Ultragenyx Wins FDA Approval for FAYUVI Gene Therapy
Novato, California, U.S., September 17, 2026 Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has...
Gene Therapy
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Mission Bio Launches Tapestri Triomics for Single-Cell Analysis
SOUTH SAN FRANCISCO, Calif. — September 16, 2026 Mission Bio announced the launch of Tapestri® Triomics, a new expansion...
Rocket Pharmaceuticals Advances RP-A501 Toward Danon Disease Approval
CRANBURY, N.J. — September 15, 2026 Rocket Pharmaceuticals, Inc. announced alignment with the U.S. Food and Drug Administration (FDA)...
Tenaya Therapeutics Gains RMAT Designation for TN-401
SOUTH SAN FRANCISCO, Calif. — September 14, 2026 Tenaya Therapeutics, Inc. announced that the U.S. Food and Drug Administration...
Cyllene Therapeutics’ EG110A Gets FDA RMAT Designation
Paris, France and New York, USA, September 14, 2026 Cyllene Therapeutics announced that the U.S. Food and Drug Administration...
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Nanoscope’s MOGENRY BLA Accepted by FDA for Retinitis Pigmentosa
Dallas, Texas, USA, September 9, 2026 Nanoscope Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has...
Opus Genetics Advances OPGx-BEST1 Toward Phase 3
RESEARCH TRIANGLE PARK, N.C. — September 9, 2026 Opus Genetics, Inc. announced positive 3- and 6-month data from the...
Intellia Therapeutics Gains FDA Priority Review for Lonvo-z in HAE
CAMBRIDGE, Mass. — September 8, 2026 Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted...
RZNOMICS Advances High-Efficiency Circular RNA Production
Seoul, South Korea, September 7, 2026 RZNOMICS Inc., a biopharmaceutical company focused on RNA-based gene therapies, has reported major...
Alkeus Reports Positive Gildeuretinol Results in Stargardt Disease
Cambridge, Massachusetts — September 3, 2026 Alkeus Pharmaceuticals announced the publication of TEASE-1 efficacy and safety results in JAMA...
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