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Category Archives: Genetic Therapy

Advarra Announces New Gene Therapy Ready Site Network – PRNewswire

This will address an accelerating gene therapy market that is expected to grow globally by 16.6 percent from 2020-2027.

"The Gene Therapy Ready network demonstrates our commitment to empowering sites and supporting our industry partners as they pursue advanced genetic engineering to find cures for the world's most pressing health conditions," said Scott Uebele, President and Chief Research Services Officer at Advarra. "Our commitment to efficient study activation is unwavering, and this is another example of how Advarra bringslife sciences companies,CROs, research sites, investigators,andacademiatogether at the intersection of safety,compliance,technology, and collaboration."

All Gene Therapy Ready sites stand ready to help industry sponsors conduct clinical trials that advance cures, develop vaccines, and find treatments for rare disease. By placing clinical trials with a Gene Therapy Ready site, research sponsors can save significant time during study startup.

"This innovative network is truly the first of its kind. We constantly look for ways to support our sponsors in rapidly starting trials in a safe, compliant, and quality manner. With the Gene Therapy Ready network, we can improve study startup times by a month or more, potentially placing cures in the hands of patients faster," said James Riddle, Vice President of Research Services and Strategic Consulting at Advarra. "The Gene Therapy Ready site network charts a course to success by providing our sponsor clients with a clear choice for IBC review services."

About Advarra

Advarra advances the way clinical research is conducted: bringing life sciences companies, CROs, research sites, investigators, and academia together at the intersection of safety, technology, and collaboration. With trusted IRB and IBC review solutions, innovative technologies, experienced consultants, and deep-seated connections across the industry, Advarra provides integrated solutions that safeguard trial participants, empower clinical sites, ensure compliance, and optimize research performance. Advarra is advancing clinical trials to make them safer, smarter, and faster. For more information, visit

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Taysha Gene Therapies Receives Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5…

There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease, said RA Session II, President, Founder and CEO of Taysha. We are encouraged by the early evidence of TSHA-105s disease-modifying approach and believe these designations will help us potentially accelerate the development of this exciting program. We look forward to working with the FDA to make TSHA-105 available to patients as expeditiously as possible.

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. This rare form of epilepsy manifests as developmental delay, and seizures beginning within the first few days of life.

We are pleased that the FDA recognizes TSHA-105s potential as an innovative therapeutic option for SLC13A5 deficiency, said Rachel Bailey, Ph.D., Assistant Professor in Pediatric Neurology at UT Southwestern. This disease is a debilitating form of genetic epilepsy in children that significantly impacts movement, motor control, cognition and quality of life, and there remains a need to alter the course of this disease early in life.

As a mother of two children with SLC13A5 deficiency, I have witnessed firsthand the devastating impact that numerous seizures and comorbidities accompanying the disease has on those affected by this disease, said Kim Nye, Founder of TESS Research Foundation. Tayshas commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-105, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Chinese scientists develop gene therapy which could delay ageing –

BEIJING: Scientists in Beijing have developed a new gene therapy which can reverse some of the effects of ageing in mice and extend their lifespans, findings which may one day contribute to similar treatment for humans.

The method, detailed in a paper in the Science Translational Medicine journal earlier this month, involves inactivating a gene called kat7 which the scientists found to be a key contributor to cellular ageing.

The specific therapy they used and the results were a world first, said co-supervisor of the project Professor Qu Jing, 40, a specialist in ageing and regenerative medicine from the Institute of Zoology at the Chinese Academy of Sciences (CAS).

The team of biologists from different CAS departments used the CRISPR/Cas9 method to screen thousands of genes for those which were particularly strong drivers of cellular senescence, the term used to describe cellular ageing.

They identified 100 genes out of around 10,000, and kat7 was the most efficient at contributing to senescence in cells, Qu said.

Kat7 is one of tens of thousands of genes found in the cells of mammals. The researchers inactivated it in the livers of the mice using a method called a lentiviral vector.

"We just tested the function of the gene in different kinds of cell types, in the human stem cell, the mesenchymal progenitor cells, in the human liver cell and the mouse liver cell and for all of these cells we didn't see any detectable cellular toxicity. And for the mice, we also didn't see any side effect yet."

Despite this, the method is a long way from being ready for human trials, Qu said.

"It's still definitely necessary to test the function of kat7 in other cell types of humans and other organs of mice and in the other pre-clinical animals before we use the strategy for human ageing or other health conditions," she said.

"In the end, we hope that we can find a way to delay ageing even by a very minor the future."

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Promising new gene therapy is allowing paralysed mice to walk again – Happy Mag

Some good news is on the horizon for 2021, after a team of researchers were able to restore movement in mice that had suffered a complete spinal cord crush, thanks to the development of a signalling protein.

The protein, known as hyper-interleukin-6 (hIL-6), does not occur naturally and can only be produced via genetic engineering.

Published in the journal of Nature Communications, the study authors found that using gene therapy to stimulate the mices neurons allowed the animals to start producing the protein, causing the damaged nerve cells (axons) to regrow in just a few weeks.

Currently, paralysis resulting from severed nerve fibres in the spinal cord has been permanent and irreparable. A type of signalling molecule known as a cytokine, hIL-6 can signal select cells to regenerate.

In fact, scientists from the Department for Cell Physiology at Ruhr-Universitt Bochum (RUB) in Germany previously found that the hIL-6 protein could also cause damaged axons in areas as complex as the visual cortex to regenerate.

However, to determine hIL-6s effectiveness at repairing damaged spinal axons, the team injected the brains of injured mice with a virus that contained the necessary genetic code for the production of hIL-6. This virus delivered the blueprint for protein production to specific nerve cells, called cortical motoneurons. These motoneurons are easily accessible and can communicate with other parts of the central nervous system, which are vital for movement processes like walking.

Most amazingly, these cortical motoneurons are also linked via axons to the raphe nuclei, which sit within the brainstem and are the primary producers of the neurotransmitter serotonin. Technobabble aside, serotonin not only plays a crucial role in big-nights, but also in locomotor recovery following spinal cord injuries. But, the raphe nucleis position made them impossible for researchers to access directly until now.

Thus, gene therapy treatment of only a few nerve cells stimulated the axonal regeneration of various nerve cells in the brain and several motor tracts in the spinal cord simultaneously, Professor Dietmar Fischer noted.

Ultimately, this enabled the previously paralyzed animals that received this treatment to start walking after two to three weeks. This came as a great surprise to us at the beginning, as it had never been shown to be possible before after full paraplegia.

The research team is now investigating the extent to which hIL-6 can be combined with other measures to achieve additional regenerations. Furthermore, the team are also exploring whether the treatment still works in mice, even if the injury occurred several weeks previously.

This aspect would be particularly relevant for application in humans, Professor Fisher said.

We are now breaking new scientific ground. These further experiments will show, among other things, whether it will be possible to transfer these new approaches to humans in the future.

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Regenerative Medicine Market Size Worth $23.57 Bn By 2027; High demand for 3D bioprinting of tissues and organs to better understand their mechanism…

New York, Jan. 18, 2021 (GLOBE NEWSWIRE) -- Increased investment in advanced technologies for treatment of genetic and chronic diseases is driving growth of the regenerative medicine market.Market Size USD 7.34 Billion in 2019, Market Growth - CAGR of 15.6%, Market TrendsApplications in COVID-19 vaccine.

The global regenerative medicine market is forecast to reach a market size of USD 23.57 Billion by 2027, and register a robustly incline revenue growth, according to a new report by Reports and Data. Primary factors driving demand for regenerative medicines are advancements in surgical technology and monitoring devices, and major increase in prevalence of complex and degenerative diseases. Upsurge in incidence of cancers has been resulting in increasing research into stem cell therapy. Growth in research and development activities in emerging countries and rising focus on stem cell research is resulting in significant growth in the global revenue of regenerative medicine market.

Stem cell technology is growing rapidly and continues to play a crucial role in regenerative medicine and the related field. This technology opens up the possibility of treating Parkinsons Disease, arthritis, and spinal cord injury. Increase in demand for stem cell technology is a major factor driving growth of the regenerative medicine market.

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Recent developments in regenerative medicine for 3D bioprinting, stem cell treatment for heart repair, and vision loss has created demand for additional investments in the R&D of the technology to help with other diseases.

The COVID-19 impact:

Demand for regenerative medicine has witnessed increased demand during the COVID-19 pandemic. Regenerative medicine helps in understanding a mechanism of infection and to develop ways to prevent the spread of the virus. It is also being used to create advanced treatments to treat persons infected by the COVID-19 virus. Private companies are also using it to develop an effective vaccine for COVID-19.

Regenerative Medicine Market Size, Share & Industry Demand By Product (Tissue-Engineered Products, Cell Therapies, Gene Therapies, Progenitor & Stem Cell Therapies), By Application (Musculoskeletal Disorders, Oncology, Wound Care, By Material), and Region, Segment Forecast to 2027, To identify the key trends in the industry, click on the link below:

Further key findings from the report suggest

List of Key Companies Identified in the Regenerative Medicine Market Report:

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For the purpose of this report, Reports and Data has segmented into the global regenerative medicine market on the basis of product, application, material, and region:

Browse similar research reports:Cell Therapy Market By Therapy Type (Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy), By Therapeutic Area (Malignancies, Autoimmune Disorders, Musculoskeletal Disorders), By Cell Type, And By End User, Forecasts To 2027

Tissue Engineering Market Size, Growth & Analysis, By Material, By Application (Cancer, Urology, Neurology, Dental, Cell Banking & Cord Blood, Gynecology, Integumentary/Skin, Spine, Musculoskeletal, & Orthopedics, Vascular & Cardiology), And Region, Segment Forecasts To 2027

Gene Expression Market By Product And Services (Equipment, Consumables, And Services), By Capacity (Low- To Mid- Plex Gene Expression Analysis And High-Plex Gene Expression Analysis), By Application (Diagnostic, Drug Discovery, Research), And Segment Forecasts To 2027

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Regenerative Medicine Market Size Worth $23.57 Bn By 2027; High demand for 3D bioprinting of tissues and organs to better understand their mechanism...

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COVID-19 Slashed the US Life Expectancy by More Than a Year – Futurism

Thanks to the coronavirus pandemic, the average life expectancy in the United States dropped more suddenly than it had previously in over 40 years.

Researchers from the University of Southern California (USC) and Princeton University found that U.S. life expectancy dropped by 1.13 years in 2020, according to research published last week in the journal PNAS. And thats the national average marginalized groups like Black and Latino Americans faced an even more precipitous drop in life expectancy, revealing the stark inequalities within American society and healthcare.

A one-year drop may not sound massive, but as Live Science reports, its a tenfold decrease over recent drops in life expectancy that were linked to higher rates of deaths by suicide or drug overdose. It also brings the expected lifespan of an average American to its lowest point since 2003, undoing the progress made by nearly two decades of medical advancements.

The study also found that the average life expectancy for marginalized groups dropped by even more: 2.1 years for Black Americans and 3.05 years for Latino ones, compared to a drop of just 0.68 years for white Americans.

The generally good health of Latinos prior to the pandemic, which should have protected them from COVID-19, has laid bare the risks associated with social and economic disadvantage, study coauthor and Princeton population research expert Noreen Goldman said in a press release on the study.

Thankfully, multiple coronavirus vaccines are already being distributed. But that doesnt mean that the expected lifespan will immediately climb back to pre-pandemic levels, according to the study.

While the arrival of effective vaccines is hopeful, the U.S. is currently experiencing more daily COVID-19 deaths than at any other point in the pandemic, study coauthor and USC gerontologist Theresa Andrasfay said. Because of that, and because we expect there will be long-term health and economic effects that may result in worse mortality for many years to come, we expect there will be lingering effects on life expectancy in 2021.

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