Where some saw an imperfect compromise, other attendees apparently left the talks in disgust. In published reports, for ...

Childs Cure co-founders Jainu and Shruti Jogani founded the organization to address the genetic causes of rare diseases, ...

UNC Charlotte researchers are contributing to pioneering breakthroughs in cancer therapeutics, communicable disease ...

Researchers from London's Moorfields Eye Hospital, biotech firm MeiraGTx and University College London have demonstrated that ...

Danish start-up Fuse Vectors has received $5.2 million in preseed financing to further develop their gene therapy technology.

Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia's technology platform specializes in Clustered Regularly Interspaced Short ...

Anthony Shum, MD, a pulmonologist at UC San Francisco, has tried to understand the random path of devastation that a rare ...

As the world recognizes Rare Disease Day on Feb. 28, Rockville-based biotechnology company, REGENXBIO is working to change ...

Four children born with a rare genetic form of severe blindness have all experienced significant improvements in their ...

An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood ...

Because his drug was a gene therapy, public grant money and private ... patented technology needed to make and administer it, for example. It also meant that Zolgensma had to do more than save ...