INS1201 / Insmed - LARVOL DELTA

Design of the Phase 1 ASCEND Study to Investigate the Safety and Biodistribution of INS1201 Gene Therapy in Males With Duchenne Muscular Dystrophy (ASGCT 2025) - "The ASCEND study will assess the safety and biodistribution of INS1201 to inform RP2D selection. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Clinical • Gene therapy • P1 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

CSF Delivery of INS1201 AAV9-Micro-Dystrophin Demonstrates Long-Term Durability of Efficacy in a Mouse Model of DMD (ASGCT 2025) - "These findings support ongoing studies to evaluate the potential of INS1201 as a therapeutic option for DMD. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Immunology • EP400

Recent Progress and Anticipated Milestones by Program (PRNewswire) - "Insmed completed the Phase 2b study of treprostinil palmitil inhalation powder (TPIP) in pulmonary arterial hypertension (PAH), with topline data anticipated in June of 2025; The Company plans to initiate a Phase 3 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) in the second half of 2025...Insmed initiated the Phase 1 ASCEND clinical study of INS1201, an intrathecally-delivered gene therapy for patients with Duchenne muscular dystrophy (DMD), in the first quarter of 2025, and expects to dose the first patient in the second quarter of 2025; The Company's next two gene therapy candidates, which target amyotrophic lateral sclerosis (ALS) and Stargardt disease, are currently advancing toward the clinic."

New P3 trial • P2b data • Pipeline update • Trial status • Amyotrophic Lateral Sclerosis • Duchenne Muscular Dystrophy • Genetic Disorders • Interstitial Lung Disease • Ophthalmology • Pulmonary Arterial Hypertension

ASCEND: A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Insmed Gene Therapy LLC | Not yet recruiting ➔ Recruiting | Initiation date: Feb 2025 ➔ May 2025

Enrollment open • Trial initiation date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update (PRNewswire) - "Insmed plans to initiate a clinical study of INS1201 in the first half of 2025."

P1 data • Duchenne Muscular Dystrophy