Muscular dystrophy

A new licensing agreement between Vita Therapeutics and MilliporeSigma (the US and Canada Life Science business of Merck) will see Vita leverage MilliporeSigma’s foundational CRISPR patents to advance its preclinical asset VTA-100 for limb-girdle muscular dystrophy (LGMD2A). VTA-100 utilises this CRISPR genome editing technology to insert a functional copy of the Calpain 3 (CAPN3) gene, […]

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. It is approved in ambulatory paediatric patients aged […]

The US Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) have voted eight to six in favour of a new gene therapy for Duchenne muscular dystrophy. The vote supports the accelerated approval of Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a […]

The National Institute for Health and Care Excellence (NICE) has published its Final Guidance recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales.   Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged two years and older who can walk.  The final […]

The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.  RPDs are designed to encourage the development of new drugs with high unmet medical needs, including rare diseases in children.  The […]

Myosana Therapeutics has completed a seed funding round totalling over $5 million led by investor John Ballantyne.  The capital will go towards developing Myosana’s gene therapy platform for neuromuscular and cardiac disease, with the goal of identifying the first development candidate for Duchenne muscular dystrophy by 2025.  “Built on decades of expertise in neuromuscular diseases, […]

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School.  Animal data presented in 2022 showed that the gene therapy outperformed the benchmark therapy along multiple key endpoints in a mouse model of […]

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy.  Fukuyama congenital muscular dystrophy (FCMD) is the second most common form of childhood muscular dystrophy in Japan. The disease manifests itself in the form of a severe neuromuscular disorder.  One form of FCMD is caused […]

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy.   The therapy, which is being developed in partnership with Roche, has been granted Priority Review by the FDA, with a regulatory action date […]