gene therapy

Abeona Therapeutics Inc (NASDAQ: ABEO) announced magnetic resonance imaging (MRI) data from the Phase 1/2 Transpher A study of ABO-102 gene therapy in Sanfilippo Syndrome Type A (MPS IIIA). Data indicated that ABO-102 increased grey matter, corpus callosum, and amygdala volumes in...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from open-label Phase 1/2 trial of valoctocogene roxaparvovec, an investigational gene therapy candidate for severe hemophilia A. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH)...

Spark Therapeutics, a unit of Roche Holdings AG (OTC: RHHBY), has announced updated data from the ongoing Phase 1/2 trial of SPK-8011 gene therapy in hemophilia A. Data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2021. These data demonstrated...

Lineage Cell Therapeutics Inc (NYSE: LCTX) reported updated interim results from its Phase 1/2a study of its lead product candidate, OpRegen cell therapy for dry age-related macular degeneration (AMD) with geographic atrophy (GA).  Overall, 10/12 (83%) of the Cohort 4 patients...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from GENEr8-1 Phase 3 study for valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.  Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021...

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) marketing application seeking approval for hemophilia A gene therapy, valoctocogene roxaparvovec. With today's validation, the application review can now commence. A CHMP opinion...

Sana Biotechnology Inc (NASDAQ: SANA) has entered into a lease agreement to develop a 163,000 square foot manufacturing facility in Fremont, California.  The facility will support the manufacture of Sana's late-stage clinical development and early commercial product...

European Medicines Agency's (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that the benefit-risk of Bluebird Bio Inc's (NASDAQ: BLUE) Zynteglo (betibeglogene autotemcel gene therapy) remains favorable. Hence EMA is lifting the voluntary marketing...

Abeona Therapeutics Inc (NASDAQ: ABEO) has announced updated data from Phase 1/2a trial for up to six years following treatment with EB-101 for recessive dystrophic epidermolysis bullosa (RDEB).  The data showed that investigator assessment of wound healing of over 50% from...

OncoSec Medical Inc (NASDAQ: ONCS) will evaluate its DNA-plasmid interleukin-12 (IL-12) TAVO (tavokinogene telseplasmid) with Merck & Co Inc's (NYSE: MRK) Keytruda (pembrolizumab) in KEYNOTE-C87 Phase 3 trial.  The planned trial will evaluate the overall survival...

Beam Therapeutics Inc (NASDAQ: BEAM) joins forces with Apellis Pharmaceuticals Inc (NASDAQ: APLS) to create new gene-editing treatments for complement-driven diseases. The partnership will leverage Beam’s base-editing technology to discover new treatments against various...

Orchard Therapeutics plc (NASDAQ: ORTX) has provided updates on the progress of its lead gene therapy programs targeting metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I Hurler syndrome (MPS-IH), and Wiskott-Aldrich syndrome (WAS). After receiving feedback from the...

UniQure N.V. (NASDAQ: QURE) has announced 52-week data from its Phase 3 HOPE-B gene therapy trial of etranacogene dezaparvovec to treat hemophilia B.  Data demonstrated durable, sustained increases in Factor IX (FIX) activity at 52-weeks post-infusion with a mean FIX activity...

The FDA notified VBL Therapeutics Ltd (NASDAQ: VBLT) that clearance of new VB-111 batches for use in the U.S. is currently pending, as review by the Chemistry, Manufacturing, and Controls (CMC) group is still ongoing. VB-111 (ofranergene obadenovec) gene therapy for ovarian cancer. Until...

CRISPR Therapeutics (NASDAQ: CRSP) has signed a deal with Capsida Biotherapeutics, a gene therapy player specializing in AAV engineering, to carve out a delivery mechanism for the company’s gene-editing technology in familial amyotrophic lateral sclerosis (ALS) and rare...