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 ...
Pharmabiz1h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
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Top Biotech Neurogene Crashes 44%, Wiping Out Its Sprint On A $200 Million Deal
Biotech stock Neurogene collapsed Tuesday after a patient experienced a serious side effect in the company's Rett syndrome ...
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Ascend continues to climb with deal that elevates the CDMO’s US footprint
Ascend Advanced Therapies, which has been on an expansion tear since debuting three years ago, inked a deal with EW ...
Managed Healthcare Executive1d
FDA Approves Gene Therapy for Debilitating Enzyme Deficiency
Developed by PTC Therapeutics, Kebilidi is an adeno-associated virus vector-based gene therapy indicated for the treatment of ...
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StitchR technology delivers large genes for muscular dystrophy treatment
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.
Transforming Viral Vector Analytics: Matica Bio’s Innovative Approach to Capsid Analysis
Matica Biotechnology (Matica Bio), a leading contract development and manufacturing organization (CDMO) for viral vectors, ...
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IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA
The multicenter Phase1b open-label dose escalation study (NCT05973630) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose ...
Stockhouse2d
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
KEBILIDI is authorized to be administered using the SmartFlow Neuro Cannula. KEBILIDI is an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...