BBP-812 / BridgeBio - LARVOL DELTA

CANaspire gene therapy trial for Canavan Disease: imaging and motor outcome data from low-dose and high-dose cohorts (ASGCT 2026) - P1/2 | "Three severe adverse events (SAEs) of thrombotic microangiopathy, each managed with eculizumab, occurred, and there was one death from acute gastroenteritis leading to hypernatremia and cardiac arrest, deemed unrelated to BBP-812. These changes have occurred faster in participants having received high dose compared to those who received low dose BBP-812. Of the high dose participants followed >26 weeks post dosing, 6 of 8 (75%) showed improved HINE-2 head control and 7 of 8 (88%) showed improved HINE-2 sitting ability within 15 months, motor skills deemed meaningful by caregivers and prioritized as motor function efficacy endpoints."

First-in-human • Gene therapy • Cardiovascular • CNS Disorders • Gastroenterology • Gastrointestinal Disorder • Gene Therapies • Hematological Disorders • Thrombocytopenia

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Aspa Therapeutics | Trial completion date: Oct 2030 ➔ Oct 2032

Trial completion date • Gene Therapies

Results from the CANaspire Gene Therapy Trial for Canavan Disease: Safety, Biomarker, Imaging, and Clinical Outcome Data from the Completed Low-dose Cohort (ESGCT 2024) - P, P1/2 | "The low-dose cohort has been completed and participants entering the study are currently receiving BBP-812 at a dose level of 2.6E14 vg/kg. Preliminary data from the higher-dose group will be included in this presentation as available."

Biomarker • Clinical • Clinical data • Gene therapy • CNS Disorders • Gene Therapies • Hematological Disorders • Pediatrics • Thrombocytopenia

CANaspire: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Aspa Therapeutics | N=18 ➔ 26 | Trial completion date: Mar 2028 ➔ Oct 2030 | Trial primary completion date: Oct 2024 ➔ Oct 2026

Enrollment change • Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies

Preliminary Results from CANaspire, a First-in-Human Phase 1/2 Controlled Open-Label Study of BBP-812, a Recombinant AAV9-hASPA Vector for the Treatment of Canavan Disease (EPNS 2023) - P1/2 | "CANaspire is the first clinical trial of a systemically administered gene therapy candidate for CD. Preliminary data from 4 participants have supported the overall tolerability of this approach and have demonstrated reduced NAA levels in urine, CSF and brain along with initial signs of clinical efficacy after BBP-812 treatment."

Clinical • P1/2 data • Gene Therapies • Pediatrics

Initial Biomarker and Clinical Findings from the CANaspire Canavan Disease Gene Therapy Trial: Exploration of Connections between NAA and Disease Severity (ASGCT 2023) - P=N/A, P1/2 | "Preliminary data have supported the overall tolerability of BBP-812 and demonstrated robust and durable post-dose NAA reductions in urine, CSF and brain along with initial suggestions of clinical stabilization. While the literature and the ongoing natural history study suggest an association between NAA levels and phenotype, whether these early signs of PD and clinical activity translate to clinical efficacy awaits confirmation with additional participant data and longer follow-up in CANaspire."

Biomarker • Clinical • Gene therapy • CNS Disorders • Gene Therapies • Pediatrics

CANaspire: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Aspa Therapeutics | Trial completion date: Oct 2028 ➔ Mar 2028

Gene therapy • Trial completion date • Gene Therapies

CANaspire: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (clinicaltrials.gov) - P1/2; N=18; Not yet recruiting; Sponsor: Aspa Therapeutics

New P1/2 trial • CNS Disorders • Developmental Disorders • Gene Therapies • Genetic Disorders • Metabolic Disorders • Psychiatry

CANaspire: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Aspa Therapeutics; Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Developmental Disorders • Gene Therapies • Genetic Disorders • Metabolic Disorders • Psychiatry