Dyne Therapeutics

Dyne Therapeutics' DYNE-302 demonstrated functional improvement in a severe FSHD mouse model, with a single intravenous dose restoring treadmill running ability at peak muscle weakness.

Health Secretary Robert F. Kennedy Jr. announced plans to fast-track FDA approvals for rare disease treatments and remove regulatory obstacles during a meeting focused on cell and gene therapies.

• Dyne Therapeutics has appointed Vikram Ranade, PhD, as Chief Business Officer and Ranjan Batra, PhD, as Chief Scientific Officer, bolstering its executive team ahead of potential product launches in 2027. • Dr. Batra, an RNA biology expert with experience developing therapies for rare neuromuscular disorders, will lead research strategy and pipeline development for Dyne's FORCE™ platform technology. • Current CSO Oxana Beskrovnaya, PhD, will transition to Chief Innovation Officer, focusing on maximizing the potential of Dyne's TfR1 delivery platform for new therapeutic applications in neuromuscular diseases.

Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD).

• Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose. • The therapy, designed for patients amenable to exon 51 skipping, showed improvements across multiple functional endpoints including mobility assessments, positioning Dyne for potential accelerated FDA approval submission in early 2026. • If approved, DYNE-251 could generate significant market impact, with GlobalData projecting revenue growth from $5 million in 2025 to $129 million by 2030 in the competitive DMD treatment landscape.

Dyne Therapeutics' DYNE-101, an oligonucleotide therapy for myotonic dystrophy type 1 (DM1), may qualify for accelerated FDA approval based on promising surrogate biomarker data.

• Dyne Therapeutics strengthens its financial position with $783M in pro forma cash through ATM share issuance, ensuring operational funding into second half of 2026. • Company maintains momentum in DMD treatment development, targeting completion of DELIVER cohort enrollment for DYNE-251 therapy in Q1 2024. • Piper Sandler projects potential accelerated approval for DYNE-251 by late 2026, adjusting price target to $48 while maintaining optimistic outlook.

• Dyne Therapeutics' DYNE-101 has been granted Fast Track Designation by the FDA, potentially accelerating its development for myotonic dystrophy type 1 (DM1). • Clinical data for DYNE-101 demonstrates robust splicing correction and broad functional improvements in patients with DM1, including CNS benefits. • Oppenheimer maintains an Outperform rating with a $60 price target, citing consistent data correlating splicing correction to functional improvements. • Dyne plans to initiate registrational cohorts in 2025, aiming for accelerated approval in the first half of 2026, with splicing as the primary endpoint.

Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1).

• The myotonic dystrophy therapeutic landscape is experiencing a surge in R&D, with over 20 companies actively involved in developing 22+ novel therapies. • Key players like Avidity Biosciences, Dyne Therapeutics, and Vertex Pharmaceuticals are advancing promising drugs such as AOC 1001, DYNE-101, and VX-670 through clinical trials. • Regulatory designations, including Breakthrough Therapy and Fast Track, have been granted to several pipeline drugs, expediting their development for myotonic dystrophy. • Recent clinical trial initiations and data presentations highlight the progress in addressing unmet needs in myotonic dystrophy treatment, fostering hope for improved patient outcomes.