to 2029 |... SeeDance News
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Cell and Gene Therapy Manufacturing Quality Control Market Growing Trends and Technology Forecast to 2029 |... - SeeDance News
Genetic therapies aim to treat or cure conditions by correcting problems in your DNA. Your DNA, including specific genes, contains instructions for making proteins that are essential for good health. Mutations, or changes in your DNA, can lead to proteins that do not work properly or that are missing altogether. These changes can cause genetic, or inherited, disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease.
Genetic therapies are approaches that treat genetic disorders by providing new DNA to certain cells or correcting the DNA. Gene transfer approaches, also called gene addition, restore the missing function of a faulty or missing gene by adding a new gene to affected cells. The new gene may be a normal version of the faulty gene or a different gene that bypasses the problem and improves the way the cell works.
Genome editing is a newer approach that allows precise correction or other targeted changes to the DNA in cells to restore a cells function. Genome editing can:
Gene transfer or genome editing treatments can directly modify the cells in your body, or your cells can be collected and treated outside of your body and then returned to you. For example, a doctor can remove immune system cells, cells that are part of your bodys natural defense system, or bone marrow cells from your body, modify their DNA, and then re-introduce them to your body.
The only genetic therapies that are currently approved by the U.S. Food and Drug Administration (FDA) are for a rare inherited eye condition, as well as certain types of cancer. Genetic therapies that are in development could treat or cure other inherited disorders; treat other cancers; or treat infections, including HIV.
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Genetic Therapies - What Are Genetic Therapies? | NHLBI, NIH
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The genes in your bodys cells play a key role in your health. Indeed, a defective gene or genes can make you sick.
Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition.
This research is paying off, as advancements in science and technology today are changing the way we define disease, develop drugs, and prescribe treatments.
The U.S. Food and Drug Administration has approved multiple gene therapy products for cancer and rare disease indications.
Genes and cells are intimately related. Within the cells of our bodies, there are thousands of genes that provide the information to produce specific proteins that help make up the cells. Cells are the basic building blocks of all living things; the human body is composed of trillions of them.
The genes provide the information that makes different cells do different things. Groups of many cells make up the tissues and organs of the body, including muscles, bones, and blood. The tissues and organs in turn support all our bodys functions.
Sometimes the whole or part of a gene is defective or missing from birth. This is typically referred to as a genetically inherited mutation.
In addition, healthy genes can change (mutate) over the course of our lives. These acquired mutations can be caused by environmental exposures. The good news is that most of these genetic changes (mutations) do not cause disease. But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.
Depending on what is wrong, scientists can do one of several things in gene therapy:
To insert new genes directly into cells, scientists use a vehicle called a vector. Vectors are genetically engineered to deliver the necessary genes for treating the disease.
Vectors need to be able to efficiently deliver genetic material into cells, and there are different kinds of vectors. Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.
Gene therapy can be used to modify cells inside or outside the body.When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient.
When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. The vector containing the desired gene is introduced into these cells. The cells are later injected into the patient, where the new gene is used to produce the desired effect.
Before a gene therapy can be marketed for use in humans, the product must be tested in clinical studies for safety and effectiveness so FDA scientists can consider whether the risks of the therapy are acceptable considering the potential benefits.
The scientific field for gene therapy products is fast-paced and rapidly evolving ushering in a new approach to the treatment of vision loss, cancer, and other serious and rare diseases. As scientists continue to make great strides in this therapy, the FDA is committed to helping speed up development by interacting with those developing products and through prompt review of groundbreaking treatments that have the potential to save lives.
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How Gene Therapy Can Cure or Treat Diseases | FDA
For Immediate Release: December 18, 2017
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The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.
Todays approval marks another first in the field of gene therapy both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses, said FDA Commissioner Scott Gottlieb, M.D. Were at a turning point when it comes to this novel form of therapy and at the FDA, were focused on establishing the right policy framework to capitalize on this scientific opening. Next year, well begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters including new clinical measures for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.
Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the U.S. Biallelic mutation carriers have a mutation (not necessarily the same mutation) in both copies of a particular gene (a paternal and a maternal mutation). The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.
Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patients vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.
The approval of Luxturna further opens the door to the potential of gene therapies, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.
Luxturna should be given only to patients who have viable retinal cells as determined by the treating physician(s). Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.
The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subjects ability to navigate an obstacle course at various light levels. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.
The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.
The FDA granted this application Priority Review and Breakthrough Therapy designations. Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
The sponsor is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.
To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.
The FDA granted approval of Luxturna to Spark Therapeutics Inc. The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene
Andrea Fischer301-796-0393
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FDA approves novel gene therapy to treat patients with a rare form of ...
Ever since the dawn of mankind, diseases have plagued humans over the ages. Years of innovations and advancements in science has provided us with a deeper understanding of how diseases work. This has led to lower mortality rates and longer lifespans. But there are some diseases that just cannot be cured using traditional medicine or surgery. Gene therapy is an experimental technique that caters to patients with such diseases.
Gene therapy is a technique which involves the replacement of defective genes with healthy ones in order to treat genetic disorders. It is an artificial method that introduces DNA into the cells of the human body. The first gene therapy was successfully accomplished in the year 1989.
The simple process of gene therapy is shown in the figure below:
In the figure, the cell with the defective gene is injected with a normal gene which helps in the normal functioning of the cell. This technique is employed mainly to fight against the diseases in the human body and also to treat genetic disorders. The damaged proteins are replaced in the cell by the insertion of DNA into that cell. Generally, improper protein production in the cell leads to diseases. These diseases are treated using a gene therapy technique. For example, cancer cells contain faulty cells which are different from the normal cells and have defective proteins. Hence, if these proteins are not replaced, this disease would prove to be fatal.
Basically, there are two types of gene therapy
This type usually occurs in the somatic cells of human body. This is related to a single person and the only person who has the damaged cells will be replaced with healthy cells. In this method, therapeutic genes are transferred into the somatic cells or the stem cells of the human body. This technique is considered as the best and safest method of gene therapy.
It occurs in the germline cells of the human body. Generally, this method is adopted to treat the genetic, disease causing-variations of genes which are passed from the parents to their children. The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms. Germline gene therapy is not legal in many places as the risks outweigh the rewards.
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Gene Therapy - Discover How It Works Its Types And Applications - BYJUS
IVERIC bio Subsidiary Sells Assets of Gene Therapy Product Candidates for Treatment of Retinal Diseases Marketscreener.com
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IVERIC bio Subsidiary Sells Assets of Gene Therapy Product Candidates for Treatment of Retinal Diseases - Marketscreener.com
News Release
Thursday, September 8, 2022
Gene augmentation rescues cilia defects in light-sensing cells derived from patients with blinding disease.
Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood. Using patient-derived retina organoids (also known as retinas-in-a-dish), the researchers discovered that a type of LCA caused by mutations in the NPHP5 (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. The findings not only shed light on the function of NPHP5 protein in the primary cilium, but also led to a potential treatment for this blinding condition. NEI is part of the National Institutes of Health.
Its so sad to see little kids going blind from early onset LCA. NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration, said the studys lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory. Weve designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease.
LCA is a rare genetic disease that leads to degeneration of the light-sensing retina at the back of the eye. Defects in at least 25 different genes can cause LCA. While there is an available gene therapy treatment for one form of LCA, all other forms of the disease have no treatment. The type of LCA caused by mutations in NPHP5 is relatively rare. It causes blindness in all cases, and in many cases it can also lead to failure of the kidneys, a condition called Senior-Lken Syndrome.
Three post-doctoral fellows, Kamil Kruczek, Ph.D., Zepeng Qu, Ph.D., and Emily Welby, Ph.D., together with other members in the research team collected stem cell samples from two patients with NPHP5 deficiency at the NIH Clinical Center. These stem cell samples were used to generate retinal organoids, cultured tissue clusters that possess many of the structural and functional features of actual, native retina. Patient-derived retinal organoids are particularly valuable because they closely mimic the genotype and retinal disease presentation in actual patients and provide a human-like tissue environment for testing therapeutic interventions, including gene therapies. As in the patients, these retinal organoids showed defects in the photoreceptors, including loss of the portion of the photoreceptor called outer segments.
In a healthy retina, photoreceptor outer segments contain light-sensing molecules called opsins. When the outer segment is exposed to light, the photoreceptor initiates a nerve signal that travels to the brain and mediates vision. The photoreceptor outer segment is a special type of primary cilium, an ancient structure found in nearly all animal cells.
In a healthy eye, NPHP5 protein is believed to sit at a gate-like structure at the base of the primary cilium that helps filter proteins that enter the cilium. Previous studies in mice have shown that NPHP5 is involved in the cilium, but researchers dont yet know the exact role of NPHP5 in the photoreceptor cilium, nor is it clear exactly how mutations affect the proteins function.
In the present study, researchers found reduced levels of NPHP5 protein within the patient-derived retinal organoid cells, as well as reduced levels of another protein called CEP-290, which interacts with NPHP5 and forms the primary cilium gate. (Mutations in CEP-290 constitute the most common cause of LCA.) In addition, photoreceptor outer segments in the retinal organoids were completely missing and the opsin protein that should have been localized to the outer segments was instead found elsewhere in the photoreceptor cell body.
When the researchers introduced an adeno-associated viral (AAV) vector containing a functional version of NPHP5 as a gene therapy vehicle, the retinal organoids showed a significant restoration of opsin protein concentrated in the proper location in outer segments. The findings also suggest that functional NPHP5 may have stabilized the primary cilium gate.
The study was funded by the NEI Intramural program. Patient samples were collected at the NIH Clinical Center.
NEI leads the federal governments efforts to eliminate vision loss and improve quality of life through vision researchdriving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit https://www.nei.nih.gov.
About the NIH Clinical Center:The NIH Clinical Center is the worlds largest hospital entirely devoted to clinical research. It is a national resource that makes it possible to rapidly translate scientific observations and laboratory discoveries into new approaches for diagnosing, treating, and preventing disease. Over 1,600 clinical research studies are conducted at the NIH Clinical Center, including those focused on cancer, infectious diseases, blood disorders, heart disease, lung disease, alcoholism and drug abuse. For more information about the Clinical Center, visit:https://www.cc.nih.gov.
About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.
NIHTurning Discovery Into Health
Kruczek K, Qu Z, Welby E, et al. In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells. Stem Cell Reports. Sept 8, 2022.
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NIH researchers develop gene therapy for rare ciliopathy - National Institutes of Health (.gov)
Repeated muscle injections with Engensis (VM202), Helixmiths investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial.
While the sample size was too small to determine the therapys efficacy, muscle biopsies were collected and will be examined to further evaluate the underlying mechanisms of Engensis.
These data suggest that high dose, repeated treatments of Engensis, were safe and well tolerated, providing a great deal of flexibility in designing dosing schemes for future clinical studies, Helixmith stated in a company press release.
Trial analysis will continue once the full dataset is available, and the company plans to present such findings at a future conference. The next steps for Engensis development will be determined at that time.
Engensis is a non-viral gene therapy that uses Helixmiths proprietary small circular DNA molecule to deliver the hepatocyte growth factor (HGF) gene to cells in the muscle environment.
HGF provides instructions to produce a protein of the same name that helps the body form new blood vessels, prevents muscle loss, and promotes the growth and survival of nerve cells. The therapyis delivered via intramuscular (into-the-muscle) injections.
Helixsmith believes that by increasing HGF production, Engensis has the potential to promote nerve cell and muscle regeneration, thereby countering the progressive loss of motor control that characterizes ALS.
The therapy has been granted orphan drug and fast track designations by the U.S. Food and Drug Administration, both of which are intended to speed its clinical development and regulatory review.
A previous open-label Phase 1/2 trial (NCT02039401) found that four once-weekly intramuscular injections of Engensis (to a total dose of 64 mg) were safe and well-tolerated among 18 ALS patients. Signs that the therapy could slow disease progression were also observed.
These promising findings prompted the launch of a placebo-controlled Phase 2a trial, called REViVALS-1A (NCT04632225), which began patient enrollment last year. A total of 18 ALS patients experiencing motor symptoms in their limbs for four years or less were recruited at four sites in the U.S. and one in Korea.
Participants were randomized in a 2:1 ratio to receive three cycles of either Engensis or a placebo: at studys start, at two months, and at four months. Each cycle consisted of two days of injections to upper and lower limb target muscles, spaced two weeks apart (64 mg total of Engensis or a placebo).
This meant that Engensis-treated patients received a total of 192 mg of medication over the four-month period. All participants were monitored for six months from the studys start.
The trials main goal was to assess the safety and tolerability of Engensis, while efficacy measures were included as exploratory outcomes. These included changes in disability, muscle and lung function, survival, ALS-specific health-related quality of life, and the levels of muscle shrinkage biomarkers.
Top-line data showed that the investigational treatment was generally safe and well-tolerated, with no difference in the frequency of adverse events observed between the Engensis and placebo groups (83% for each).
One case of bronchitis a condition characterized by inflammation in the main airways of the lungs due to infection was observed in the Engensis group but was determined unrelated to treatment.
Injection site reactions were reported by 50% of Engensis-treated patients and 66.7% of those in the placebo group. Most of these reactions were mild or moderate in severity and temporary; no participant discontinued treatment due to the number of injections.
According to Helixmith, efficacy was unable to be evaluated due to the fact that four participants dropped out early from the small study.
Still, muscle tissue biopsies were obtained from injection sites to undergo analyses of muscle atrophy (shrinkage) biomarkers and others.
Since data on Engensis underlying mechanisms have been largely based on animal models, these results are expected to provide valuable information on the understanding of the mechanisms of actions of Engensis, and its effects on the [activity] of human genes, which will greatly help in the development of innovative medicines, the company stated in the release.
Helixmith greatly appreciates the generous and eager participation of the ALS patients, the company added.
Engensis is also being investigated across a range of conditions associated with deficits in circulation, and nerve and/or muscle damage, such as diabetic neuropathy, coronary artery disease, and Charcot-Marie-Tooth disease.
More than 500 patients have been treated with Engensis to date across 10 clinical trials and six different diseases, according to Helixmith. Data from these studies have also supported the therapys favorable safety profile and its ability to increase HGF production.
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Engensis Gene Therapy for ALS Found Safe in Small Phase 2a Trial |... - ALS News Today
DUBLIN--(BUSINESS WIRE)--The "Cancer Gene Therapy Market By Therapy, By End User: Global Opportunity Analysis and Industry Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.
Cancer Gene Therapy Market was valued at $1,389.42 million in 2020 and is estimated to reach $11,359.35 million by 2030, registering a CAGR of 23.3% from 2021 to 2030.
Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer. Owing to the high success rate during the preclinical and clinical trials, cancer gene therapy has gained popularity.
Many techniques are used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of the gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.
The rise in the prevalence of cancer, the benefits of cancer gene therapy over conventional cancer therapies, and the advancement in this field are the major factors that drive the market growth.
In addition, the surge in government support, ethical acceptance of gene therapy for cancer treatment, and rise in biotechnological funding encouraging the R&D activities for cancer gene therapy and thus fuel the growth of the cancer gene therapy market.
In addition rise in awareness regarding cancer gene therapy is a major factor that drives the global cancer gene therapy market growth.
In addition, an increase in government support for research in gene therapy, ethical acceptance of gene therapy for cancer treatment, and a rise in the prevalence of cancer boost the growth of the cancer gene therapy market. However, the high cost associated with the treatment and unwanted immune responses is expected to restrain the market growth.
Key Benefits For Stakeholders
Key Market Segments
By Therapy
By End User
By Region
Key Market Players
Key findings of the Study
Key Topics Covered:
CHAPTER 1: INTRODUCTION
CHAPTER 2: EXECUTIVE SUMMARY
CHAPTER 3: MARKET OVERVIEW
CHAPTER 4: CANCER GENE THERAPY MARKET, BY THERAPY
CHAPTER 5: CANCER GENE THERAPY MARKET, BY END USER
CHAPTER 6: CANCER GENE THERAPY MARKET, BY REGION
CHAPTER 7: COMPANY LANDSCAPE
CHAPTER 8: COMPANY PROFILES
For more information about this report visit https://www.researchandmarkets.com/r/ipoqor
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Global Cancer Gene Therapy Market Report 2022: Benefits of Gene Therapy Over Conventional Therapies Driving Adoption - ResearchAndMarkets.com -...
Hopes run high in Philadelphia that the region the scientific home of two of the first cell and gene therapies approved by the FDA will remain a major player as the cutting-edge treatments assume a bigger role in medicine.
To make that happen, Philadelphias life sciences industry will need not just scientists, management, and money, but also skilled workers to help laboratories run smoothly at an ever-growing number of biotech companies in the region and eventually to manufacture cures and treatments for rare diseases and elusive types of cancer.
To help build that skilled workforce, the Wistar Institute, the University City Districts West Philadelphia Skills Initiative, and partners have launched a new biomedical technician training program.
It will enroll 18 students in a 12-week paid training program at Wistar, potentially followed by an additional 10 weeks of hands-on work at Iovance Biotherapeutics Inc. in the Navy Yard and then a $23-an-hour manufacturing job. Iovance, which now employs 150 people in Philadelphia, is developing cancer treatments using cell therapy.
Iovance did not say how many of the trainees it would hire. Iovance officials will interview them after they complete the Wistar part of the training.
We expect to have a number of opportunities to which program participants can apply, Tracy Winton, Iovances senior vice president for human resources, said in a statement.
Cell and gene therapies are still in the early stages of development, but Philadelphia scientists have long played a central part. Luxturna, a gene therapy cure for a rare form of congenital blindness, and Kymriah, a cell therapy treatment for some forms of leukemia, are based on the work of Philadelphia scientists. Both received FDA approval in 2017.
Cell therapy uses modified cells to carry treatment into the body. Gene therapy involves the replacement of defective genes that cause what are typically rare diseases.
The new training effort, scheduled to start Sept. 22, builds on one started in 2000 at Wistar, a nonprofit biomedical research institute in University City, in partnership with Community College of Philadelphia. The original Wistar program, which provided general preparation for work in biotech and until this year was spread over two summers for each cohort, has graduated 196 students.
Recruitment for the new program, which Wistar designed to specifically prepare individuals for jobs at Iovance, started Aug. 23 and runs through Friday. As of last Friday morning, 263 people had applied, according to the West Philadelphia Skills Initiative (WPSI), which for a decade has been training Philadelphians for specific jobs at individual employers, such as Childrens Hospital of Philadelphia and SEPTA.
WPSI is handling recruitment selection for the Iovance training. The selection process for the 18 open spots includes an assessment of mathematical ability and an interview, said Cait Garozzo, managing director of WPSI.
Some folks, obviously, are very desperate for a job, any job, and were not trying to connect people that just want any job to this opportunity. Were trying to connect people that want a career in this industry to this opportunity, Garozzo said.
This is the first time WPSI and Wistar have worked together. Other supporters are the Chamber of Commerce of Philadelphia and the Philadelphia Industrial Development Corp.
If this is successful, we really think this could be a game changer for this region, said Kristy Shuda McGuire, dean for biomedical studies at Wistar. We think this is something we could repeat. We could have more cohorts each year if there are single employers who are interested in this and have a lab-based position and would be interested in taking a whole cohort.
The total budget for the training program was not disclosed.
Wistars original training program which expanded this year to include Montgomery County Community College and will be open to students at Bucks County Community College and Camden County College next year typically sends graduates into biotech jobs or on to further education, McGuire said.
Among the graduates of the Wistar program that have gone on to build careers in life sciences is Lois Tovinsky, 36, who completed the program in 2013 and is now laboratory operations manager for Chimeron Bio, a biotech start-up in the Curtis Building that is working on RNA therapeutics against cancer.
Tovinsky graduated from college with a degree in political science in 2008, when the economy collapsed and jobs were hard to find. She heard about the Wistar program in a science class at Community College of Philadelphia and saw it as a chance to fulfill her interest in science and leap from her job as a dog walker into a science career.
I came to the program with no practical skills in the lab, and my knowledge of science was really just the few courses I had taken and my own interest and enthusiasm that I had for it, said Tovinsky, who now mentors students in the Wistar program.
Tylier Driscoll, 21, a biology major at Community College of Philadelphia, was one of 15 students in the Wistar training cohort that finished early last month.
I definitely wanted to do something over the summer that wasnt working at Aldi, Driscoll said. Before this, I hadnt had any lab experience and I really wanted to get a feel for what it was like to work in a lab. I was working at a supermarket at the time. This is the perfect opportunity for me to get into my field.
As part of his training, he spent five weeks working at BioAnalysis LLC, a contract research organization in Kensington that performs quality analysis on the viruses used in gene therapy.
Now, Driscoll has a part-time job at BioAnalysis that he starts Tuesday, the same day he goes back to CCP for the fall semester. He plans to finish his associate degree in the spring and then attend either Drexel University or Temple University for his bachelors degree.
Lake Paul, the president and founder of BioAnalysis, which he called a minority-owned biotech, said the Wistar program is an awesome opportunity and one that reminds him of his own experience. Paul said he grew up in the hood in Miami and wouldnt have obtained his doctorate at Purdue University without the Upward Bound programs that helped him pursue education.
It is a wonderful, exciting, and unique opportunity for these students, both underrepresented folks and regular folks. And to give them actual training like this is unparalleled, said Paul.
The Philadelphia Inquirer is one of more than 20 news organizations producing Broke in Philly, a collaborative reporting project on solutions to poverty and the citys push toward economic justice. See all of our reporting at brokeinphilly.org.
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As Philly becomes a hub for life sciences, a new program will train workers for jobs in the field - The Philadelphia Inquirer
ALDERLEY PARK, England--(BUSINESS WIRE)--Charles River Laboratories International, Inc. (NYSE: CRL) and Cure AP-4, a non-profit foundation dedicated to raising funds and awareness about Adapter-Protein 4 Hereditary Spastic Paraplegia (AP-4 HSP), today announced a manufacturing collaboration. Charles River, a contract research and development manufacturing organization (CRO/CDMO), will provide High Quality (HQ) plasmid DNA for Cure AP-4s Phase I/II gene therapy trials against AP-4 HSP.
Founded in 2016 by the families of two newly diagnosed AP-4 HSP (SPG47) patients, Molly Duffy and Robbie Edwards, Cure AP-4s gene therapy treatment will look to address the root cause of AP-4 HSP, a rare neurodegenerative disorder, and is intended as a one-time, curative treatment for the patient.
What is AP-4 HSP? AP-4 HSP, also known as AP-4 Deficiency Syndrome, includes four sub-types of HSP: SPG47, SPG50, SPG51 and SPG52. Each of these HSP sub-types is associated with a defective autosomal recessive gene which causes a failure in the AP-4 Adaptor Complex. The phenotype and prognosis for each sub-type is extremely similar. Patients afflicted with any of the AP-4 HSP genetic disorders generally present with symptoms including global developmental delay, microcephaly, seizures, brain malformation, and hypotonia (low-muscle tone). The few patients who learn to walk independently tend to lose that ability a few months or few years later as they develop hypertonia (high-muscle tone) and muscle spasticity. Of the 249 currently confirmed global AP-4 HSP cases, most patients experience mobility in some or all extremities as the disorder progresses and are severely intellectually challenged.
Plasmid DNA Manufacturing ServicesThe collaboration will leverage Charles Rivers market leading expertise in plasmid DNA production, specifically HQ plasmid, which combines key features of GMP manufacture with rapid turnaround times to accelerate the timeline to clinic. DNA plasmids are a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply. In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field.
Charles River, with the acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021, has extended its comprehensive cell and gene therapy portfolio to include CDMO capabilities spanning viral vector, plasmid DNA and cellular therapy production for clinical through to commercial supply.
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About Cure AP-4Cure AP-4, originally known as Cure SPG47, was founded in 2016 by the families of two newly diagnosed SPG47 patients, Molly Duffy and Robbie Edwards. At the time there were only nine other documented cases worldwide, and due to the extreme rarity of the disorder there are no known treatments or cures.
About Charles RiverCharles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit http://www.criver.com.
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Charles River and Cure AP-4 Announce Gene Therapy Manufacturing Collaboration - Business Wire
WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc., (Myrtelle or the Company), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the European Medicines Agency (EMA) has classified the Company's lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of Canavan disease, as an Advanced Therapy Medicinal Product (ATMP), specifically a Gene Therapy Medicinal Product (GTMP). ATMP classification, which is determined by the Committee for Advanced Therapies (CAT), was established to regulate cell and gene therapy and tissue engineered medicinal products, support development of these products, and provide a benchmark for the level of quality compliance for pharmaceutical practices. As a designated GTMP product, rAAV-Olig001-ASPA will follow the Centralized Procedure through the EMA and benefit from a single evaluation and authorization process. Additional benefits established through the ATMP regulation include pathways for Scientific Advice and significant fee reductions for such advice.
rAAV-Olig001 is a novel vector from a class of recombinant AAVs (rAAVs) that selectively target oligodendrocytes the cells in the brain responsible for producing myelin, the insulating material that enables proper function of neurons and makes up the brains white matter. The Companys lead program is in Phase 1/2 clinical development for Canavan disease (CD) a fatal childhood genetic disorder characterized by the degeneration of the white matter in the brain. The production of myelin is affected in CD due to a mutation in the Aspartoacylase gene (ASPA) leading to deficiency in Aspartoacylase enzyme (ASPA). The oligodendrocyte-targeted gene therapy using the rAAV-Olig001 vector is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartic Acid (NAA), a neurochemical abundant in the brain, and supporting myelination. Myrtelle entered into an exclusive worldwide licensing agreement with Pfizer Inc. in 2021 to develop and commercialize this novel gene therapy for the treatment of CD.
In addition to ATMP classification, rAAV-Olig001-ASPA has been granted US Orphan Drug, Rare Pediatric Disease, and Fast Track designations by the FDA which support the Companys mission to provide treatments for patients with CD. "The designation by the EMA of rAAV-Olig001-ASPA as a Gene Therapy Medicinal Product as a potential treatment for patients with Canavan disease provides important benefits in the development of this innovative therapy. The ATMP classification will facilitate discussions with the EMA as part of our strategy to seek product registration in the EU," said Nancy Barone Kribbs, PhD, Senior Vice President of Global Regulatory Affairs at Myrtelle.
ABOUT MYRTELLE
Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead program in Canavan disease. For more information, please visit the Companys website at: http://www.myrtellegtx.com.
ABOUT CANAVAN DISEASE
Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. CD patients are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD and only palliative treatments are available. More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.
Forward-Looking Statements
This press release contains forward-looking statements. Words such as may, believe, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding rAAV-Olig001-ASPA as a potential treatment for patients with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.
IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., receives coveted Best in Category award for an interim 12-week analysis from the ongoing Phase 2a trial of an investigational novel gene therapy product, URO-902 (plasmid human cDNA encoding maxi-K channel). The award-winning abstract was presented at the 2022 International Continence Society annual meeting on September 8, 2022. The 2022 ICS Annual meeting is being held September 7-10, 2022, in a hybrid format with both online and in person participation (Vienna, Austria).
According to ICS, this honor is awarded to the highest-scoring abstract in each category. Scores are awarded by the ICS scientific committee members, external reviewers, and scientific session chairs. Abstracts are judged based on criteria of scientific merit, originality/topicality, and clinical relevance. Review the full 2022 Abstract Awards List here.
The podium presentation at ICS 2022 took place on Thursday, September 8, at 10:20 Central European Time (CET). Presentation #6 in Scientific Podium Session S1, Best Urology, was titled, Efficacy and Safety of a Novel Gene Therapy (URO-902; PVAX/HSLO) in Female Patients with Overactive Bladder Syndrome and Urge Urinary Incontinence: Results from a Phase 2A Trial. The presentation described a prespecified, 12-week interim analysis of a 48-week multicenter, randomized, double-blind, placebo-controlled, dose-escalation study (NCT04211831). URO-902 was administered using direct intradetrusor injections via cystoscopy under local anesthesia. The presenting author was Kenneth Peters, M.D., Principal Investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Womens Urology and Pelvic Health Center; and Professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Mich.
We are delighted that this presentation has received the Best in Category Prize: Overactive Bladder, reflecting the high-quality scientific research involved, said Dr. Peters. The promising interim safety and efficacy findings from this prespecified analysis indicate that URO-902 has potential as a therapeutic option for overactive bladder patients who have failed oral pharmacologic therapy.
At week 12, both URO-902 24 mg and 48 mg were associated with clinically relevant improvement in mean daily micturition (urination), urgency episodes, UUI episodes, OAB questionnaire symptom bother score, and proportion of patient global impression of change responders. Treatment-emergent adverse events occurred in 45.5% of patients receiving URO-902 24 mg, 46.2% receiving 48 mg, and 50.0% receiving placebo. The most commonly occurring adverse event was urinary tract infection (0% in individuals receiving the 24 mg dose of URO-902; 15.4% in those receiving the 48 mg dose; and 3.8% in those receiving placebo). One patient in the 48 mg arm of the study had asymptomatic elevated post-void residual urine volume at week 2; this resolved spontaneously and did not require catheterization.
URO-902 is a unique potential treatment for OAB. It brings together the accessibility of the anatomy of the condition with a new innovative approach to therapy, said Sef Kurstjens, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Urovant Sciences. Later this year, Urovant anticipates 48-week data from the Phase 2a trial, at that point, well have a greater sense of the durability of the therapy and our proposed next steps.
The data were first presented earlier this year at the 2022 annual meeting of the American Urological Association (AUA2022) in New Orleans, La., from May 13-16, 2022.
About Overactive Bladder
Overactive bladder (OAB) is a clinical condition that occurs when the bladder muscle contracts involuntarily. Symptoms may include urinary urgency (the sudden urge to urinate that is difficult to control), urgency incontinence (unintentional loss of urine immediately after an urgent need to urinate), frequent urination (usually eight or more times in 24 hours), and nocturia (waking up more than two times in the night to urinate).1
While 33 million US adults experience the bothersome symptoms of OAB, approximately 546 million people 20 years are affected by OAB worldwide. 1,2
About the Phase 2a Study of URO-902
The 48-week multicenter study was a randomized, double-blind, placebo-controlled trial to evaluate the efficacy, safety, and tolerability of a single physician administered dose of URO-902, a novel gene therapy being developed for patients with OAB who have not been adequately managed with oral or transdermal pharmacologic therapy. URO-902 is administered via direct intradetrusor injections into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and urge urinary incontinence (UUI).
The Phase 2a trial enrolled 80 female patients in two cohorts: the first cohort received either a single administration of 24 mg of URO-902 or matching placebo, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Multiple outcome measures were explored, including the effect on the number of micturitions, urgency episodes, and quality-of-life indicators compared to placebo, 12 weeks post-administration, as well as an assessment of the safety and tolerability of this potential new therapy. Patients were followed for up to 48 weeks after initial administration.
About URO-902
URO-902 (plasmid human cDNA encoding maxi-K channel) has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment has the potential to address an unmet need for patients who have failed oral pharmacologic therapies.
References: 1. Irwin DE, Kopp ZS, Agatep B, Milsom I, Abrams P. Worldwide prevalence estimates of lower urinary tract symptoms, overactive bladder, urinary incontinence and bladder outlet obstruction. BJU Int. 2011;108(7):1132-1138. doi:10.1111/j.1464-410X.2010.09993.x
2. Leron E, Weintraub AY, Mastrolia SA, Schwarzman P. Overactive bladder syndrome: evaluation and management. Curr Urol. 2017;11:117-125. doi:10.1159/000447205
About Urovant Sciences
Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need.
About Sumitovant Biopharma
Sumitovant is a technology-driven biopharmaceutical company accelerating development of new potential therapies for patients with high unmet medical need. Through our subsidiary portfolio and use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovants subsidiary portfolio includes wholly-owned Enzyvant, Urovant, Spirovant, and Altavant, and one majority-owned subsidiary that is publicly listed: Myovant (NYSE: MYOV). Sumitomo Pharma is Sumitovants parent company. For more information, please visit http://www.sumitovant.com.
UROVANT, UROVANT SCIENCES, the UROVANT SCIENCES logo are trademarks of Urovant Sciences GmbH, registered in the U.S. and in other countries. All other trademarks are the property of their respective owners. 2022 Urovant Sciences. All rights reserved.
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Urovant Sciences Receives Best in Category Award for Abstract Highlighting Investigational Novel Gene Therapy, URO-902, Presented at 2022...
Tegan Taylor: There are a few things in life that are just inevitable; death, taxes, the genes you're born with. At least, that has been the case for pretty much every generation up until now. Gene therapies have the potential to change the trajectory of disease, and I've been talking to two people on the frontline of that shift.
Until about three years ago, Robert Lamberth had a disease that was incurable. I mean, it was literally in his genes.
Robert Lamberth: Not as a newborn, but yes, very, very young when I had my first bleed. Three, I think it might have been for me, back in the early '80s it was, a long time ago now.
Tegan Taylor: When he was born, he inherited a certain recessive gene that stopped his body from producing one of the essential factors you need for your blood to clot.
Robert Lamberth: Bleeding internally into my major weight-bearing joints, so ankles and knees. And as I got older, I'd have more odd bleeding into muscles in my legs and parts of my stomach and those sorts of things, so it was a little bit more serious when you have large muscle bleeds. The pressure of the bleeding can affect your organs, so that's quite serious.
Tegan Taylor: Managing haemophilia A is miles easier than it was a couple of decades ago. When he was little, Robert needed intravenous injections of his missing clotting factor, given in a hospital. When he got older, he didn't need to go to hospital anymore. Regular injections of the clotting factor were a feature of his life all the way through into his 30s. But not anymore.
John Rasko: We dream of cures in gene therapy but hesitate to use the word
Tegan Taylor: For decades, John Rasko has been chasing ways to change people's fates.
John Rasko: For the last 20-plus years we've been doing clinical trials using viral vectors to transfer a gene into humans for a therapeutic purpose.
Tegan Taylor: Professor Rasko is a haematologist and pathologist who spent much of his career studying genes, stem cells and basically how to hack processes inside the human body. And he is one of many scientists around the world trying to figure out ways of swapping out disease-causing genes in a way that, in time, could be used for pretty much any genetic disease.
John Rasko: When we reflect on rare diseases, it's often worth remarking and reminding ourselves that rare diseases of course by definition are rare, usually less than one in 5,000 or 10,000 people, but collectively rare diseases are very common when you add them all up because there are many thousands of them, lead to a burden of disease such as the commonality of diabetes or even some forms of cancer. So the problem is that of all the rare diseases, which some people say are more than 4,000 affecting humans, 80% of those rare diseases have a genetic basis. And of those diseases, only 5% have a specific therapy. So this is an incredible unmet need in human health.
Tegan Taylor: And the solution he and his colleagues have come up with might sound a bit familiar. It works in a similar way to the Covid vaccine made by AstraZeneca. It uses a harmless virus to take a genetic message into the body.
John Rasko: And that vector system is used to then ferry that genetic payload intravenously to the liver where it takes up residence, and hopefully after a single injection, corrects that person's genetic abnormality for the rest of their life. It's unimaginable, but a single injection can alter the course of a genetic disease that would otherwise affect a person from birth to death.
Tegan Taylor: Robert was part of the clinical trial Professor Rasko was involved in, testing the gene therapy.
Robert Lamberth: It would be three years ago now in May 2019 when I had that one single dose of the good stuff, and then that clearly worked its magic and now I'm growing my own factor VIII. I've had one breakthrough bleed.
Gene therapy for me, Tegan, has been quite revolutionary, so from a position of having 0.5% of clotting factor in my blood, I'm now growing my own factor VIII in my liver and I'm at about 15% clotting factor, which is an extraordinary growth.
Tegan Taylor: In August, Europe granted conditional approval for a haemophilia A therapy like the one Robert received. It hasn't been approved in Australia yet, although we do use gene therapy for other conditions, like spinal muscular atrophy, and genetic causes of blindness.
John Rasko: We are only at the very start of this genetic revolution. There are thousands of genetic diseases that affect humans, and we've only just started scratching the surface of where we can go with these gene-based therapeutics.
Tegan Taylor: Because Robert got the gene therapy as an adult, he's still living with the damage haemophilia A had already done to his body, but that doesn't mean it hasn't been transformative.
Robert Lamberth: I can just do so much more. I can be out there doing everything that I love at work and at play and going to the gym, without fear of having a micro-bleed the next day and being cross and crotchety and painful and grumpy at work, and then it turning into a more major bleed and then having to go and seek therapy, which means even more down-time. The sooner that we could roll out some gene therapy for younger people would be great.
Tegan Taylor: Robert Lamberth, who received gene therapy for haemophilia A, finishing us off there. And we also heard from Professor John Rasko from Royal Prince Alfred Hospital and the Centenary Institute at the University of Sydney.
Norman Swan: It's interesting how things have advanced there, Tegan. A few years ago, not so long ago, gene therapy could have been quite toxic because of the virus that they were using to carry the gene in, and you've got to hit the target, it can't be wasteful, and sometimes the virus did harm in its own right. So it's taken a long time to get that right, but the potential, as John Rasko says, is huge and it goes from cancer through to these inborn errors that you get such as haemophilia A.
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The gene therapy that could transform the lives of millions - ABC News
GRENOBLE, France--(BUSINESS WIRE)--Carroucell, the microcarrier supplier for cell culture in bioreactor, announced today that it has raised a total of 1.5 million. The funding includes the closing of a Series A financing, led by the Novalis Biotech Acceleration fund and with participation of Crdit Agricole des Savoie (CADS), as well as support from Bpifrance. The funding will be used to accelerate corporate growth through industrialization of the companys platform technology and ramping up of mass production processes to GMP standards.
Carroucell has developed a disruptive technological platform that offers unique flat shape microcarriers with a glass xenofree composition for cell culture in bioreactors. Unlike existing technologies, the combination of these novel microcarriers combined with the flexibility of the production process enables a faster, more optimized scale-up of the clinical phases. This more cost-effective process could provide customers with a more accelerated time and pathway to market.
For the first time, microcarrier customization and a more customer-oriented service are available for the development of the new applications into the cell culture and bioproduction market. There are many challenges with biomanufacturing performance. We believe our unique microcarrier technology and ability to address customer specific needs will overcome most challenges and stimulate a revolution in the sector moving forward, said Tarek Fathallah, Founder and President of Carroucell.
Carroucell is creating a new standard in biomanufacturing, which could help to facilitate patient access to many more innovations in cell and gene therapy in the future, said Jan Van den Berghe, co-founder and managing director of Novalis Biotech, who has also been appointed to the board of directors. When customers adopt Carroucell's technology platform, they are able to optimize the yield and the quality of the cell culture, solving the low-performance problem in bioproduction we see today.
The complex environment of cell culture in bioreactors and the increasing number of new applications requires an innovative approach to guarantee the balance of the system. Carroucells microcarrier plays the role of regulator of this system by ensuring its optimization, said Takis Breyiannis, CEO of Carroucell.
About Novalis BiotechNovalis Biotech (Ghent, Belgium) is an early-stage venture capital investor in technologies that revolutionize healthcare. The companys core competence lies in digitalization in the life sciences with a focus on bioinformatics, genomics and diagnostics. Novalis strongly believes in applying innovative enabling technology to advance the prevention, diagnosis, or treatment of a disease. For more information, please visit http://www.noval.is.
About CarroucellCarroucell is disrupting the biomanufacturing sector with its patented, innovative microcarrier and flexible process solution for customers. The microcarriers are based on a major innovation in the field of sol-gel process, which allows the production of bioactive microstructures not achievable by existing technologies. In the bioreactor, cells can cling and multiply in "3D" and allows cultivation of a large quantity of cells in a restricted volume. Carroucell has a partnership with Etablissement Franais du Sang (EFS), which enabled the validation of its microcarriers and facilitated first commercial orders. Carroucell was founded in 2016 by Tarek Fathallah. For more information, visit http://www.carroucell.com.
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Carroucell Raises 1.5 Million to Introduce Breakthrough Microcarriers and Customizable Processes to Cell and Gene Therapy Market - Business Wire
At 44, Janet Waterhouse should have been the picture of health; a former Division I soccer player, she taught yoga, enjoyed running, and didnt drink alcohol. Despite her healthy and active lifestyle, over a span of decades she experienced a number of unexplained symptoms.
Her symptoms continued to worsen into her 20s when she began to sporadically lose function of her hands and experience severe bouts of vertigo. Most doctors attributed her symptoms to stress and anxiety. During this time, Waterhouse was seeing a pain management specialist, who was concerned enough about her worsening symptoms to run a blood test, where he found irregularly shaped blood cells, called acanthocytes.
A series of serendipitous referrals led Waterhouse to Ali Hamedani, an assistant professor of neurology and ophthalmology in the Perelman School of Medicine. Based on her symptoms and exam, he suspected a genetic condition called chronic progressive external ophthalmoplegia (CPEO) and referred her to Laynie Dratch, a certified genetic counselor in the Penn Neurogenetics Therapy Center, for genetic testing.
In May of 2022, Dratch gave Waterhouse what she had been chasing for decades: a diagnosis. When the genetic counselor told me they found the genetic mutation they were looking for, I cried for a solid five minutes out of relief, Waterhouse says.
Waterhouses case of CPEO was found to be caused by a variation on her RRM2B gene, which affects the mitochondria in her cells. While the condition is very rare and can sometimes take years to locate and diagnose, Hamedanis hunch about the gene mutation led them right to it.
Because little is known about CPEO, treatment options are limited. Most people would be discouraged by the uncertainty, she says, but it thrills me to get to be the blueprint. I get to show people how to live with this.
Launched in March 2020, the Penn Neurogenetics Therapy Center has a team of clinicians, nurses, genetic counselors, and clinical research staff who are devoted to the care of patients with inherited neurological disorders and to participating in clinical trials of novel gene and molecular therapies.
The programs mission is twofold: first, they utilize the expertise of clinicians and researchers throughout the department of Neurology and across Penn Medicine to achieve a genetic diagnosis for as many patients like Waterhouse as possible, creating a database of eligible patients for new treatments and clinical trials. Second, they work to establish clinical trials using novel gene and molecular therapies for patients with genetically-based neurological disorders.
Our genetics counselors are some of the best in the country, and are incredibly effective at diagnosing patients and matching them with effective treatments and clinical trials, says Steven Scherer, a professor of neurology and director of the Neurogenetics Therapy Center. Now we can utilize this expertise to design tomorrows therapies.
Read more at Penn Medicine News.
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Solving medical mysteries with genetics: The Penn Neurogenetics Therapy Center | Penn Today - Penn Today
BEIJING, Sept. 08, 2022 (GLOBE NEWSWIRE) -- George Clinical, a global clinical research organization with an extensive presence throughout the Asia-Pacific region, continues to expand the organizations team in China with the addition of Helen Xu as Project Director and Cell Gene Therapy Head. She will be based in Beijing and joins a rapidly growing team responsible for expanding clinical research activity in China with biopharmaceutical, medical device and diagnostic sponsors.
Dr. Xu obtained her MD in clinical medicine from Peking University Health Center. She is a licensed physician specialized in central nervous system (CNS) and has worked in the hospital setting for four years. Dr. Xu entered the pharmaceutical industry in 2007 starting as a clinical research associate (CRA) and has since accumulated 15 years of valuable clinical research experience in China.
I am sure Helen will make an incredibly valuable contribution to the growth and development of clinical research operations and cell gene therapy studies across China, said Zhenfei Yin, country head and regional head project operations, China.
Before joining George Clinical, Dr. Xu had served GSK, BI, Wuxiapp, a Chinese clinical research organization, CASI, and Carsgen, a CAR-T biotech firm. Her clinical trial experience covers the whole development lifecycle starting from phase I through PMS, the majority of the trial experiences being with pivotal trials. Therapeutic areas of expertise include cell therapy, immunotherapies, blood tumors (MM, AML, thalassemia), solid tumors (lymphoma, gastric and pancreatic cancer, prostate cancer, lung cancer), respiratory disease (IPF, asthma), SSc-ILD, CNS (stroke, schizophrenia, GAD), HCV, cirrhosis, psoriasis, and in vitro diagnostics (IVDs) devices. Her responsibilities spanned the full duration of studies from bid defense, strategic planning, feasibility, and start-up to project close-out. The majority of these pivotal global trials experienced global audits and authority inspections from entities such as FDA, EMA, and NMPA.
Additional new team members will be joining the organization to support the team in China with medical expertise
With a growing presence across the country, it is an honor to be part of global CRO able to bring further clinical research to China that can positively impact cancer care around the world, Xu said.
About George Clinical
George Clinical is a leading global clinical research organization founded in Asia-Pacific driven by scientific expertise and operational excellence. With over 20 years of experience and more than 400 people managing 39 geographical locations throughout the Asia-Pacific region, USA, and Europe, George Clinical provides the full range of clinical trial services to biopharmaceutical, medical device, and diagnostic customers, for all trial phases, registration and post-marketing trials.
LinkedIn: https://www.linkedin.com/company/george-clinical-pty-ltd
Twitter: https://twitter.com/george_clinical
Facebook: https://www.facebook.com/georgeclinical
Wechat: https://mp.weixin.qq.com/.
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George Clinical Expands China Team with New Project Director and Cell Gene Therapy Head Helen Xu - AsiaOne
Mumbai: Nearly 14 months after the clinical trials of the indigenously developed CAR-T technology for blood cancer treatment was kicked off at the Tata Memorial Centre (TMC), researchers have concluded the first phase of trials and called the results encouraging.
This is the first time that gene therapy indigenously developed by researchers at the Indian Institute of Technology (IIT), Bombay, was tested on patients in India.
Phase I clinical trial data demonstrates that Indias first indigenously developed novel CAR-T Cell therapy is safe and shows promising early sign of efficacy in treating Lymphoma, a type of blood cancer, said a statement released by IIT B, late on Sunday evening.
In June 2020, the central governments National Biopharma Mission (NBM) -Biotechnology Industry Research Assistance Council (BIRAC) had approved 18.96 crore to the team for conducting a first-in-human phase-I/II clinical trial of the CAR-T cells. The drug has the potential to benefit cancer patients, who currently are forced to opt for only palliative care.
While existing treatments work towards increasing the life of patients by a few years or months, CAR-T technology holds the promise of curing certain types of cancers. Unlike chemotherapy, this drug is administered only once to a patient.
Dr Gaurav Narula, principal investigator of the paediatric-Acute Lymphocytic Leukemia (ALL), TMC and Dr Hasmukh Jain, principal investigator of the adult B-cell lymphoma study, started recruiting patients in early 2021. So far, six patients in pediatric-ALL and 10 patients in adult lymphoma studies were treated with indigenous HCAR19.
The trials were conducted at the Advanced Centre for Treatment, Research and Education in Cancer (ACTREC), the research and development wing of TMC.
The participants received autologous HCAR19 therapy. None of the participants had immune effector cell-associated neurotoxicity syndrome. Three out of ten participants had a complete response post CAR-T cell therapy and none of the participants required ICU admission. There was no CAR-T treatment related death. Overall, the novel humanized HCAR19 tested in Phase I clinical trials for adult lymphoma was found to be safe and has shown promising early signs of activity, said Dr Hasmukh Jain, who presented the data in the Annual Symposium of Cell and Gene Therapy, CMC Vellore.
Chimeric Antigen Receptor T (CAR-T) cells are genetically engineered to produce an artificial T-cell receptor, which is widely used in developed nations for immunotherapy during treatment for cancer. As part of gene therapy, these cells are used with an intent to cure certain types of blood cancers. However, the technology is still unavailable in India.
IIT Bombay and Tata Memorial Hospital (TMH) started their R&D collaboration in 2015 to develop the novel CAR-T cell therapy platform for cancers and immune-disorders. Dr Rahul Purwar, Associate Professor, IIT Bombay (on-lien) and currently appointed as CEO of ImmunoACT, designed and developed the indigenous CAR-T platform and patented anti-CD19 CAR-T product (HCAR19). In early 2021, HCAR19 product entered into two Phase 1 clinical trials at TMH, Mumbai.
Dr Gaurav Narula will present the results of Phase I trial of paediatric B-ALL in the Asia Pacific Blood and Marrow Transplantation (APBMT) 2022 meeting soon. The clinical trials will now enter Phase-II, post approvals from the Central Drug Standard Control Organisation (CDSCO) and is expected to be available for commercial clinical usage in 2024.
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IIT Bombay and Tata Memorial Hospital (TMH) started their R&D collaboration in 2015 to develop the novel CAR-T cell therapy platform for cancers and immune-disorders
This is the first time that gene therapy indigenously developed by researchers at the Indian Institute of Technology (IIT), Bombay, was tested on patients in India
CAR-T cells are genetically engineered to produce an artificial T-cell receptor, which is widely used in developed nations for immunotherapy during treatment for cancer
While existing treatments work towards increasing the life of patients by a few years or months, CAR-T technology holds the promise of curing certain types of cancers. Unlike chemotherapy, this drug is administered only once to a patient.
Shreya Bhandary is a Special Correspondent covering higher education for Hindustan Times, Mumbai. Her work revolves around finding loopholes in the current education system and highlighting the good and the bad in higher education institutes in and around Mumbai....view detail
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IIT-B-Tata hosp cancer therapy trials show promising results - Hindustan Times