FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
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 ...
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FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
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
EVRY, France, November 12, 2024--"This IND clearance in an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease" ...
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Neurogene Halts Testing High-Dose Rett Syndrome Gene Therapy After Adverse Event
Following this update, the company's stock price dropped more than 30 percent from market close Friday to $23.79 per share Monday morning.
4DMT Reports Third Quarter 2024 Financial Results, Operational Highlights and Expected Upcoming Milestones
Presented positive interim data for 4D-150 in wet age-related macular degeneration (wet AMD) from PRISM Phase 1/2 clinical trial highlighting ...
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Using FT-NIR and Machine Learning for Identifying Authenticate Kimchi
By using the near-infrared region of the electromagnetic spectrum, this method can reveal a sample's chemical composition ...
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...
4D Molecular Therapeutics, Inc.: 4DMT Reports Third Quarter 2024 Financial Results, Operational Highlights and Expected Upcoming Milestones
Presented positive interim data for 4D-150 in wet age-related macular degeneration (wet AMD) from PRISM Phase 1/2 clinical trial highlighting robust and durable clinical activity across diverse patien ...
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PTC Therapeutics' AADC Deficiency Gene Therapy Kebilidi Gains FDA Accelerated Approval
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.