Solid Biosciences ((SLDB)) announced an update on their ongoing clinical study.
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Solid Biosciences is conducting a study titled ‘A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich’s Ataxia (FA)’. The study aims to assess the safety and tolerability of the gene therapy SGT-212 in individuals with Friedreich’s Ataxia, a rare genetic disorder. This research is significant as it represents a first-in-human trial for this treatment approach.
The intervention being tested is SGT-212, a gene therapy delivered using adeno-associated virus serotype AAVhu68. Its purpose is to introduce a codon-optimized complementary DNA (cDNA) to potentially treat Friedreich’s Ataxia.
The study is designed as an interventional trial with a non-randomized, parallel assignment. It is open-label, meaning no masking is involved, and its primary purpose is treatment-focused. Participants will receive the treatment through both IDN and IV methods.
The study is not yet recruiting, with a start date set for August 22, 2025. The last update was submitted on October 17, 2025. These dates are crucial for tracking the study’s progress and ensuring timely updates for stakeholders.
This update may influence Solid Biosciences’ stock performance as it progresses towards a potential treatment for Friedreich’s Ataxia. Investors may view this as a positive development, given the unmet need in this area. Competitors in the gene therapy space may also be monitoring these developments closely.
The study is ongoing, with further details available on the ClinicalTrials portal.