StemCell Therapy

Both studies were conducted as pilot studies in a pre-post observational design. Details are already reported elsewhere [13]. The primary outcome for both studies was the proportion of days with reaching the fasting goal of 15h out of the total number of days recorded per participant in the diary. According to the study protocol, secondary outcomes were, among others, changes in sleep quality and duration, and HRQoL between baseline and follow-up.

Participants at the Ulm University were recruited with the support of the occupational health management and by flyers. Exclusion criteria were pre-existing metabolic conditions. Patients at the GPs office were informed about the study by flyers in the waiting room or were invited by the doctor during a consultation. Exclusion criteria were insulin dependent diabetes or any other disease for which fasting is contraindicated [3]. Finally, 63 participants at the Ulm University and 40 participants at the GPs office were included in the studies.

Participants in both studies were asked to limit their daily food intake to 89h and subsequently extend their nightly fasting period to 1516h. The duration of the intervention was 3 months. At baseline, participants had an introductory conversation with the principal investigator or the physician to clarify possible questions and problems in advance, and were given an information brochure. In addition, all participants were offered to contact the respective study centre at any time if they had questions or problems.

Baseline assessment comprised a questionnaire to collect data on lifestyle, health behaviour and HRQoL (EQ-5D VAS) [14], and anthropometric measurements of waist, height, and weight (for details see [13]). All participants were given a diary to record the times of their first and their last meal, and the quality and duration of their sleep. The latter was assessed on a visual analogue scale ranging from 0 (worst possible sleep quality) to 100 (best possible sleep quality). The waist-to-height ratio (WHtR) was calculated by the division of waist by height in centimetre, abdominal obesity was then defined as WHtR 0.5, as recommended by the literature [15]. Body weight in kilogram was divided by height in meters squared to determine body mass index (BMI), and subsequently categorized into overweight ( 25) and obesity ( 30).

After 3 months, follow-up measurements were performed in the same manner, with some additional items in the questionnaire regarding the individual experience and attitudes towards TRE.

Baseline characteristics are reported descriptively for each study group and for both groups combined. Differences between groups were tested by applying t-test, Welchs t-test or Mann Whitney U test according to distribution and heterogeneity in variance for continuous data, and Fishers exact test for categorical data.

Follow-up data, and computational differences between baseline and follow-up data, presented as the respective , were treated the same way. Pre and post comparisons for both groups taken together were determined by the Wilcoxon signed-rank test for related samples.

For each participant, mean values and standard deviations were calculated for the data from the diaries. Time of first meal and time of last meal were utilized to determine the duration of the fasting and the eating phase. For all days recorded, the percentage of days with fasting target reached was calculated. Differences between groups were tested as described above.

To assess differences between sleep duration and quality at the beginning and at the end of the TRE intervention period, mean values were calculated for the first 10% and the last 10% of data (or days), respectively. Subsequently the differences between the first and the last 10% of the data were calculated as the respective . They are reported together with the average number of days recorded per group and for the whole group.

Pearsons correlation coefficient was applied to test bivariate correlations between continuous variables.

Linear regression analyses were conducted for the pre-post differences in HRQoL and the differences in sleep quality between the first 10% and the last 10% of days recorded. Potential factors that might correlate with the HRQoL or sleep quality were identified and, together with variables that differed at baseline between both groups, tested in a stepwise backward elimination. Sex, age, baseline values of HRQoL, the sleep quality and sleep duration on the first 10% of reported days, mean duration of fasting, percentage of fasting target reached, and finally group membership were considered as potential associated factors. Anthropometric measures represented both, potential associated factors and differences between groups at baseline. Therefore, weight, waist circumference, BMI, WHtR, overweight, obesity, abdominal obesity as well as the respective between pre and post measures of the continuous variables were considered in the regression analysis. All assumptions of linear regression (linear relationship, multivariate normality, multicollinearity, auto-correlation, homoscedasticity) were examined.

The significance level for two-sided tests was set at =0.05. All statistical analyses were carried out by using the statistical software packages IBM SPSS Statistics for Windows, Version 25.0. (IBM Corp., Armonk, NY, USA).

Link:
Associations of time-restricted eating with health-related quality of life and sleep in adults: a secondary analysis of two pre-post pilot studies -...

Eat This, Not That!

For months, Dr. Anthony Fauci, the nation's top infectious disease expert and the director of the National Institute of Allergy and Infectious Diseases, said the coronavirus vaccines would be so safe, he'd take one. And in fact others have been looking to him to see if it's safe before taking theirs. "People like Anthony Fauci, who I know, and I've worked with, I trust completely," former President Barack Obama said. "So if Anthony Fauci tells me this vaccine is safe, and can vaccinate, you know, immunize you from getting Covid, absolutely I'm going to take it."Today, Fauci took it. Read on to see what he saidand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had Coronavirus.Fauci Feels "Extreme Confidence" in the VaccineBefore getting the first dose of the Moderna shot, Dr. Fauci said "it's important for two reasons. One is that I'm an attending physician here on the staff at the National Institutes of Health Clinical Center. And so I do see patients, but as important or more important is as a symbol to the rest of the country, that I feel extreme confidence in the safety and the efficacy of this vaccine. And I want to encourage everyone who has the opportunity to get vaccinated so that we could have a veil of protection over this country that would end this pandemic."When You'll Get Your Own VaccineWith two vaccinesone from Pfizer-BioNTech and one from Modernadistribution is expected to "speed up," say experts. "We know we have the vaccine available to get to 20 million people by the end of December and then a total of 50 million by the end of January," Alex Azar, the United States Secretary of Health and Human Services, told the Today Show.Fauci has previously said he's hopeful that "by the time you get into the middle, towards the end of the first quarter of 2021, you will have accounted for and vaccinated those who are in the higher priority groups." Then, "I would project by the time you get to April, it will be 'open season,' in the sense of anyone, even the non-high priority groups could get vaccinated," Fauci said.His next few months will be devoted to convincing you to get yours; the vaccine needs to be taken by enough people so we can develop herd immunity. "Why do I say that it is the only way that we're going to end this epidemic?" he asked earlier this month. "If we get, I would say a range from 70 to 85% of the population vaccinated, we will create an umbrella or a blanket as it were of herd immunity over the population to the point that the virus is not going to have any place to go in us. In other words, we could essentially end this outbreak in this country, as we know it, and put it in the rear view mirror.""I think now that we have in our hands vaccines that are 94 to 95% effective," he said, "so we need to make sure that we make that efficacy turn into effect."RELATED: If You Feel This, You May Have Already Had COVID, Says Dr. FauciHow to Survive This PandemicAs for yourself, follow Fauci's fundamentals and help end this surge, no matter where you livewear a face mask, social distance, avoid large crowds, don't go indoors with people you're not sheltering with (especially in bars), practice good hand hygiene, get vaccinated when it becomes available to you, and to protect your life and the lives of others, don't visit any of these 35 Places You're Most Likely to Catch COVID.

See the original post here:
Robert Lewandowski is the best football player of 2020 - Yahoo Canada Shine On

The argument for continued investment

C> is a high potential and maturing sector, and is an already crowded environment, playing host to numerous start-ups and now, through M&A, recognised big pharma firms. Much like the rush to find a COVID-19 vaccine that dominates headlines worldwide, not every company involved will be able to succeed.

But finnCaps finnLife watch list of 50 leading AIM-listed biotech companies demonstrates that there is room for numerous companies to contribute to, and profit from, C>. Examining three entirely different approaches to CAR-T therapy, it is possible to see just how much space there is for this exciting sector, therefore displaying the case for continued investment.

Innovative CAR-T therapy demonstrates the depth of C> potential

CAR-T therapy in its existing form is a relatively new and specialised approach at treating cancer. It takes T cells from a patients bloodstream and genetically modifies them in a laboratory. These T cells are then injected back into the bloodstream with the aim of targeting and killing cancer cells.

While it has been shown to be an effective treatment, there are risks and side effects. One is the two-step autologous process (the slow time it takes for cell expansion sometimes as long as two weeks) while another is cytokine release syndrome (CRS), which occurs when cytokine molecules are inadvertently released, but too quickly to target just the tumours and instead target healthy cells.

The next generation of CAR-T treatments shows that there is space for a multitude of start-ups to be active in the C> space as they all help find varied solutions to these problems without negating the effectiveness of CAR-T.

One example is Horizon Delivery, a company that is developing its CYAD-02 project, which will help transport T cells more effectively to the tumour via the use of SMARTvector products.

The product underwent its first phase 1 trial test in January 2020 with a patient who was suffering from acute myeloid leukaemia. Horizon Delivery is also an industry leader in CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) screenings, meaning they can identify key genes or genetic sequences that draw out specific functions of a cell type from thousands of potential variants.

In a cancer context, this means they can route out and exclusively eliminate problematic cells that may have shown signs theyd resist a future cancer treatment.

Another example is Maxcyte, a global cell- based therapies and life sciences company that is developing its CARMA process, where a patients peripheral blood mononuclear cells (PBMCs) are removed and modified. The modified cells can then be used to target an array of different cancers.

Currently the company is conducting a phase 1 trial for advanced ovarian cancer in a dose escalation trial that will treat four separate cohorts the fourth of which was administered in March 2020.

Another example which shows the versatility of new CAR-T innovation is provided by Oxford Biomedica, a gene and cell therapy company specialising in the development of gene-based medicines.

Rather than a contained project or platform, its contribution to CAR-T is through a contract manufacturing development organisation. Collaborating with pharma companies, Oxford Biomedica uses its infrastructure to produce other companies licensed products, including Novartis Kymriah treatment (alongside other undisclosed CAR-T-related products).

With fast-moving innovation finally allowing multiple C> treatments to gain regulatory approval, along with a huge pipeline of upcoming therapies and an influx of funding and M&A activity, investing in C> no longer entails taking a bet on potential the future is finally here.

More here:
After years of potential, cell and gene therapy is ready for the pharmaceutical mainstream - PMLiVE

Neurogene has raised $115 million in a second-round financing that will accelerate its plans to start clinical trials of a range of gene therapies for inherited neurological diseases, initially focusing on a form of Batten disease.

Batten disease is a group of disorders caused by a deficiency in proteins that allow fatty substances build up in nervous tissue, causing seizures, visual impairment, mobility loss and early death.

The New York biotechfounded by former Wall Street analyst Rachel McMinn, Ph.D., two years agosaid some of the cash will be used to advance its lead programs targeting Batten disease caused by CLN5 and CLN7 mutationstwo rare and rapidly progressive subtypes that occur in later childhood.

Compliantly Digitize Your Global Operations and Quality Process with a Remote Workforce

With the COVID-19 pandemic disrupting global supply chains and forcing many to work from home, life sciences organizations are under pressure to quickly adapt while adhering to rigorous compliance standards. Please join experts, as they discuss best practices on he challenges and opportunities COVID-19 poses on the life sciences industry and more. Register Today!

It will also fund development of candidates for lysosomal storage disorders Charcot-Marie-Tooth disease (CMT) type 4J, caused by changes in the FIG4 gene, and aspartylglucosaminuria (AGU), which arises when the AGA gene is mutated.

The funding will also go toward developing its gene therapy platform and scaling up its manufacturing capacity. It adds to the $68.5 million the biotech raised in its series A round last year.

The AGU and CMT4J programs had been scheduled to reach the clinic this year, but Neurogene has been making quicker progress with the Batten disease drive.

Furthest along right now is its CLN5 candidate, which should start clinical trials next year. Batten disease is an autosomal recessive disorder, which means it only develops if a person inherits two copies of a faulty gene for their parents.

Neurogene intends to treat it by using an adeno-associated virus vector to deliver a replacement CLN5 gene, restoring the activity of the protein it codes for. While the function of that protein isnt well understood, the hope is that restoring its activity will slow down or halt the progression of the disease.

Neurogene isnt the only group eyeing the potential for gene therapies to make a difference to the lives of patients with Batten disease.

Abeona Therapeutics has been working in this area for a few years, and earlier this year licensed its ABO-202 candidate for CLN1 diseasealso known as infantile Batten diseaseto Taysha Gene Therapies for $7 million upfront and up to $56 million in milestones. It is also developing ABO-201 for CLN3 disease.

Last year, Amicus Therapeutics presented the first results with its CLN6 gene therapyacquired as part of its $100 million takeover of Celenex in 2018that seem to indicate a slowing of neurological decline.

Meanwhile, researchers at Cornell University have carried out a phase 1 gene therapy trial in late- infantile (CLN2) disease with similar positive results, and have also started a phase 1/2 study in this group.

Gene therapy has generated tremendous hope for the many families and patients with severe genetic disorders, McMinn said.

We believe our focus on improved product design, innovative technology, cutting-edge vector manufacturing and premier analytics will help fulfill the potential of genetic treatments.

Read more here:
Neurogene adds another $115M to the pot for trials for its gene therapies - FierceBiotech

Tada Images/Shutterstock

Thermo Fisher Scientific has been as busy as Santas elves this December. The company is significantly expanding its footprint with new facilities in the United States and in Europe.

This week, the company announced it started construction of a new cGMP plasmid DNA manufacturing facility inCarlsbad, Calif.This expansion builds on the company's continued investment in cell and gene therapy services. The site will expand the company's clinical and commercial capabilities for cGMP plasmid DNA used as a critical raw material to develop and manufacture cell and gene-based therapies including life-saving cancer treatments as well as mRNA vaccines. In addition, the site will have the capability to produce large-scale plasmid DNA as a primary drug substance for DNA therapies.

The California site will span 67,000 square feet and is expected to be completed in the first half of 2021. The facility will feature advanced technologies, including single use equipment with up to 1,000L scale, digital connectivity and data visibility to enable operational efficiencies and operator training, the company said. The new commercial facility will add approximately 150 jobs over the next 12 months.

The race to develop new transformative cell and gene therapies and vaccines is outpacing supply of commercial-quality plasmid DNA that can be produced at scale, Mike Shafer, senior vice president and president of pharma services at ThermoFisher said in a statement. Our new state-of-the art site will not only tackle the supply bottleneck for our customers, but also uniquely positions us to deliver robust, end-to-end cell and gene therapy capabilities.

Shafer said Thermo Fisher is making strategic investments in capacity, technology and expertise across its global network so the company can accelerate innovation and enhance productivity for its customers.

The California site isnt the only expansion the company is undertaking. To meet accelerating demand for robust clinical supply chain services throughout Europe, Thermo Fisher Scientific has expanded its pharma services footprint with two new facilities inRheinfeldenand Weil am Rhein, Germany. The new sites will boost clinical supply chain continuity and specialized cold chain and cryogenic expertise across Europeand globally.

TheRheinfeldensite opens in late December 2020 and the Weil am Rhein site will open in January 2021.InRheinfelden, the new 86,000-square-foot/8,000-square-meter facility significantly increases the company's footprint for secondary packaging, storage, logistics and distribution of clinical supplies to investigator sites across Europe. In Weil am Rhein, the new 9,600-square-foot/890-square-metercryocenterprovides specialized ultra-low-temperature, cryogenic storage and cold chain expertise for clinical supply chain needs for cell and gene-based therapies, including COVID-19 vaccine candidates.

These facilities combined with our established regulatory expertise will give customers the continuity and in-region capabilities to support clinical trials across multiple therapy areas, Shafer said.

In addition to the construction of new facilities, earlier this month, Thermo Fisher announced the expansion of several of its existing sites that will increase the companys capabilities for sterile drug product development and commercial manufacturing of critical medicines, therapies and vaccines.

Thermo Fisher is expanding its facilities in Greenville, N.C., Swindon, England and two sites in Italy, Ferentino and Monza. Not only will the expansion of these facilities boost commercial production lines and support capabilities for aseptic liquid and lyophilized vial filling, but the projects will also add approximately 1,000 new jobs. The expansions are expected to be completed over the next two years, Thermo Fisher said in its announcement.

In addition to expansions inNorth AmericaandEurope, the company recently announced significant projects inAsia-Pacific, including a new sterile manufacturing facility inSingaporeand a new integrated biologics and sterile drug development and manufacturing site inHangzhou, China.

Most Read Today

See the article here:
Thermo Fisher Invests in Gene Therapy Future with Huge Expansions - BioSpace

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 22 December 2020 18:50Hits: 116

TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct

TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118s open investigational new drug application for CLN1

Interim data from Phase 1/2 trial anticipated in 2021

DALLAS, TX, USA I December 21, 2020 I Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queens University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Tayshas investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis.

TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. GM2 is a devastating lysosomal storage disease with no approved treatments and todays CTA approval marks a formative moment for children suffering from this rapidly progressive and fatal disease.

The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queens University and led by Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Professor Head, Division of Medical Genetics (Department of Pediatrics) at Queens, and Director of Research (Department of Pediatrics), at the Kingston Health Sciences Centre.

Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival, said Dr. Jagdeep S. Walia. We are pleased to have the support of Health Canada as we continue to advance TSHA-101.

Todays CTA approval is a culmination of our teams and Dr. Walias tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers, said RA Session II, Founder, President and CEO of Taysha. We are grateful to our partners at Queens University for their work to advance this gene therapy into the clinic.

About GM2 Gangliosidosis

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

About TSHA-101

TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes HEXA and HEXB driven by a single promoter within the same AAV9 construct, also known as a bicistronic vector. This approach allows the simultaneous expression of a 1:1 ratio of the two subunits of protein required to generate a functional enzyme. It is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA).

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

SOURCE: Taysha Gene Therapy

The rest is here:
Taysha Gene Therapies Announces Queen's University's Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of...

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases.

Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline.

This reflects a potential consideration of up to $26.50 per share in cash for a total consideration of approximately $1.04bn.

For Lilly, the acquisition will extend its focus into developing gene therapies, establishing an in-house gene therapy programme anchored by Prevails current portfolio and AAV-based technology.

Prevails pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

The companys preclinical pipeline also includes PR004, a potential gene therapy for patients with specific synucleinopathies, as well as candidates for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders.

"The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy programme anchored by well-researched assets, said Mark Mintun, vice president of pain and neurodegeneration research at Lilly.

We look forward to completing the proposed acquisition and working with Prevail to advance their ground-breaking work through clinical development, he added.

For Prevail to achieve the full value of the contingent CVR payment, the first regulatory approval arising from its current gene therapy pipeline must happen by 31 December 2024.

Failing regulatory approval by this date, Lilly said in a statement that the value of the CVR will decrease by approximately 8.3 cents per month until the expiration date 1 December 2028.

Within Prevails clinical pipeline, PR001 has already scored a US Food and Drug Administration (FDA) fast-track designation for the treatment of PD-GBA patients and nGD.

It has also been granted an FDA orphan drug designation for the treatment of Gaucher disease, and rare paediatric disease designation for the treatment of nGD.

Prevails PR006 gene therapy also has an FDA and European Commission orphan designation for the treatment of FTD, with the FDA also handing it a fast-track designation for FTD-GRN.

In November, Lilly signed a deal with Precision BioSciences focused on genome editing research, with an initial focus on developing in vivo therapies for Duchenne muscular dystrophy and two other undisclosed gene targets.

View original post here:
Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal - PMLiVE

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

See the article here:
After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News

Taysha Gene Therapies went public with a bang earlier this year with a $157 million IPO just five months after its Series A financing. Now, with the help of the city of Durham, North Carolina, Taysha is plotting an expansive development and manufacturing facility to ramp up supplies for the clinic and potentially the commercial market.

The new 187,000-square-foot cGMP plant will create roughly 200 jobs for Taysha Gene Therapies, which formed in late 2019 with the sole focus of developing therapeutics for monogenic diseases using an adeno-associate virus methodology. The announcement continues a rapid ascent for Taysha, which launched Series A financing just in April, and a mere five months later had its own Nasdaq ticker, offering a $157 million IPO and pricing shares at $20 apiece.

The facility is the result of a public-private partnership between Taysha, the city of Durham and the state of North Carolina. Taysha will invest $75 million into the facility, with additional state and local incentives totaling another $9.4 million. All told, the 200 jobs will come to fruition over a two-and-a-half-year period across all functions, including gene therapy development, analytics, manufacturing and quality control testing.

Tayshas facility is expected to be fully operational by 2023 and will add 2,000 liters of capacity supporting all aspects of scalable gene therapy manufacturing. The company said in a news release it will now be able to meet the clinical and commercial demands within the field when combined with its existing collaborations with the University of Texas Southwestern Medical Center and CDMO Catalent.

With our outstanding team of experts leading the charge, we expect this facility will serve as a center of excellence for gene therapy development, from preclinical studies through commercialization, and will further our leadership position in gene therapy as well as support our next phase of growth, RA Session II, Tayshas president, founder and CEO, said in a press release.

The new facility will serve as an integral part of rapid expansion for the Dallas-based biotech, which said it expects to have four open Investigational New Drug applications in 2021. Tayshas most advanced of those is a drug being developed for the treatment of GM2 gangliosidosis, a family of neurodegenerative disorders that includes Tay-Sachs disease and Sandhoff disease.

The other three such programs are looking to treat Rett Syndrome, epilepsy and SURF1 deficiencies.

Visit link:
Taysha Gene Therapies looks to build on rapid IPO with $85M dev-manufacturing plant in North Carolina - Endpoints News

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/fltbmv

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

http://www.researchandmarkets.com

Read this article:
Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,...

NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium being held February 8-12, 2021.

Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening, said Michael Amoroso, Chief Operating Officer of Abeona. We are excited to share new analyses from the Transpher A study that will add to the understanding of the potential for ABO-102 to help preserve neurocognitive development in patients with MPS IIIA when they are treated at a young age, and new results from the Transpher B study that will provide insights into ABO-101s biologic effect in patients with MPS IIIB.

Presentation Details

Title: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)Abstract Number: 390Presenter: Kevin Flanigan, M.D., Center for Gene Therapy at Nationwide Childrens HospitalDate/Time: Friday, February 12, 2021, time to be determined

Title: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)Abstract Number: 407Presenter: Maria Jose de Castro, M.D., Hospital Clnico Universitario Santiago de CompostelaDate/Time: Friday, February 12, 2021, time to be determined

About the Annual WORLDSymposium The WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium, please visit https://worldsymposia.org/.

About the Transpher A Study The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About the Transpher B Study The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102 ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101 ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

Go here to see the original:
Abeona Therapeutics Announces Acceptance of Late-Breaker Abstracts Highlighting New Clinical Data for Novel AAV-based Gene Therapies in MPS IIIA and...

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

View original post here:
Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

Originally posted here:
Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

I have heard about people using genes to treat diseases nowadays, but I am not sure what this gene therapy means.

Gene therapy involves trying to alter or modify the genes inside your bodys cells in order to treat or stop a disease.

Since 2017, the US Food and Drug Administration (FDA) has approved three different types of gene therapy.

Maybe we can start at the beginning: what are genes?

Genes are the basic physical and functional unit of heredity.

Our genes are made out of DNA (deoxyribonucleic acid).

Each person has two copies of each gene one inherited from your mother and the other inherited from your father.

Each human being has around 20,000 to 25,000 genes.

These genes code for the way your body and mind are structured.

Some genes act as instructions (a blueprint) for your body to make various proteins, which in turn form your cells and organs, and the enzymes and hormones that regulate your body.

Other genes do not code for proteins.

Most genes are the same for all human beings, which is why we all look like human beings (and not a kangaroo, fish, bird or an alien)!

However, just under 1% of our genes vary slightly between each person.

That is why we have different races, heights, propensity for different diseases, curly or straight hair, etc.

These small differences also contribute to why we all look different from one another.

Genes that dont work as they should also cause diseases in the human body.

What types of diseases are caused by faulty genes?

These are what we call genetic disorders.

A genetic disease is any type of disease caused by an abnormality in our genetic blueprint.

This abnormality can range from very minor to significantly major.

What we consider minor is, for example, a small mutation in the DNA of a single gene resulting in the change of a single base protein.

What we consider major is a gross chromosomal abnormality, such as the addition of a whole chromosome or the subtraction of one.

Some genetic disorders are inherited from our parents.

Others are caused by mutations due to our environment.

Examples of single gene disorders, which are caused by the alteration of just one gene in our bodies, are:

Examples of multifactorial inheritance, which are caused by a combination of environmental factors and mutations in many of our genes, are:

If we inherited these genes from our parents, then how can we possibly modify or alter them? This sounds terribly like science fiction.

We are rapidly approaching that era where what used to be science fiction could become part of our everyday life.

In gene therapy, scientists can:

How do they do this? Do they have to harvest my cells? Im scared just thinking about it!

Many of the vectors are viruses, especially adenoviruses (not coronaviruses!).

Viruses have a natural ability to deliver genetic material into our cells.

After all, their main purpose is to attach themselves to cells and reproduce themselves.

Sometimes, the vector or virus is injected straight into our bodies, where they will deliver the gene that will modify our cells.

They are injected straight into the part of our body that has those defective cells.

Other times, we have to harvest healthy tissue from our body that needs to be modified.

These are usually tissues containing immune cells or stem cells, e.g. blood or bone marrow.

These tissue samples are then taken to the lab and specific cells are separated out.

The viral vector containing the corrective gene is then introduced to the harvested cells in the lab.

The modified cells are left to multiply, and then injected back into us.

Once inside our bodies, they will continue to multiply and eventually treat the disease or correct the defect within us.

Learn more about gene therapy in the next Tell Me About column on Dec 31 (2020).

Dr YLM graduated as a medical doctor, and has been writing for many years on various subjects such as medicine, health, computers and entertainment. For further information, email starhealth@thestar.com.my. The information contained in this column is for general educational purposes only. Neither The Star nor the author gives any warranty on accuracy, completeness, functionality, usefulness or other assurances as to such information. The Star and the author disclaim all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.

Read more here:
What is gene therapy? - The Star Online

University of Minnesota Twin Cities researchers in the Department of Chemistry have created a new polymer to deliver DNA and RNA-based therapies for diseases. For the first time in the industry, the researchers were able to see exactly how polymers interact with human cells when delivering medicines into the body. This discovery opens the door for more widespread use of polymers in applications like gene therapy and vaccine development.

The research is published in the Proceedings of the National Academy of Sciences (PNAS), a peer-reviewed multidisciplinary scientific journal.

Gene therapy involves altering the genes inside the bodys cells to treat or cure diseases. It requires a carrier that packages the DNA to deliver it into the celloftentimes, a virus is used as a carrier. Packaging of nucleic acids is also used in vaccines, such as the recently developed messenger RNA (mRNA) COVID-19 vaccine, which is enclosed in a lipid.

The research team is led by chemistry professor Theresa Reineke and associate professor Renee Frontiera. Reinekes lab synthesizes polymers, which are long-chain molecules that make up plastics, to use for packaging the nucleic acids instead.

Its kind of like ordering something from Amazon, and its shipped in a box, Reineke explained. Things get broken if theyre not delivered in a package. Thats basically what were doing here but on a nano-level. Were taking these really sensitive RNA and DNA cargo that are susceptible to enzymatic degradation, that wont get to their target unless you have something to protect them.

The researchers designed the copolymer using quinine, a naturally occurring substance used in tonic water, and 2-hydroxyethyl acrylate (HEA), which makes the material soluble and is used in a variety of personal care and medical materials. Because quinine is fluorescent, the research team was able to track the DNA package throughout the body and into the cells using Raman spectroscopy, a chemical imaging technique.

Weve discovered a new packaging tool with this natural product thats important for all of these high-flying, important fields like gene therapy and vaccines, said Reineke, who is also a Distinguished McKnight University Professor. And, it works in a variety of cell-types. On top of that, its got all of these cool featuresits fluorescent, we can track it, its Raman active, and that allowed us to understand a lot of fundamentals about these packaging systems that were impossible to probe before we incorporated this natural product.

Polymer-based drug delivery is significantly cheaper than using viruses, especially for gene therapy, which can cost up to $2 million for a single injection. However, the main barrier preventing widespread polymer use was that scientists didnt know a lot about how the polymer package actually interacts with cells in the body.

This research helps clear up that uncertainty. Frontieras lab specializes in chemical imaging. Using Raman spectroscopy, they discovered that a cells own proteins play a key role in unpacking the nucleic acid cargo once the polymer carrier enters the cell.

Its very satisfying to know how this is actually happening, what the process of delivery is, and to actually see that in real-time, Frontiera said. A key point is that these polymers also work very well. For all the beneficial attributes, theyre also incredibly effective at getting the payload into cells, and we were able to tell why, which doesnt always happen in this field.

Reineke and Frontiera have been collaborating since 2013. Reinekes lab has patented the quinine polymers, and the researchers hope that a company might license this technology in the future. The College of Science and Engineering team also collaborated with University of Minnesota Medical School Distinguished McKnight University Professor Jakub Tolar to test the effectiveness of the polymer carriers in relevant cell types.

Other members of the research team include chemistry researchers Craig Van Bruggen (Ph.D. student) and David Punihaole (postdoctoral associate), chemical engineering and materials science student Andrew Schmitz, and genetics Ph.D. student Allison Keith. This research was funded by the National Science Foundation and the National Institutes of Health.

Read the full research paper entitled Quinine copolymer reporters promote efficient intracellular DNA delivery and illuminate a protein-induced unpackaging mechanism on the PNAS website.

Read more from the original source:
Researchers discover new way to deliver DNA-based therapies for diseases - UMN News

A Boutique Pilates Studio in Verona, NJ. We offer Private and Duet Pilates sessions at the studio as well as remote pilates classes online.

We know that many people feel intimidated walking into a gym or Pilates Studio.At Longevity Pilates of New Jersey, our goal is to offer you a warm, personalized, nurturing, non-threatening approach to fitness.We've been helping people attain their fitness goals for more than 15 years.

With more than 15 years of teaching experience our certified teachers are well equipped to work with people of all ages, sizes and fitness levels.Whether you are training for a marathon, a retired grandmother, looking to improve your golf swing, trying to lose weight or recovering from an injury, our certified Pilates teachers will put together a Private or Duet session specifically designed for your individual needs. Live Long. Live Strong.

COVID-19 Update: At Longevity Pilates, we are doing all we can to keep you safe. This includes masking, disinfecting, and observing social distancing. If you prefer to work from home, we also offer remote Pilates training and group mat Zoom class on Saturdays at 1pm.

See the rest here:
Longevity Pilates of New Jersey

Eating healthy doesnt have to be complicated. Theres not a long list of rules you need to follow, or a complicated diet plan to stick to. Just ask one of the many centenarians living in the Blue Zones, who will tell you a big part of living a long and happy life is enjoying a wholesome, plant-forward diet. And your refrigerator is a good place to start stocking foods for longevity.

Through his research, Dan Buettner, longevity expert and author ofThe Blue Zones Kitchen, has found its mostly about making healthy choices every day that, together, add up to a pretty amazing life. None of the Blue Zones centenarians Ive ever met tried to live to 100. No one said at age 50, You know what, Im going to get on that longevity diet and live another 50 years! They dont count calories, take vitamins, weigh protein grams, or even read labels. They dont restrict their food intakein fact, they allcelebrate with food, says Buettner. As we have applied the wisdom of the worlds Blue Zones diet to transform cities in the United States, Ive begun to believe that we can create the same sort of culture here.

Turning your home into a Blue Zone can be as simple as keeping your refrigerator, freezer, and pantry stocked with healthy staples to promote longevity.

According to Buettner, nut-eaters outlive those who dont eat nuts. Because of that, hes a big proponent of eating a handfulaka two ouncesof mixed nuts a day. Nuts come in a variety of flavors, and theyre full of nutrients and healthy fat that satiate your appetite, says Buettner. Small quantities are best, since the oils in nuts degrade (oxidize). Larger quantities can be stored in the refrigerator or freezer for a couple of months.

Buettner says 95 percent of the food you eat should be plant-based.In the Blue Zones, people eat an impressive variety of garden vegetables when they are in season, and then they pickle or dry the surplus to enjoy during the off-season, he says. The best of the best longevity foods in the Blue Zones diet are leafy greens such as spinach, kale, beet and turnip tops, chard, and collards. The Blue Zones also recommends broccoli, Brussels sprouts, cabbage, cauliflower, and peppersreally, anything you like.

Fresh fruit is another Blue Zones refrigerator staple. Think apples, melons, grapefruit, oranges, and clementines. Fruit that lasts a long time, he says. You can also keep bananas in your fridge. He says doing so when theyre almost ripe will make them last longer. Aside from filling your fridge with fruit, placement is also key. Buettner recommends keeping your fruit (as well as your veggies) on the top shelf of your refrigerator, as doing so puts them at eye level and encourages you to eat more of them.

Animal protein is limited when youre eating like you live in the Blue Zones. Designate two days a week when you eat meat or other animal-derived foodand enjoy it only on those days, Buettner says. Instead, opt for plant-based protein, like beans in your pantry and tofu in your fridge. Find plant-based substitutes for the meat Americans are used to having at the center of a meal, he says. Try lightly sauted tofu, drizzled with olive oil, or tempeh. There are so many different ways you can use tofu, too, all of which are anything but boring.

While meat is limited, Buettner says you can eat up to three ounces of fish daily, as those who ate a plant-based diet that included small portions of fish lived the longest. Favor mid-chain fish like trout, snapper, grouper, sardines, and anchovies, he says. And steer clear of farmed fish, as they are typically raised in overcrowded pens that make it necessary to use antibiotics, pesticides, and coloring.

Dairy should be reduced when youre eating a Blue Zones diet. While Americans have relied on milk for calcium and protein for decades, in the Blue Zones diet people get these nutrients from plant-based sources, Buettner writes. One cup of cooked kale or two-thirds of a cup of tofu, for instance, provides just as much bioavailable calcium as a cup of milk. He also recommends using alt-milks, including unsweetened soy, coconut, or almond milk: Most have as much protein as regular milk and often taste as good or better.

Blue Zones guidelines stipulate that eggs arent necessary for living a long life. Keep your egg consumption to no more than three eggs per week, says Buettner. If you choose to eat eggs, select a variety from chickens that range freely, eat a wide variety of natural foods, and dont receive hormones or antibiotics, Buettner says.

Tracy Lockwood Beckerman, RD, says eggs are part of a healthy diet: Eggs are cheap, satiating, and easy to find, and theyre an excellent source of high quality protein, she says. Your body is able to fully absorb all the protein from the eggs to help lower blood pressure. If you choose to eat eggs regularly, just make sure theyre high-quality to keep them Blue Zones-approved.

For more healthy recipes and cooking ideas from our community, join Well+Goods Cook With Us Facebook group.

Read the original:
6 Foods You Should Always Have in Your Refrigerator, According to a Longevity Expert - Well+Good

NEW YORK, Dec. 18, 2020 /PRNewswire/ --Capt. Franz Almeida has a practiced eagle's eye, honed over thousands of flight hours as a pilot. Junaid Mian, RPh, understands the challenges inherent to maintaining human health through the lens of work as a pharmacist. Both followed unique paths, which led them to angel investing. The two met at the NY chapter of the Harvard Business School.

Together they identified a yawning gap between space technology and biotechparticularly longevityverticals. They recognized that the world's economy is in the throes of change so radical that future generations will see the 21st century as a clear delineation marking a decidedly Earthbound humanity split from humanity that can freely live in and explore space.

The space economy explosion is happening, and there is no better time to invest than as early on as possible during that explosion. The latest estimates of the global space economy are well over $400 billion, and if growth continues to accelerate, many analyses point to a $1 trillion space economy just around the corner. The human longevity market is nearly as massive: A Merrill Lynch analysis revealed that the sector generates over $110 billion annually now and is growing to over $600 billion by 2025.

Without massive and ongoing investment and progress across both space technology and human longevity verticals, we will never be able to truly unlock the potential that our Solar System, and our galaxy, holds.

"A big part of why progress against aging used to be so slow was that so few experts on aging had an engineer's way of thinking. The convergence of those communities is making all the difference."

Aubrey de Grey, VP of New Technology Discovery at AgeX Therapeutics, Inc and Chief Science Officer of the SENS Research Foundation and Human Longevity advisor at SP8CEVC

And that's where Junaid Mian, RPh, and Capt. Franz Almeida comes in. The pair's varied perspectives have been a decisive match when it comes to examining the potential in merging investments across the space tech and human longevity verticals. Almeida and Mian are launching a new Rolling Fund in partnership with AngelList, with an exacting focus on solving the problem that is the future of humankind.

Under the umbrella of SP8CEVC, LPs will be able to gain exposure to deals to empower growing ventures in space technology and human longevity. Why these two rather specific categories? Simple: Moving to an economy based on the resources of our entire Solar System enables a much more substantial timeline for humanity's existence, and biotechnology work in longevity enables people to live and work in space.

The SP8CEVC partners who initially started with a traditional fund structure have chosen to use AngelList's rolling fund Reg 506(c) model to open up investment possibilities in these most critical verticals. As a series of quarterly pooled investment funds, SP8CEVC will give investors access to their deal flow quarterly on a subscription basis.

This new structure accelerates the pace of investing and, as a benefit, also helps promote innovation in those businesses.

The SP8CEVC team launched yesterday at TechCrunch Sessions: SPACE 2020. Investors and LPs interested in setting the pace for future advancement can have access to SP8CEVC's deal flow before anyone else here.

Media Contact:Franz Almeida917-287-5674 [emailprotected]

SOURCE SP8CEVC

https://www.sp8cevc.com/

Read more from the original source:
Introducing SP8CEVC: The World's First Space Technology and Human Longevity Focused Rolling Fund in Partnership with AngelList - PRNewswire

MUMBAI: From the start of daily soaps in 2000, Hindi GEC fiction haswitnessed multiple eras: From the now-notorious saas-bahu dramas in the first decade, to shows addressing societal evils and women empowerment,tofamily-based love stories that brought in the younger viewers. With each passing era, characters in the Hindi GEC category havebecome smarter, more fashionable and more progressive in their outlook. Whats interesting, however, is that characters who have managed to retain long-term popularity among the audience have one aspect common to their personalities: they are generally over-indexed on the personality dimension sincerity.

In her brand personality framework, psychologist Jennifer Aaker identifies five dimensions to a personality sincerity, excitement, competence, sophistication and ruggedness. Most brands will have some image associations with these dimensions, but the more enduring brands largely have one emphasised primary dimension, and optionally a secondary one.

While originally designed for brands, the framework works equally well for celebrities and characters. In the Hindi GEC category, the one emphasised dimensionacross most long-standing popular fiction characters is the sincerity dimension. Five of the seven popular Hindi GEC characters between 2016-19 (based on Ormax Characters India Loves)had sincerity as their strongest, or a strong number two, dimension. These five characters span a width of age, profession and content genres. Theres themiddle-aged shop keeper and familyman Jethalalin a sitcom; young dentistIshita engulfed in across-cultural family drama where she falls in love with a divorced father;the quintessential girl-to-bahu journey of Akshara; the young and quirky Pragya who marries a rock star accidentally; and the talentedvillage singing prodigy Kullfi,who moves to the city in search of her father.

The trend continues in 2020. The undisputed success story of this year has been Star PlusJuly launch Anupamaa. The show has achieved record ratingsin a challenging and disruptive Covid2019 period. The lead character of the showraced to number two on Ormax Characters India Loves within a month of the shows launch, a new record in character popularity tracking in the last decade.

Anupamaas rapid growth is a combination of various factors, such as a unique yet relevant concept (it addresses the anti-family topic of extra-marital relationship in a very family-palatable way), strongwriting and direction, and the performance by thelead actor herself. It is no surprise, however, that Anupamaas character too is heavily loaded on the sincerity dimension, even more than the five characters listed above.

So why do sincere characters strikesuch adeep chord with the audience?

Storytelling on Indian televisionis different from films and web-series, because in serials, audience look for ideas that are important to them. They connect with charactersthatare avatars of themselves. They feel their feeling, live their emotions. As a collectivistic society, most Indians value family cohesion and cooperation as a non-negotiable aspect of their identity. They take great pride, and seek comfort, in the strong emotional bonds they share with their family members. Watching stories driven by characters who reinforce family values give these shows a higher purposebeyond entertainment.

In this context, characters like Jethalal and Anupamaa are the quintessential Indianfamily man and Indian family woman respectively, who value their families and relationships above everything else. While the former is in a sitcom and the latter leads a familydrama, it is their fundamentallyearnest personalities thatendear them to the Indian value system. Coupled with good storytelling, suchcharacters build long-term equity, engaging the audience with their journeys that fundamentally revolve around their large families in a manner thats truly Indian at heart, i.e.,sincere!

Story and storytelling will continue to get more progressive with time. But the cherished place characters high on sincerity have in the hearts of Indian TV audience is unlikely toweaken anytime soon.

(The author is Ormax Media partner. Indiantelevision.com may not subscribe to their views.)

View original post here:
Sincerely yours: Key to longevity in the Hindi GEC category - Indiantelevision.com

The iconic former goalkeeper, who worked alongside the Portuguese in Spain, feels the five-time Ballon dOr winner can continue for a very long time

Cristiano Ronaldo's small rivalry with Lionel Messi has made him a better player, claims Iker Casillas, with the Juventus superstar being backed to remain at the top for a very long time.

The Portugal international missed out on the latest FIFA Best prize to Bayern Munich striker Robert Lewandowski,but he has collected that award on two previous occasions and has five Ballons dOr to his name.

Only Barcelona icon Messi can claim to have bettered that return, with the mercurial Argentine another of those to have sent records tumbling over the course of a remarkable career.

Ronaldo has matched the South American stride for stride, re-writing the history books himself on a regular basis, with two all-time greats using the exploits of one another as added motivationwhen raising their own individual bars.

Real Madrid icon Casillas admits as much, with the World Cup winner telling The Nationalof his former Blancos team-mate Ronaldo and why the 35-year-old has starred for so long: Obviously hes a good professional, he looks after himself, eats well, gives the necessary importance to rest also.

So this is just a result of being what a footballer is. Other than that, hes also a great athlete. The reason is hes always hungry and always wanting to improve and better himself.

Perhaps also his small rivalry with [Lionel] Messi has helped him better himself. But its basically down to his professionalism and ambition; that has taken him to the top of the footballing world.

Ronaldo has been showing no sign of slowing down during his time in Italy, with 79 goals recorded through 101 appearances for Juve.

He has also reached 102 efforts for his country, as he looks to become international footballs all-time leading marksman,and appears to have plenty left in the tank.

Casillas cannot see the Portuguese hanging up his boots any time soon, adding on how much longer a modern-day phenomenon can go on for: I hope he can continue to play for a very long time. But, of course, football and its nature is what it is.

We all know that everything must come to an end and time does go by for everyone. Hopefully we will be able to still enjoy his football for a long time, but its difficult to say.

Ronaldo is tied to a contract in Turin through to the summer of 2022 and has stated in the past that he intends to play on beyond his 40th birthday.

Link:
Ronaldos rivalry with Messi has made him better Longevity of Juventus star doesnt surprise Real Madri... - Goal.com