Elixirgen Therapeutics Inc. has entered into an option agreement with Nippon Shinyaku Co. Ltd. for the development and commercialization of EXG-7001 for Duchenne muscular dystrophy. EXG-7001 is a locally administered, full-length dystrophin mRNA therapeutic that is currently in preclinical development for the treatment of Duchenne muscular dystrophy.
Gen1e Lifesciences Inc. has obtained both orphan drug and rare pediatric disease designations from the U.S. FDA for GEn-1123 for the treatment of Duchenne muscular dystrophy (DMD).
Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten (EDG-5506), to Servier SA. The deal is potentially worth up to $2.65 billion when including a potential $1.1 billion in milestone payments.
AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload.
How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.
A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.
Italian family-owned Angelini Pharma SpA is making its first move into the U.S. market, acquiring rare diseases specialist Catalyst Pharma Inc. in an all-cash deal worth $4.1 billion. The acquisition gives Angelini ownership of three marketed drugs for treating epilepsy and neuromuscular diseases that had combined sales of $589 million in 2025, a 19.8% increase over 2024.
Roche Holding AG is making good on its promise to try and convince the EMA of the benefits of Elevidys (delandistrogene moxeparvovec), announcing a further global phase III trial of the Duchenne muscular dystrophy gene therapy.
Currently available disease management options for Duchenne muscular dystrophy (DMD) are mostly symptomatic. Several strategies based on exon-skipping or gene transfer have been proposed to restore dystrophin expression, but can only address specific subsets of DMD patients and/or provide limited clinical benefits. Upregulating utrophin (UTRN), a structural and functional paralogue of dystrophin, has been proposed as an alternative therapeutic approach that may be suitable for all DMD patients, regardless of their genetic defect.
Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis.
