Boston – October 28, 2026 -- People living with Friedreich's ataxia (FA) have a new gene therapy candidate heading toward human testing: Solid Biosciences says the FDA has cleared its Investigational New Drug (IND) application for SGT-212, with a Phase 1b trial planned for the second half of 2025.
SGT-212 attacks FA through two delivery routes
The company describes SGT-212 as the first gene therapy in the industry to use a dual route of administration. It delivers full-length frataxin protein in two ways:
- An intravenous (IV) infusion targets the cardiac manifestations of FA, with potential to treat other affected organ systems.
- An intradentate nuclei (IDN) infusion goes directly to the region of the cerebellum most affected by FA-associated neurologic decline.
Solid Biosciences says the design aims for precision delivery and optimal dosing to critical tissues. It also aims to address both the neurologic symptoms and the life-threatening cardiac effects of the disease, a combination families have consistently asked for.
FA families and research partners helped build the program
The program originated with FA212 LLC, a company founded by FA families and led by Tom Hamilton. The Friedreich's Ataxia Research Alliance (FARA) and the University of Pennsylvania also contributed to its development, according to Solid Biosciences. The company says feedback from individuals and families living with FA shaped the program.
Phase 1b trial will follow participants for five years
The Phase 1b study will evaluate the safety and tolerability of SGT-212 in non-ambulatory adults with FA, followed by ambulatory adults. Participants will be followed for five years.
Investor call and community webinar are on the calendar
Solid Biosciences will hold a live conference call on January 8, 2025, at 8:30 AM ET. The call is primarily for investors, but FA community members are encouraged to join. The company will also host a community webinar with FARA in the coming weeks, where participants can ask questions about SGT-212. Details have not yet been announced.