SAN FRANCISCO–(BUSINESS WIRE)–AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, today announced a multiyear collaboration with 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.
“Taysha has brought together accomplished and knowledgeable gene therapy and CNS disease experts to develop potentially transformative therapies”
The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome, a deadly rare disease that primarily affects infants. AllStripes will use its platform, which gives patients control over their health histories, to unify otherwise scattered and fragmented SURF1-associated clinical data, allowing researchers to uncover new insights into the natural history and burden of disease and better inform the development of clinical studies.
“This collaboration will allow us to leverage the AllStripes technology platform to optimize our therapeutic strategy and to potentially accelerate the development of TSHA-104 in SURF1-associated Leigh syndrome,” said RA Session, II, president, founder and chief executive officer of Taysha. “We remain committed to developing a safe and effective gene therapy for patients suffering with this devastating disease, and data generated from this unique collaboration could bring us one step closer to our goal.”
Mutations in the SURF1 gene prevent mitochondria from producing enough energy for cells in the body to function normally, leading to Leigh syndrome, a severe and rare neurological disorder characterized by progressive loss of mental and movement abilities. SURF1-associated Leigh syndrome typically presents during infancy or early childhood, and often results in death within a few years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation. There is currently no targeted treatment or cure for SURF1-associated Leigh syndrome.
“Taysha has brought together accomplished and knowledgeable gene therapy and CNS disease experts to develop potentially transformative therapies,” said Nancy Yu, co-founder and chief executive officer of AllStripes. “With no available treatment for SURF1-associated Leigh syndrome, we are very pleased to empower patients and their families with an avenue to participate in research that will support the development path of TSHA-104. We are hopeful that this novel gene therapy will bring meaningful benefit to children and their families, and give them more time together.”
TSHA-104 has been granted rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of SURF1-associated Leigh syndrome. An Investigational New Drug (IND) application for TSHA-104 in SURF1-associated Leigh syndrome is expected to be submitted to the FDA in 2021.
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 team’s 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 platform—an engine for potential new cures—with a goal of dramatically improving patients’ lives. More information is available at www.tayshagtx.com.
AllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates FDA-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, Spark Capital, Maveron Capital, Village Global, Garuda Ventures and a number of angel investors. For more information, visit www.allstripes.com.
Katie Engleman, 1AB
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