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Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome

CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (IONS) (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ...
EurekAlert!

The Foundation for Angelman Syndrome Therapeutics to advance gene therapy candidate through IND-enabling studies conducted in partnership with the University of Pennsylvania

October 17, 2023—The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development ...
Healio

FDA grants orphan drug designation to biologic therapy for Angelman syndrome

Please provide your email address to receive an email when new articles are posted on . The FDA has granted orphan drug designation to a microbiome-modifying biologic to treat Angelman syndrome, ...
News Medical

FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the ...
Business Wire

Encoded Demonstrates Therapeutic Potential of a Vectorized miRNA-based Approach for Angelman Syndrome at the FAST Annual Global Science Summit

ORLANDO, Fla.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that ...
Yahoo Finance

Ionis doses first subject in Phase III trial of Angelman syndrome therapy

Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582, aimed at treating individuals with Angelman syndrome (AS), a rare ...
BioWorld

UCLA receives grant to support development of HSC gene therapy for Angelman syndrome

California Institute for Regenerative Medicine (CIRM) has awarded a $5.8 million translational research grant to a scientist at the University of California, Los Angeles (UCLA) to further support the ...