neuromuscular disease

Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD

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Liz Soragni, PhD, Director of Research at the Friedreich’s Ataxia Research Alliance In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD

CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders 

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Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) announced in January $100 million in funding for a broad range of projects, including four to support clinical programs.  One of these was a $2.8 million award to Patricio Sepulveda, PhD, MBA, CEO of Amplo Biotechnology, Inc. That grant will support the development of a gene … Continue reading CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders