AS2 Bio

AS2 Bio is a private, pre-clinical stage biotech dedicated to creating therapies for Angelman syndrome (AS), a rare neurodevelopmental disorder. The company is building a pipeline across multiple therapeutic modalities, including targeted protein degradation, and is led by a team with deep experience in rare disease drug development and direct personal connections to the AS community. Its strategy is closely tied to the Foundation for Angelman Syndrome Therapeutics (FAST), leveraging established research consortia and regulatory pathways to advance its programs.

Multi-modal therapeutic approach for Angelman syndrome, including targeted protein degradation (molecular glues, PROTACs), potentially extending to other modalities like biologics and viral technology.

The Angelman syndrome therapeutic landscape is becoming increasingly competitive, with companies like Ultragenyx (via GeneTx acquisition) advancing an ASO, Roche, and others exploring gene therapy and other modalities. AS2 Bio's differentiation lies in its multi-modal strategy and deeply embedded leadership within the patient advocacy and research consortium framework.