PTC, gene therapy and FDA

FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA's accelerated approval is based on the saf
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.
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 gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during a surgical procedure, and replaces the human dopa decarboxylase (DDC) gene that is mutated in the disease, allowing dopamine synthesis to take place.
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
Sami Corwin has given his Buy rating due to a combination of factors that favor PTC Therapeutics’ market position. The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company.
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 added hundreds of workers this year
Seaport-based PTC Inc. (Nasdaq: PTC), one of the state's largest public companies by revenue, added 270 workers in the 12 ...
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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 ...
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PTC Industries reports over 100% jump in Q2 PAT; shares up 125% in 1 year
PTC Industries is a smallcap company. It is a manufactures of high precision metal castings for a variety of industries. The ...
Nicer fall air moving in and tracking PTC #19
Meteorologist Devon Lucie tracks the last of the rain moving out tonight, then steps us through a big drop in temperatures ...
PTC forecasts quarterly revenue below estimates on tight tech spending
Industrial software maker PTC forecast first-quarter revenue below estimates on Wednesday, signaling continued weakness in ...
PTC Therapeutics’ AADC deficiency gene therapy granted FDA approval
PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever ...
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PTC India Q2 Results Live: Profit Rises by 19.77% YoY
PTC India Q2 Results Live : PTC India has declared its Q2 results on November 12, 2024, showcasing a mixed performance in ...
Multibagger Stock: PTC Industries surges over 275% in 2 years, achieves 3,200% growth in 4 years
PTC Industries stock surged 278% in two years, boosted by strong financials and significant orders, including one from Israel ...