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 Dive5h
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 ...
Nanowerk5h
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 News6h
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 MSN6h
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.
BioMarin's Narrow Moat Supported by Rare Disease Therapies and Developing Pipeline
BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...
Is Sarepta Therapeutics, Inc. (SRPT) The Best Fast Growth Stock To Buy Right Now?
We recently published a list of 15 Best Fast Growth Stocks To Buy Right Now. In this article, we are going to take a look at ...