Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Phase 1/2 Enrolling by invitation 12 enrolled Guangzhou Jiayin Biotech Ltd

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).

Timeline

Start: 2023-02-16
Primary completion: 2025-08
Completion: 2025-08

Publications

Song C, Wang Q, Zhu P, Li J, Dai L, Hu W, Yang W, Zhang R, Wang D, Li Z, Chen S, Wang L, Ye G, Wu Z. Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy. Mol Ther Methods Clin Dev. 2025 Apr 18;33(2):101475. doi: 10.1016/j.omtm.2025.101475. eCollection 2025 Jun 12.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	EXG001-307	Gene therapy (AAV / viral vector)	—	Intravenous

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Summary

Timeline

Publications

Drugs

Indications