Dear Friedreich’s Ataxia Community,
We are excited to officially introduce Solid Biosciences to the FA community. Solid Biosciences was founded by individuals directly impacted by Duchenne muscular dystrophy, driven by a deeply personal mission to develop meaningful therapies for patients and families. Since that time, our mission has expanded to include other urgent, rare neuromuscular and cardiac conditions, but we continue to be guided by the same passion, urgency and patient centricity that inspired our beginnings.
Today, we are proud to connect with the FA community to share news of a meaningful update to the developing treatment landscape: SGT-212, our first-in-industry dual-route of administration gene therapy has received FDA IND clearance.
This program originated with FA212 LLC, a company founded by FA families, led by Tom Hamilton; the development of SGT-212 would not have been possible without their visionary efforts. Additionally, the Friedreich’s Ataxia Research Alliance (FARA), the University of Pennsylvania, and most importantly, the FA patient community all contributed to the development of this program. The insights and feedback shared by individuals and families living with FA have been instrumental in shaping this program, ensuring that it reflects the real hopes and needs of the community. Your resilience and determination inspire us every day, and we are honored to bring SGT-212 to patients.
A Therapy Built on Listening to the FA Community
SGT-212 represents an innovative approach to treating FA, shaped by the patient community’s concerns and priorities. Families have consistently emphasized the need for a therapy that addresses both the debilitating neurologic symptoms and life-threatening cardiac manifestations of FA.
To meet these needs, SGT-212 uses a dual-route of administration approach to deliver full-length frataxin protein:
- Intravenous (IV) infusion targets the cardiac manifestations of FA, with potential to treat other impacted organ systems, and
- Intradentate nuclei (IDN) infusion directly targets the region of the cerebellum most affected by, and implicated in, FA-associated neurologic decline.
This unique approach aims for both precision delivery and optimal dosing to critical tissues and aspires to provide a meaningful and differentiated treatment option capable of addressing the full spectrum of the disease.
What’s Next?
In the second half of 2025, Solid Biosciences plans to initiate a Phase 1b clinical trial for SGT-212. This study will evaluate the safety and tolerability of the therapy in non-ambulatory followed by ambulatory adults with FA and will follow participants out to five years, marking an important step toward delivering this promising therapy to patients.
Join the Conversation
We invite you to join a live conference call tomorrow, January 8, 2025, at 8:30 AM ET, where we will further discuss our FA program as well as other initiatives. While this call is primarily for investors, we encourage members of the FA community to join and learn more about our broader efforts. The call can be accessed via the Events page of our website at Events • Solid Biosciences Inc.
In addition, we will join FARA in the coming weeks for a community webinar. This event will provide an opportunity to engage with our team, ask questions, and dive deeper into our plans for SGT-212. Details will be shared soon, and we hope you will join us for this important discussion.
Please see our most recent press release for more information related to our FA Program, including information related to forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 contained in the letter. The press release can be found here.
Together, We Are Solid
From our origins to this new chapter with the FA community, Solid Biosciences has always been guided by the voices of patients and families. Your feedback has shaped our programs including SGT-212, and we remain steadfast in our mission to develop therapies that address the real and pressing needs of those we serve. Thank you for welcoming us into your community. We look forward to continuing the dialogue and working alongside all of you to advance meaningful therapies for Friedreich’s ataxia and beyond.
Sincerely,
Annie Ganot, co-founder & head of patient advocacy
Solid Biosciences