FA Community Conversations Genetic Therapies Webinar | Recorded August 29, 2024

The following information was recorded as part of a genetic therapy webinar on August 29, 2024. Information shared in this webinar is for educational purposes only and should not be construed as medical advice. Those viewing the recording after August 29, 2024 are reminded that the field of genetic therapy is rapidly evolving and the information contained in this recording may have changed. Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular study.
FARA's Mission is to marshal and focus the resources and relationships needed to cure Friedreich's Ataxia (FA) by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.
Friedreich’s Ataxia is a debilitating, life-shortening, degenerative neuro-muscular disorder. About one in 50,000 people in the United States have Friedreich's Ataxia.
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[@Baernhardiner]

The Brunel University of London has a ongoing Cell an Gene Therapy approach aswell. But it's not on your pipeline chart. Do you have knowledge of it?

[@CureFA]

Absolutely! We are excited about the work being done at Brunel University of London, and FARA is proud to have provided grant funding for this mouse study: www.curefa.org/funded-grants/therapeutic-activity-of-a-haematopoietic-stem-cell-delivered-tissue-penetrating-peptide-in-a-friedreichs-ataxia-mouse-model/
There are many promising academic research projects underway, and FARA is committed to supporting these efforts. While projects like the one at Brunel are currently in the academic research phase, we're excited to add them to our drug development pipeline if they progress to a company or enter human clinical trials. We're always closely watching these developments and are excited to see how they advance!