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.
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.
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,
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) 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,
Scientists use cow gut enzyme to disrupt bacterial biofilms
Most of the disease-causing bacteria secrete matrix-like layers around themselves called biofilms. These biofilms act as ...
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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.
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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 ...
Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...
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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 ...
New Target for Alzheimer’s: Researchers Discover Enzyme Behind Tau Protein Build-Up
Researchers found that inhibiting the enzyme TYK2 may reduce the harmful accumulation of tau protein in Alzheimer’s disease, ...
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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 ...