First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have decreased AADC activity that causes severe disability, including gross motor function deficits,
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The U.S. Food and Drug Administration approved PTC Therapeutics' gene therapy to treat a potential fatal enzyme deficiency disorder, the company said on Wednesday, sending its shares up about 2% in aftermarket trade.
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy has already been approved to treat AADC deficiency in Europe and the U.K., among other countries,
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 voucher for PTC.
FDA Approves Gene Therapy to Treat AADC Deficiency
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
William Blair analyst Sami Corwin has maintained their bullish stance on PTCT stock, giving a Buy rating on November 7. Sami Corwin has given
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of children and adults with AADC deficiency, a rare genetic disorder that impairs the ability to generate dopamine,
JP Morgan Downgrades Gene Therapy Focused Bluebird Bio, Cites Limited Funding Options
On Thursday, Bluebird Bio Inc. (NASDAQ:BLUE) reported a third-quarter EPS loss of 31 cents, compared to a loss of 80 cents a ...
Harvard Medical School10h
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 Daily21h
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.
STAT1d
Neurogene and the race to the bottom of gene therapy
At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.
FierceBiotech1d
Muscular dystrophy gene therapy uses cells to stitch together mRNA strands in mice
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
GEN18h
Gene Therapy for Muscular Dystrophy Could Use StitchR to Assemble XL Gene from S Segments
StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.
dermatologyadvisor6h
Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Under Review
The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.