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 Dive8h
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 ...
Nanowerk8h
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 News9h
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 MSN10h
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 ...
Pharma giant's pullback from gene therapy puts spotlight on North Carolina operations
One of the largest pharmaceutical companies in North Carolina is discontinuing a treatment produced in the state.
BioPharma Dive4d
Pfizer stops selling hemophilia gene therapy, citing weak demand
Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for ...
FiercePharma4d
Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio
Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. | ...
Massachusetts families, lawmakers fighting to keep NIH funding for life-saving medical research
Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.