Dyne Therapeutics

Dyne Therapeutics Strengthens Leadership Team with Key Scientific and Business Appointments

a month ago

• 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 Secures $68 Million to Advance First Epigenetic Therapy for FSHD

2 months ago

• 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). • New Zealand's Medsafe has approved Epicrispr's clinical trial application, with the first-in-human study of EPI-321 expected to begin in 2025 to evaluate its safety and biological activity in adults with FSHD. • EPI-321 uses CRISPR technology to silence the DUX4 gene that drives FSHD progression and has received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations.

Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results

2 months ago

• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production. • The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment. • If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.

Dyne's DYNE-251 Shows "Unprecedented" Functional Improvements in Duchenne Muscular Dystrophy Trial

2 months ago

• 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 Advances DMD Treatment Program Despite Price Target Adjustment

3 months ago

• 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 Receives FDA Fast Track Designation for Myotonic Dystrophy Type 1

4 months ago

• 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 Shows Promise in DM1 Trial, FDA Grants Fast Track Designation

4 months ago

• Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1). • The FDA grants Fast Track designation to DYNE-101, expediting its development and regulatory review process. • Dyne plans to initiate a global Registrational Expansion Cohort, aiming for U.S. Accelerated Approval submission in H1 2026. • DYNE-251 for Duchenne muscular dystrophy (DMD) is also advancing, with potential regulatory submissions expected in early 2026.

Bayer's Elinzanetant Shows Promise in Reducing Hot Flashes for Breast Cancer Patients

4 months ago

• Elinzanetant met primary endpoints in the Phase III OASIS 4 trial, significantly reducing the frequency of moderate to severe vasomotor symptoms (VMS) in women undergoing endocrine therapy for breast cancer. • The study also achieved secondary endpoints, demonstrating a reduction in the severity of VMS, improvements in sleep disturbances, and enhanced menopause-related quality of life compared to placebo. • Elinzanetant is a dual neurokinin-1 and 3 receptor antagonist, representing a potential non-hormonal treatment option for VMS in breast cancer patients and women at high risk. • Bayer is advancing regulatory submissions for elinzanetant in the US, EU, and other global markets, with potential launch expected later this year, pending regulatory approvals.

Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001

5 months ago

• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients. • The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results. • SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients. • If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.

Elevidys Gene Therapy Shows Sustained Benefits in Duchenne Muscular Dystrophy Patients

5 months ago

• Sarepta Therapeutics' Elevidys demonstrates sustained benefits and disease stabilization in ambulatory Duchenne muscular dystrophy (DMD) patients, according to Phase 3 EMBARK trial results. • Crossover-treated patients showed a 2.34-point improvement on the North Star Ambulatory Assessment (NSAA) compared to matched external controls after 52 weeks of Elevidys treatment. • Patients treated with Elevidys in Part 1 of EMBARK maintained clinically meaningful improvements in NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) at two years. • Muscle biopsies showed consistent micro-dystrophin expression, and MRI scans indicated minimal muscle pathology progression, reinforcing Elevidys's long-term efficacy and safety.