EXG001-307 / Exegenesis Bio - LARVOL DELTA

Next-Generation AAV-Based Gene Therapy for Spinal Muscular Atrophy: Safety and Efficacy of EXG001-307 in Clinical Trials (ASGCT 2025) - "Two patients in High-dose Cohort experienced thrombotic microangiopathy (TMA), which resolved following treatment with eculizumab (anti-C5 antibody)...This novel therapy offers a promising, targeted approach to SMA treatment, advancing the standard of care for patients with this devastating condition. Disease Focus of Abstract:Neuromuscular Disorders"

Clinical • Gene therapy • Gene Therapies • Genetic Disorders • Hepatology • Infectious Disease • Liver Failure • Movement Disorders • Muscular Atrophy • Novel Coronavirus Disease • Rare Diseases

Nonclinical Evaluation of AAV Gene Therapy EXG001-307 for Spinal Muscular Atrophy (ASGCT 2025) - "Regulatory communication with the FDA has been initiated, and IND submission is targeted for June 2025. Disease Focus of Abstract:Neuromuscular Disorders"

Gene therapy • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy (clinicaltrials.gov) - P1 | N=4 | Enrolling by invitation | Sponsor: Hangzhou Jiayin Biotech Ltd

New P1 trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA (clinicaltrials.gov) - P=N/A | N=18 | Enrolling by invitation | Sponsor: Hangzhou Jiayin Biotech Ltd | Not yet recruiting ➔ Enrolling by invitation | Initiation date: Sep 2024 ➔ Mar 2025

Enrollment open • Trial initiation date • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA (clinicaltrials.gov) - P=N/A | N=18 | Not yet recruiting | Sponsor: Hangzhou Jiayin Biotech Ltd

New trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1 (clinicaltrials.gov) - P1/2 | N=12 | Enrolling by invitation | Sponsor: Hangzhou Jiayin Biotech Ltd | Recruiting ➔ Enrolling by invitation | Trial completion date: Mar 2025 ➔ Aug 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025

Enrollment status • Trial completion date • Trial primary completion date • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • SMN1

Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I (clinicaltrials.gov) - P1 | N=2 | Completed | Sponsor: Hangzhou Jiayin Biotech Ltd

New P1 trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-037, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head Control and Sitting Without External Assistance (Businesswire) - P1/2 | N=12 | NCT05614531 | Sponsor: Hangzhou Jiayin Biotech Ltd | "Exegenesis Bio...is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 Phase 1/2 clinical trial in Spinal Muscular Atrophy (SMA) Type I at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland on May 8, 2024 (poster #627)...Data Highlights: EXG001-307 has a unique engineered AAV design that includes a novel pro-NS promoter, which is believed to contribute to improved efficacy and safety in SMA Type 1...Patients in the EXG001-307 low-dose group (1.1 E 14 vg/kg) achieved head control 3 to 6 months after dosing and sitting without external assistance 11 months following dosing...'This is leading us to accelerate development in the US. We expect to file our US IND for SMA Type 1 in 4Q 2024.'....'We look forward to sharing further details during our poster session at ASGCT in Baltimore on May 8, 2024.'"

IND • P1/2 data • CNS Disorders • Muscular Atrophy

Clinical Safety and Efficacy of EXG001-307 in SMA Type 1 Patients: A Next-Generation AAV-Based Gene Therapy (ASGCT 2024) - "Nonclinical studies have demonstrated that EXG001-307 improved the survival rate, further enhanced the motor function, and significantly reduced toxicity in the SMA mouse model.In a Phase I clinical study, six patients received 1.1x1014 vg/kg or 1.5x1014 vg/kg doses of EXG001-307 and showed good tolerability with no dose-limiting toxicity or > grade 2 serious adverse events (SAEs). All patients achieved significant motor milestones, suggesting EXG001-307 as a next-generation SMA treatment for improved patient outcomes."

Clinical • Gene therapy • Cardiovascular • CNS Disorders • Gene Therapies • Genetic Disorders • Hepatology • Liver Failure • Movement Disorders • Muscular Atrophy • Rare Diseases • SMA4 • SMN1

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type 1 (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Hangzhou Jiayin Biotech Ltd

New P1/2 trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases