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.
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain. This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
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,
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FDA Approves Gene Therapy for Debilitating Enzyme Deficiency
Kebilidi is a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic ...
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Cracking the Code: Enzyme from Cow's Gut Targets Biofilm Barriers
This innovative enzyme therapy holds promise for integration into wound dressings and medical device coatings, offering hope for improved outcomes in patients with chronic infections.
Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
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Breaking down bacterial barriers using bovine gut enzyme
“[This] will be a combinatorial therapy – we can use the enzyme plus the antibiotic for treating K. pneumoniae ...
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 ...
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Spanish gene editing biotech licenses DNA-cutting enzymes from Caszyme for up to $43M
Spanish biotech Integra Therapeutics has upped its arsenal of gene editing tools by licensing Caszyme’s Cas12I nucleases, the ...
SEKISUI Completes £15.7 Million Expansion In Cgmp Biopharma CDMO Capacity
Growth of UK facility will enable clinical-grade manufacturing of drug substances for range of biopharmaceutical products ...