SNUG-01 / SineuGene - LARVOL DELTA

A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS (clinicaltrials.gov) - P1/2 | N=21 | Not yet recruiting | Sponsor: SineuGene Therapeutics Co., Ltd.

New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Novel Gene Therapy for ALS cleared for global trials in China and the U.S. (The Manila Times) - " This global Phase I/IIa trial will evaluate the safety and potential efficacy of SNUG01 and identify dosage for further clinical development in adults with ALS."

New P1/2 trial • Amyotrophic Lateral Sclerosis

SineuGene's SNUG01 Receives FDA Orphan Drug Designation for ALS (PRNewswire) - "SineuGene Therapeutics... announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its gene therapy, SNUG01, for the treatment of amyotrophic lateral sclerosis (ALS). SNUG01 is a first-in-class gene therapy targeting TRIM72 (Tripartite Motif Containing 72), designed to provide broad neuroprotection in ALS....SineuGene plans to initiate a Phase I/IIa international, multicenter trial to evaluate the therapy's safety, tolerability, and preliminary efficacy in adults with ALS. The study will enroll participants at clinical sites across both countries, including Massachusetts General Hospital in the U.S., which will join other sites in China."

New P1/2 trial • Orphan drug • Amyotrophic Lateral Sclerosis

Administration of a single intrathecal injection of SNUG01 in patients with amyotrophic lateral sclerosis (ChiCTR) - P=N/A | N=7 | Not yet recruiting | Sponsor: The Second Affiliated Hospital of Zhejiang University School of Medicine; The Second Affiliated Hospital of Zhejiang University School of Medicine

New trial • Amyotrophic Lateral Sclerosis • CNS Disorders

SineuGene Therapeutics Announces FDA IND Clearance for SNUG01, a First-in-Class TRIM72-Targeted Gene Therapy for ALS (PRNewswire) - "SineuGene Therapeutics Co., Ltd...announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SNUG01 — a first-in-class TRIM72 (Tripartite Motif Protein 72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). The clearance authorizes a global Phase I/IIa clinical trial designed to evaluate SNUG01's safety, tolerability, and preliminary efficacy in adults with ALS through a dose-escalation and expansion study."

IND • P1/2 data • Amyotrophic Lateral Sclerosis

A Multicenter, Single-Arm, Open-Label Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal of SNUG01 in Patients with Amyotrophic Lateral Sclerosis (ALS) (ChiCTR) - P=N/A | N=7 | Not yet recruiting | Sponsor: Peking University Third Hospital; Peking University Third Hospital

New trial • Amyotrophic Lateral Sclerosis • CNS Disorders