Encoded Therapeutics is shrinking its headcount by 29% as the gene therapy biotech draws resources away from its early-stage ...

Dr. Eric Levine’s cutting-edge autism research at UConn School of Medicine is reprogramming the donated skin or blood cells ...

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

This is one of 22 cutting-edge projects selected to share in a total of $8.1 million in grants raised through the Eagles ...

Angelman Syndrome is a very rare neurogenetic disorder that occurs in approximately one in 15,000 births, caused by the loss of function of a gene ...

The move will provide funds to advance ETX101, a gene therapy the company is developing for SCN1A-positive Dravet syndrome, among other programs.