Nanoparticles deliver microRNAs to muscle stem cells for potential muscular dystrophy treatment
In an international collaboration, researchers have made an important breakthrough in the therapeutic delivery of microRNAs ...
Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy
Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with ...
FDA removes clinical hold on Entrada’s Duchenne therapy
Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy (DMD) therapy, ENTR-601-44, now ...
BioPharma Dive7h
Regeneron gene therapy helps deaf children hear in small study
Ten of 11 children born with a rare form of congenital hearing loss experienced improvements after receiving the company’s ...
Nanowerk7h
Using nanoparticles to transport microRNAs against Muscular Dystrophy to muscle stem cells
The scientists demonstrate that it is possible to specifically deliver the treatment to the muscle stem cells, avoiding ...
Muscular Dystrophy News8h
ENTR-601-44 trial for DMD adults amenable to exon 44 skipping OK’d
The FDA has lifted its hold and has given Entrada Therapeutics permission to launch a Phase 1b clinical trial to test ...
GlobalData on MSN8h
Success for YolTech’s hyperoxaluria in vivo gene therapy in early-stage trial
YolTech's gene therapy is intended as a one-time life-long cure for patients living with PH1, which can lead to kidney ...
FierceBiotech1d
Entrada's 2-year ordeal ends as FDA lifts hold on DMD drug
Entrada Therapeutics is finally able to get its Duchenne muscular dystrophy candidate (DMD) back on track after the FDA ...
FDA Clears Entrada Therapeutics' Early-Stage For Potential Treatment Of Duchenne Muscular Dystrophy
Entrada Therapeutics gains FDA clearance for its Phase 1b ELEVATE-44-102 study, evaluating ENTR-601-44 in adult Duchenne ...
Entrada Therapeutics, Inc.: Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with Duchenne muscular dystrophy - - Follows recently received ...
pharmaphorum1d
Pfizer drops Beqvez, leaving its gene therapy cupboard bare
Beqvez was Pfizer's first gene therapy, stemming from an alliance with Spark Therapeutics (now part of Roche) more than a ...
Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Receives FDA authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of ENTR-601-44 in adults living with ...