Breakthrough Approval for Kebilidi The FDA has granted a landmark approval to PTC Therapeutics’ Kebilidi, making it the first gene therapy in the U.S. to be administered directly into the brain. This approval represents a major step forward in gene therapy,...
– First-ever FDA approval for gene therapy directly administered to the brain – – Priority review voucher granted – – Broad label including children and adults – – PTC pioneers new approach for CNS drug delivery – ...
SHANGHAI and CLINTON (NJ), November 4, 2024 – HuidaGene Therapeutics, a global clinical-stage biotechnology company focused on genome medicines, has achieved a groundbreaking milestone. The US FDA has cleared its investigational new drug (IND) application for HG202,...
SAN DIEGO, Oct. 3, 2024 /PRNewswire/ — Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug...
DURHAM, N.C., Sept. 25, 2024 /PRNewswire/ — EydisBio, Inc. is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EYD-001, its highly selective and potent, orally bioavailable TAK1 inhibitor for the...
