BioPharma Dive19h
How a Biogen drug set the stage for a new biotech targeting ALS
Trace Neuroscience, which launched this week with $101 million, benefited from last year’s approval of Qalsody. Its CEO spoke ...
The American Journal of Managed Care22h
Delandistrogene Moxeparvovec Fails to Meet Primary Endpoint in Phase 3 Study
Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...
STAT22h
CAR-T promising for autoimmune disease while perhaps preserving fertility
Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...
Harvard Medical School23h
Second Gene Therapy Shows Promise for Syndrome Involving Blindness, Deafness
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Science Daily1d
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult ...
BioPharma Dive1d
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...