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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 currently in Phase 1 clinical development and has not been approved by the FDA.
SGT-212 is being developed by Solid Biosciences Inc. (NASDAQ: SLDB), a biopharmaceutical company focused on neuromuscular and neurological diseases.
SGT-212 is currently in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The drug has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations by the FDA.
SGT-212 is being evaluated in a Phase 1 clinical trial (NCT07180355), titled 'A Study of SGT-212 Gene Therapy in Friedreich's Ataxia'. The trial is recruiting participants in the United States and aims to enroll 10 adults aged 18 years and older.
Yes, SGT-212 is a gene therapy being developed by Solid Biosciences Inc. for the treatment of Friedreich's Ataxia. It is currently in a Phase 1 clinical trial (NCT07180355) that is recruiting patients in the United States.