Regeneron Secures FDA Approval for Otarmeni Gene Therapy and Announces Landmark U.S. Pricing Agreement
Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) announced that the U.S. Food and Drug Administration (FDA) has...
Gene therapy
Gan & Lee Pharmaceuticals Secures NMPA Clinical Approval for GLR1062, China’s First-in-Class Gene Therapy for Wet Age-Related Macular Degeneration
Gan & Lee Pharmaceuticals (SHA: 603087) has announced that China’s National Medical Products Administration (NMPA)...
FDA Issues Draft Guidance for Genome Editing Therapy Safety Assessment Using Next-Generation Sequencing
The U.S. Food and Drug Administration (FDA) released draft guidance on Monday outlining standardized methods...
FDA Approves Rocket Pharma’s Kresladi – First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I
The U.S. Food and Drug Administration (FDA) this week approved Rocket Pharmaceuticals’ Kresladi (marnetegragene autotemcel),...
Belief BioMed Partners with Grand Life Sciences on BBM-H803 – AAV Gene Therapy for Hemophilia A Targets Greater China Market
Belief BioMed (“BBM”) and Grand Life Sciences Group Co., Ltd. (HKG: 0512) announced an exclusive...
Belief BioMed Secures Macao Approval for BBM‑H901 – China’s First Hemophilia B Gene Therapy Expands to Greater China with Takeda
Belief BioMed announced marketing approval from the Pharmaceutical Administration Bureau of Macao Special Administrative Region...
YolTech’s YOLT-202 Wins FDA Phase II/III Approval – In Vivo Base Editing Gene Therapy Targets Alpha-1 Antitrypsin Deficiency
YolTech Therapeutics announced FDA clearance to initiate an open-label, single-dose expansion Phase II/III clinical study...
Porton Advanced Partners with T&L Biotechnology – CGT One-Stop Solution Targets Global Cell and Gene Therapy Market
Porton Advanced, a subsidiary of Porton Pharma Solutions, Ltd, announced a strategic partnership with T&L...
AstraGenomics and Kactus Forge Gene Editing Partnership – Global Commercial Rights and Manufacturing Scale-Up for Nucleic Acid Therapeutics
AstraGenomics and Kactus announced a strategic collaboration agreement to accelerate development and commercialization of nucleic...
uBriGene Biosciences Partners with Innovec Biotherapeutics on AAV Gene Therapy for Hereditary Eye Diseases
uBriGene Biosciences, a China-based advanced therapy medicinal product (ATMP) CDMO, has entered a strategic partnership...
FDA Announces Flexible CMC Pathway for Cell and Gene Therapies to Accelerate Development
The FDA’s Center for Biologics Evaluation and Research (CBER) announced a flexible, lifecycle‑based approach to...
GenEditBio’s GEB-101 Wins FDA IND Approval as World’s First Corneal Gene Therapy
GenEditBio announced that GEB-101, its self‑developed in vivo genome editing investigational drug, has received Investigational...
BDgene Doses First Patient with BD920 CRISPR Gene Therapy for HPV-16 HSIL
Shanghai BDgene Technology Co., Ltd. announced that BD920, its in vivo CRISPR gene editing therapy,...
IVIEW GVB-2001 Gene Therapy Treats First POAG Patient Successfully
IVIEW Therapeutics Inc. announced the successful treatment of the first patient with primary open-angle glaucoma...
Beijing Friendship Hospital Launches Cell and Gene Therapy Center for Liver Diseases
The Cell and Gene Therapy Innovation and Transformation Center was officially established at Beijing Friendship...
FDA Waskyra WAS Gene Therapy First Cell-Based
The US Food and Drug Administration (FDA) approved Waskyra (etuvetidigene autotemcel) as the first cell‑based...
Regeneron Tessera Partner on TSRA-196 Gene-Writing Therapy for AATD with $275M Deal
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Tessera Therapeutics announced a global collaboration to develop and...
Novartis’s Itvisma Wins FDA Approval as First Gene Therapy for SMA
Novartis AG (NYSE: NVS) announced that Itvisma (onasemnogene abeparvovec‑brve) received U.S. FDA approval for children...
MeiraGTx Grants Eli Lilly Worldwide Rights to AAV‑AIPL1 Gene Therapy for LCA4
MeiraGTx Holdings plc (NASDAQ: MGTX) announced a strategic ophthalmology collaboration with Eli Lilly and Company...