SBT101 / Spur Therapeutics - LARVOL DELTA

PROPEL: A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) (clinicaltrials.gov) - P1/2 | N=8 | Terminated | Sponsor: SwanBio Therapeutics, Inc. | N=16 ➔ 8 | Trial completion date: Mar 2029 ➔ Aug 2025 | Active, not recruiting ➔ Terminated | Trial primary completion date: Mar 2026 ➔ Jun 2025; The SBT101 program for AMN was terminated for business/strategic reasons, and there were no safety concerns

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Gene Therapies • Pain

PROPEL: A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) (clinicaltrials.gov) - P1/2 | N=16 | Active, not recruiting | Sponsor: SwanBio Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene Therapies • Pain

Spur Therapeutics Announces Oral and Poster Presentations Highlighting Data from Clinical and Preclinical Programs at Upcoming ASGCT Annual Meeting (GlobeNewswire) - "Spur Therapeutics...announced that it will share new data from its gene therapy programs in Gaucher disease, GBA1 Parkinson’s disease and adrenomyeloneuropathy in oral and poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting....The oral presentation will showcase clinical and preclinical durability data on FLT201, Spur’s adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, which is expected to begin a Phase 3 trial this year."

Clinical data • New P3 trial • Preclinical • Gaucher Disease • Parkinson's Disease

An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs. (PubMed, Mol Ther Methods Clin Dev) - "SBT101 was well tolerated, with no observed SBT101-related mortality or clinical signs. These findings not only provide preclinical efficacy data for SBT101 but also inform clinically relevant SBT101 dose selection for patients with adrenomyeloneuropathy."

Gene therapy • Journal • Preclinical • CNS Disorders • Gene Therapies • Pain

Safety evaluation of a novel gene therapy candidate for adrenomyeloneuropathy (SBT101) in nonhuman primates (ESGCT 2023) - "These findings had resolved by month 12. Overall, SBT101 was well tolerated over 12 months with persistent vector genome distribution, and transgene expression in healthy NHPs, supporting its investigation in the phase 1/2 trial."

Clinical • Gene therapy • Gene Therapies • Pain

Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy (ESGCT 2022) - No abstract available

Clinical • Pain

PROPEL: A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) (clinicaltrials.gov) - P1/2 | N=16 | Recruiting | Sponsor: SwanBio Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene Therapies • Pain