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 ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

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

The data to date, funded by TransformaTx Biotherapeutics, a biotechnology company founded by the Foundation for Angelman Syndrome Therapeutics (FAST), has demonstrated complete symptomatic ...

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

to treat Angelman syndrome (AS). "This program has consistently delivered remarkable animal data, reinforcing its potential as a groundbreaking treatment," said Dr. Donald Kohn, Distinguished ...