Angelman syndrome
Encoded Therapeutics Lays off 29 Percent of Workforce
The move will provide funds to advance ETX101, a gene therapy the company is developing for SCN1A-positive Dravet syndrome, among other programs.
Biogen, Ionis Stop ALS Antisense Drug Development; Biogen Drops Angelman Syndrome Collaboration
Ionis Pharmaceuticals said it will advance the ASO for Angelman syndrome independently, without a partner.
Yale-Led Team Wins $40M in NIH Funding to Develop Gene-Editing Tech for Neurogenetic Diseases
As proof of concept, researchers at Yale, Rush University, and elsewhere will initially develop CRISPR-based therapies for Angelman and HIST1H1E syndromes.