The competitive landscape for Friedreich's ataxia therapies is heating up, with companies like Voyager Therapeutics also developing gene therapies and expected to file INDs in 2025.

SGT-212 utilizes a novel delivery method that employs MRI-guided convection-enhanced delivery, allowing for precise targeting of the dentate nucleus in the cerebellum.

This innovative approach combines SGT-212 with gadolinium for MRI imaging, enabling real-time verification of drug delivery to the intended site in the brain.

Bo Cumbo, who previously worked at Sarepta Therapeutics, has taken the helm as CEO of AavantiBio, with a focus on developing gene therapies for Friedreich's ataxia.

Cumbo's initial efforts at AavantiBio faced challenges during preclinical trials, where gene therapies struggled to effectively reach both target organs without causing toxicity.

The first-in-human Phase Ib clinical trial for SGT-212 is anticipated to begin in the latter half of 2025, with plans to enroll patients over a five-year period.

The COVID-19 pandemic prompted AavantiBio to merge with Solid Biosciences, which is also focused on Duchenne muscular dystrophy (DMD) and has faced competition from Sarepta.

Following the merger in December 2022, Cumbo restructured Solid's management team and advanced their lead DMD program, SGT-003, which utilizes an engineered microdystrophin gene.

Friedreich's ataxia is a serious condition characterized by neurodegeneration and a significant mortality risk due to heart failure linked to cardiomyopathy.

Solid Biosciences has received FDA approval for its Investigational New Drug (IND) application for SGT-212, a gene therapy aimed at addressing both neurological and systemic manifestations of Friedreich's ataxia.

The SGT-212 program has reportedly overcome previous delivery challenges, effectively targeting both the brain and heart in animal models.