Pfizer today announced very positive results from a Phase III study of fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult men with moderately severe to severe hemophilia B. This caps off a breakthrough year for hemophilia B treatment, as the first gene therapy for this blood clotting…

iECURE, a gene editing startup focused on liver disorders, this week announced a $65 million Series A-1 financing. The company is collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, MD, PhD, who led the gene-therapy trial that ended with teenager Jesse Gelsinger’s death…

Researchers at University College London (UCL) have developed a new gene therapy approach for epilepsy and potentially other neurological and psychiatric diseases that works by reducing the excitability of overactive brain cells. The new closed-loop gene therapy approach enables the on-demand expression of a gene that tamps down the activity…

Applied Genetic Technologies Corporation (AGTC), a developer of adeno-associated virus (AAV)-based gene therapies, is being acquired by Syncona Limited. AGTCs initial focus has been on inherited retinal diseases (IRDs). Syncona will pay $23.5 million ($0.34 per share) in cash at the closing of the transaction plus potential future aggregate cash payments…

Xcell Biosciences and aCGT Vector have announced a collaboration for manufacturing and analysis around personalized cell and gene therapies for cancer patients. aCGT Vector will provide its point-of-care GMP-licensed manufacturing platform to validate Xcellbio’s manufacturing and analytical AVATAR AI technology for use in precision cancer treatment. The partnership pairs aCGT’s…

Eli Lilly and Company announced today that it intends to acquire Akouos, a developer of gene therapies focused on treating hearing loss, for $12.50 per share, or a total of $487 million payable at the closing of the deal. The agreement also includes one non-tradeable contingent value right per share…

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Jonathan ThonFounder, CEO, STRM.BIO A serial biotech entrepreneur, Jonathan Thon, is the founder and CEO of STRM.BIO, a pre-clinical, VC-backed biotechnology company that is leveraging extracellular vesicles (EVs) to deliver gene therapies. Prior to launching STRM.BIO, he founded and served as CEO / CSO of PlateletBio…

Just one day after it received a positive recommendation for one gene therapy, bluebird got another. This one is for betibeglogene autotemcel (beti-cel) for the treatment of people with beta-thalassemia who require regular red blood cell (RBC) transfusions. The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted…

A U.S. FDA advisory committee unanimously approved bluebird bio’s elivaldogene autotemcel (eli-cel) gene therapy for early active cerebral adrenoleukodystrophy (CALD) in certain patients. The committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a PDUFA goal date set for September 16,…

The first disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency will soon reach the market in Europe.  PTC Therapeutics’ Upstaza, a gene therapy (eladocagene exuparvovec), received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Once ratified by the…

Belief BioMed’s BBM-H901, the first intravenously injectable hemophilia B gene therapy product developed and tested in Asia, showed very promising results in a Phase I study. Findings suggest that BBM-H901 is safe one year after infusion. Factor IX coagulation (FIX:C) concentration was sufficient to prevent bleeding events and minimize the…

In a striking collaboration, four companies shared laboratory and clinical data to find what was causing certain serious adverse events (SAEs) in Duchenne Muscular Dystrophy (DMD) gene therapy trials. The companies, all frontrunners in this field, are Pfizer, Sarepta, Solid Biosciences, and Genethon. Based on this work, they propose these…

Kriya Therapeutics has announced a $270 Million Series C financing that will support advancement of the company’s pipeline and continued scaling of its engineering, manufacturing, and computational platforms. Kriya has a portfolio of gene therapies with initial focus areas in ophthalmology, oncology, rare disease, and chronic disease. “We believe gene…

A cytomegalovirus (CMV) vector-based gene therapy delivered monthly, by inhalation or intraperitoneally, reversed aging-associated decline in a mouse model. Exogenous telomerase reverse transcriptase or follistatin genes were each safely and effectively delivered. The treatment significantly improved biomarkers associated with healthy aging, and mice’s lifespans were increased up to 41% without…