Dyne Therapeutics Receives FDA Breakthrough Therapy Designation for DM1 Drug DYNE-101

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the best biotech stocks to invest in now. On June 17, Dyne Therapeutics announced that the FDA granted Breakthrough Therapy Designation to DYNE-101 for treating myotonic dystrophy type 1 (DM1). The designation expedites the development and review of drugs for serious conditions that show preliminary clinical evidence of improvement over existing therapies.

Benefits of this designation for DYNE-101 in the US include enhanced FDA support, early and frequent communication with reviewers, and eligibility for rolling and Priority Review, potentially reducing the Biologics License Application/BLA review timeline from 12 to 8 months. Following a Type C meeting with the FDA in May 2025 and an analysis of new long-term functional data, Dyne Therapeutics also unveiled an updated plan for obtaining US Accelerated Approval for DYNE-101 in DM1.

Dyne Therapeutics Receives FDA Breakthrough Therapy Designation for DM1 Drug DYNE-101

A lab technician working with chemicals and equipment to create a novel therapeutic drug.

Based on FDA feedback and new 6-month and 12-month efficacy data, Dyne submitted a revised protocol for the ongoing Registrational Expansion Cohort of the ACHIEVE trial in June 2025. Dyne expects to complete enrollment in the Registrational Expansion Cohort in Q4 2025. Data from this cohort are planned for mid-2026 to support a potential US Accelerated Approval submission in late 2026. Additionally, Dyne plans to initiate a confirmatory Phase 3 clinical trial in Q1 2026 and is pursuing expedited approval pathways for DYNE-101 globally.

Dyne Therapeutics Inc. (NASDAQ:DYN) is a clinical-stage neuromuscular disease company that discovers and develops therapeutics for neuromuscular diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.