Taysha Gene Therapies (TSHA) Announces Collaboration with AllStripes on SURF1-Associated Leigh Syndrome Clinical Development and Natural History

January 4, 2021 7:08 AM EST
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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 a multi-year collaboration with AllStripes (formerly RDMD), a healthcare technology company dedicated to accelerating research for patients with rare diseases, during which Taysha will leverage AllStripes’ platform to inform its understanding of SURF1-associated Leigh syndrome natural history and burden of disease, as well as patients’ diagnostic journeys.

The collaboration will focus on advancing the development of TSHA-104, an AAV9-based gene therapy product candidate in development for the treatment of SURF1-associated Leigh syndrome. Taysha will utilize AllStripes’ clinical database to uncover new insights into disease progression and better inform selection of endpoints for 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.”

“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.

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