Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.
Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Asklepios BioPharmaceutical (AskBio) secured a $235 million investment from Vida Ventures and TPG Capital.
Roche is buying U.S.-based gene therapy specialist Spark Therapeutics for $4.3 billion after developments in this area convinced the Swiss drugmaker to “step up”, Chief Executive Severin Schwan said.
Amicus Therapeutics inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.
The U.S. FDA issued a Complete Response Letter to Netherlands-based Pharming Group for the company’s supplemental Biologics License Application for Ruconest.
The FDA accepted Amicus Therapeutics’ NDA for migalastat to treat Fabry disease in patients with amenable mutations.
Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
Shire’s experimental treatment SHP609 to address cognitive impairment in pediatric patients diagnosed with the rare disease known as Hunter syndrome failed a key trial.
Barely a week after announcing it was moving into a new corporate headquarters, Sangamo Therapeutics announced that the company has dosed the first patient in its Phase I/II gene therapy trial.