Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome

Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021

Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

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 it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for TSHA-104 in 2021.

“We have now obtained rare pediatric disease and orphan drug designations in multiple gene therapy programs, which we believe will allow us to work more effectively with the FDA as we advance our broad portfolio,” said RA Session II, President, CEO and Founder of Taysha. “The receipt of these designations highlights the dedication that our team has to advancing our gene therapy pipeline as efficiently and rapidly as possible.”

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.

“Being diagnosed with a mutation in the SURF1 gene is a truly devastating event for families,” said Kasey Woleben, Founder of Cure SURF1 Foundation. “Taysha’s commitment to developing a gene therapy for SURF1 deficiency is greatly welcomed by the patient community and has the potential to save the lives of children afflicted with this progressive disorder.”

Taysha has secured rare pediatric disease designation and orphan drug designation for multiple of its programs, including GM2 gangliosidosis, CLN1, Rett syndrome and now SURF1. In addition to these designations, the company also has fast track status for the CLN1 program.

“SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. 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

Read more

Nation’s Largest Suicide Prevention Organization Celebrates National Suicide Hotline Designation Act (S.2661) Becoming Law

Nation’s Largest Suicide Prevention Organization Celebrates National Suicide Hotline Designation Act (S.2661) Becoming Law

PR Newswire

WASHINGTON, Oct. 19, 2020

WASHINGTON, Oct. 19, 2020 /PRNewswire/ — On Saturday, October 17, the National Suicide Hotline Designation Act (S.2661), legislation that will support the implementation of the future 9-8-8 crisis hotline, was signed into law. Robert Gebbia, CEO of the American Foundation for Suicide Prevention (AFSP), the nation’s largest suicide prevention organization, released the following statement:

AFSP.org
AFSP.org

“In July, the Federal Communications Commission announced that 9-8-8 would be the new universal hotline number for the National Suicide Prevention Lifeline by July 2022. This easy-to-remember number will increase public access to mental health and suicide prevention crisis resources, encourage help-seeking for individuals in need, and is a crucial entry point for establishing a continuum of crisis care.

“This is why AFSP applauds the U.S. Congress for prioritizing suicide prevention through unanimous passage of the National Suicide Hotline Designation Act. This historic legislation, now law, will bring our mental health crisis response system into the 21st century. We are encouraged by the federal government’s dedication to preventing this leading cause of death and showing all Americans that mental health is just as important as physical health.

“It should be noted however that the United States’ mental health crisis response system is woefully underfunded and undervalued. It is crucial that local crisis call centers are adequately equipped to respond to what we expect will be an increased call volume and provide effective crisis services to those in need when 9-8-8 is made available in July 2022.

“We would like to especially thank the legislation’s sponsors in the U.S. Senate, Sens. Cory Gardner (R-CO), Tammy Baldwin (D-WI), Jerry Moran (R-KS), and Jack Reed (D-RI), and the U.S. House, Reps. Chris Stewart (R-UT), Seth Moulton (D-MA), Greg Gianforte (R-MT), and Eddie Bernice Johnson (D-TX), as well as their staff. We would also like to thank the Committee members, Congressional leadership, and staff who have supported these efforts on Capitol Hill.

“AFSP’s nearly 30,000 volunteer Field Advocates engaged their members of Congress for years in support of the National Suicide Hotline Designation Act. They made their voices heard through thousands of letters, phone calls, and emails. They have pushed the suicide prevention movement forward, and their efforts will save lives.”

The National Suicide Prevention Lifeline’s 9-8-8 number will be active nationwide by July 2022. Until that point, those in crisis should continue to call the Lifeline at 1-800-273-TALK (8255).

For guidelines on how to report safely on suicide: https://afsp.org/for-journalists/.

The American Foundation for Suicide Prevention is dedicated to saving lives and bringing hope to those affected by suicide. AFSP creates a culture that’s smart about mental health through education and community programs, develops suicide prevention through research and advocacy, and provides support for those affected by suicide. Led by CEO Robert Gebbia and headquartered in New York, and with a public policy office in Washington, D.C.

Read more