gene therapy

While basking in the glow of the approval for its Alzheimer’s treatment, Biogen Inc (NASDAQ: BIIB) announced that a gene therapy candidate for eye disease failed a late-stage clinical trial, yet again. The company announced topline results from Phase 3 STAR study of...

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and Crispr Therapeutics AG (NASDAQ: CRSP) have announced new data from Phase 1/2 clinical trials evaluating CTX001 in beta-thalassemia and sickle cell disease. Presenting data from two trials at the European Hematology...

Bayer AG’s (OTC: BAYRY) Parkinson’s disease stem cell therapy has officially landed in the clinic, while another treatment for the neurodegenerative movement disorder is recruiting patients. Bayer’s subsidiary BlueRock Therapeutics’s trial will...

The FDA has lifted the clinical hold on Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of Bluebird Bio Inc's (NASDAQ: BLUE) LentiGlobin for sickle cell disease and two Phase 3 studies of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia. The...

Oyster Point Pharma Inc (NASDAQ: OYST) has announced the expansion of its pipeline with the introduction of Enriched Tear Film (ETF) Gene Therapy and proof-of-concept in vivo study results from its first gene therapy candidate, OC-101. Oyster Point's investigational drug, OC-01...

VBL Therapeutics Ltd (NASDAQ: VBLT) has amended the primary endpoint in the OVAL Phase 3 registration-enabling study of VB-111 (ofranergene obadenovec) gene therapy for ovarian cancer. Following discussion with the FDA, the clinical trial amendment includes a second,...

The independent Data Safety Monitoring Board (DSMB) has reviewed safety data for the fully enrolled first cohort of ten patients of the Phase 1/2 trial evaluating UniQure NV's (NASDAQ: QURE) AMT-130 for Huntington's disease. The DSMB recommended continued...

The European Medicines Agency (EMA) has granted its request for accelerated assessment review to BioMarin Pharmaceutical Inc’s (NASDAQ: BMRN) valoctocogene roxaparvovec, gene therapy to treat severe hemophilia A, with an opinion expected in the first half of 2022,...

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorizations for Bluebird bio Inc’s (NASDAQ: BLUE) Skysona (elivaldogene autotemcel) new gene therapy for children with a rare, inherited neurological...

Biogen Inc (NASDAQ: BIIB) and Ginkgo Bioworks have entered into a gene therapy collaboration to develop a next-generation adeno-associated virus (AAV)-based vectors production platform. Under the terms of the agreement, Ginkgo will utilize its cell programming platform and...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced an update to its previously reported results from Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. Data will be shared at the International Society...

Sarepta Therapeutics Inc (NASDAQ: SRPT) has announced positive 12-week results from the first 11 participants enrolled in open-label study 9001-103 ENDEAVOR being conducted in partnership with Roche Holdings AG's (OTC: RHHBY). Results demonstrated robust expression of...

Vera Therapeutics Inc has priced its 4.35 million share IPO at $11 per share, well below its targeted range of $14 to $16, raising $47.58 million in gross proceeds. The biotech will start trading on NASDAQ from Monday under the ticker “VERA.” Vera’s lead candidate...

Otonomy Inc (NASDAQ: OTIC) may have hit a snag with the development of its lead ear disorder candidate Otividex. Still, the company is trumpeting an early win for a gene therapy designed to treat congenital hearing loss. The drug candidate, dubbed OTO-825, reduced hearing loss...

Dyne Therapeutics Inc (NASDAQ: DYN) has announced new preclinical data from its myotonic dystrophy type 1 (DM1) program, including results demonstrating sustained knockdown of toxic human nuclear DMPK RNA, the genetic basis of the disease. Data were presented at the American...