At WORLDSymposium, Astellas Pharma presented interim safety data from a Phase I/II study assessing the company’s AAV gene therapy for the rare, autosomal metabolic disease Pompe disease.
The Glycogen Storage Disease Program at Connecticut Children’s and UConn Health reported success in a Phase I/II trial of a gene therapy for GSD-Ia. The gene therapy is developed with Ultragenyx and is dubbed DTX401.
One week after expanding the company’s neurodegenerative diseases partnership with Bristol Myers Squibb, Evotec entered into a drug discovery collaboration for metabolic diseases with Eli Lilly, focusing on kidney disease and diabetes.
Japan’s Takeda Pharmaceutical announced deals with two biotech companies, Seattle-based Immusoft and San Diego’s Poseida Therapeutics.
Inventiva, with headquarters in Daix, France, received Breakthrough Therapy designation from the U.S. Food and Drug Administration for the company’s lanifibranor for nonalcoholic steatohepatitis (NASH).
Waltham, Massachusetts-based biotech firm Skyhawk Therapeutics is expanding a strategic collaboration deal with Merck as the two companies are working to develop and commercialize a particular type of therapy built on modulating RNA splicing using Skyhawk’s SkySTAR technology platform.
Researchers at the University of Pennsylvania and Children’s National Hospital in Washington, D.C., utilized a hybrid approach that combined gene therapy with gene editing to treat a rare genetic disease in animal models.
Copenhagen-based Zealand Pharma announced a bid to buy substantially all the assets from New Jersey-based Valeritas in $23 million in cash and taking on certain liabilities.
With $28.5 million in financing, Cambridge, Mass.-based Comet Therapeutics is emerging from stealth mode with a focus on developing a portfolio of drug candidates that centers around efforts to re-engineer CoEnzyme-A (CoA).
TARGET PharmaSolutions Inc. – a real-world clinical data company – and Boehringer Ingelheim International GmbH entered into a multi-year strategic partnership for TARGET-NASH, a longitudinal observational study that evaluates patients with nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH).