SGT-003
Gene Therapy Target: dystrophin, microdystrophin
Solid Biosciences Inc.
Most recent annual filing: 2026-03-15
Pipeline (6 active programs)
SGT-212
Gene Therapy Target: frataxin, FXN
SGT-501
Gene Therapy Target: CASQ2, RYR2
SGT-601
Gene Therapy Target: TNNT2, cardiac troponin T
SGT-401
Gene Therapy Target: BAG3
SGT-701
Gene Therapy Target: RBM20
Technology platforms
POLARIS-101
Rationally designed next-generation AAV capsid (AAV-SLB101) engineered for systemic delivery with improved transduction in the presence of neutralizing antibodies; applications in neuromuscular and cardiac gene therapy including Duchenne Muscular Dystrophy, dilated cardiomyopathy, and FSHD
Targets: integrin receptors
Novel Capsid and Promoter Libraries
Scalable high-throughput platform for next-generation capsid design targeting cardiac and neuromuscular diseases; leverages deep capsid structure knowledge, peptide insertions, generative AI, and affinity maturation to improve biodistribution and expression. Capsid selection from first library anticipated 2H 2026.
Mayo Clinic Cardiac Gene Therapy Collaboration · Mayo Foundation for Medical Education and Research
Collaboration platform with Mayo Foundation for Medical Education and Research covering six undisclosed preclinical cardiac gene therapy programs; Mayo conducts IND-enabling studies, Solid retains rights to select programs for clinical development and exclusive commercialization.
Biotech Distilled coverage (3)
- 2026-05-12 UPDATE: Solid Biosciences (SLDB) - The Dilution Gap Closes, the Phase 3 Clock Starts 🔒 Paid
- 2026-03-22 UPDATE: Solid Biosciences (SLDB) - The S-3 Dilution Playbook, a New 13D, and the Burn Rate Reality 🔒 Paid
- 2026-03-12 Solid Biosciences (SLDB) - Scientific Deep Dive for SGT-003 and Pipeline Products 🔒 Paid
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