Gene Therapy Pioneer Jim Wilson Comments on Solid Biosciences's Announcement of FDA IND Clearance for Friedreich's Ataxia Treatment

PHILADELPHIA, Jan. 8, 2025 /PRNewswire/ -- Today, Dr. Jim Wilson, President and CEO of GEMMA Biotherapeutics ("GEMMABio") commented on Solid Bioscience's announcement of clearance by the Food and Drug Administration for its Investigational New Drug (IND) to treat Friedreich's ataxia (FA):

"We are absolutely delighted with the announcement by Solid Bio on the successful IND clearance of the dual route gene therapy to treat both the neurological and cardiac manifestations of FA. The genesis of this program stemmed from our long term commitment to the FA community and our close relationship with the Friedreich's Ataxia Research Alliance (FARA) and members of the community including Tom Hamilton who helped fund this work.

“Based on feedback from the FA patient community, we and our collaborators, including Solid Biosciences, decided the best path forward was to treat both the neurologic and cardiac manifestations of the disease which required two different routes of administration – intravenous (IV) to treat the cardiac disease and direct central nervous system (CNS) injections to treat the neurologic diseases. This ambitious plan, the preclinical aspects of which were conducted while we were at the University of Pennsylvania, required numerous mouse and nonhuman primate studies spanning over 5 years.

"This was without question the most complicated program of the 17 that we have helped bring into the clinic. I am extremely proud of our team in helping achieve this important milestone for the FA community. I want to thank Bo Cumbo and the team at Solid for their commitment to clinically develop and commercialize this ambitious program and we stand prepared to help in whatever way we can."

About GEMMABio
GEMMABio is a therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company will provide research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably.

GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia. Wilson is also the Chairperson of Franklin Biolabs, a Contract Research Organization. The collective goal of the two affiliated companies is to translate innovative scientific work into clinical trials, to then commercialize and distribute the new therapies around the world to patients who need them most.

For more information, please visit gemmabiotx.com.

Contact: 
Kristen Love
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SOURCE GEMMA Biotherapeutics