Cambridge, Massachusetts-based Dyno Therapeutics came out of stealth mode with an official launch of the company’s proprietary CapsidMap platform.

Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.

Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.

Pfizer’s IV Revatio, when added to inhaled nitric oxide, failed to meet the primary efficacy endpoint in treating newborns with persistent pulmonary hypertension in a Phase III study.

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.

New York-based Neurogene raised $68.5 million in a Series A funding round to support the company’s pipeline of therapeutics aimed at rare neurological diseases.

New research indicates that llamas may hold the key to flu vaccines.

The U.S. Food and Drug Administration lifted a clinical hold and accepted an Investigational New Drug Application (IND) for an experimental sickle cell disease treatment being jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

Amicus Therapeutics inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.