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SGT-212 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (Non-Ambulatory) | EXPERIMENTAL | Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion. |
| Cohort 2 (Ambulatory) | EXPERIMENTAL | Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion. |
| Cohort 3 (Ambulatory and Non-Ambulatory) | EXPERIMENTAL | Participants will receive bilateral IDN followed by systemic IV infusion. |
| Name | Type | Description |
|---|---|---|
| SGT-212 | DRUG | Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA) |
Inclusion Criteria: * Has history of FA symptom onset ≤25 years of age * Has a clinical and genetic diagnosis of FA * Has a staging score of ≥1 but \<6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score * Is willing to agree to the following rules for use of omavelox...
SGT-212 is an investigational gene therapy being developed for the treatment of Friedreich's Ataxia (FA), a rare inherited neurological disorder. It is designed to address the underlying genetic cause of the condition. The drug is currently in Phase 1 clinical development and has not yet been approved by regulatory authorities.
SGT-212 targets the FXN gene, which encodes the protein frataxin. In Friedreich's Ataxia, mutations in the FXN gene lead to reduced frataxin levels, causing progressive damage to the nervous system. SGT-212 is intended to deliver a functional copy of the FXN gene to restore frataxin production.
SGT-212 is being developed by Solid Biosciences Inc., a biopharmaceutical company focused on genetic medicines. The company's stock is traded under the ticker symbol SLDB. Solid Biosciences is conducting clinical trials to evaluate the safety and efficacy of SGT-212 in patients with Friedreich's Ataxia.
SGT-212 is currently in Phase 1 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing Phase 1 trial is actively recruiting participants to assess the safety, tolerability, and preliminary efficacy of SGT-212 in adults with Friedreich's Ataxia.
SGT-212 is being evaluated in a Phase 1 clinical trial registered as NCT07180355, titled 'A Study of SGT-212 Gene Therapy in Friedreich's Ataxia'. This trial is recruiting participants in the United States and aims to enroll approximately 10 adults aged 18 years and older with Friedreich's Ataxia.
SGT-212 has received several FDA designations, including Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug designation. These designations are intended to expedite the development and review of therapies for serious or rare conditions, potentially providing benefits such as priority review and market exclusivity upon approval.