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.

For patients with deadly and debilitating conditions, FDA drug approvals can be cause for celebration. Many of these patients and their families have been waiting years for better therapies – or any therapy at all. They consume every public announcement about drug approvals for their particular disease like it’s water in the desert. But there is also a sense of trepidation once the drug launches. How much will this new drug cost? What will insurance cover? What will be our share?