US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to treat a potential fatal enzyme deficiency disorder, the company said on Wednesday, sending its shares up about 2% in aftermarket trade.
Child Thriving After Becoming World's Youngest to Receive Gene Therapy for AADC Deficiency
Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene in the Brain
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,
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 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 and pediatric patients with aromatic L-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,
Alternative experimental gene therapy restores hearing, boosts vision in tests
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
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 ...
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.
Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
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.
JD Supra16h
Patent Vs. Trade Secret Considerations For Cell And Gene Therapies
Understanding what to patent versus what to keep as a trade secret is an increasingly important and challenging decision ...
Gene therapy protects against motor neuron disease in rats
University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...
dermatologyadvisor14h
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.
Harvard Medical School17h
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 ...