Industry
FAST Ventures
After an influx of funding to expand its drug discovery pipeline and clinical infrastructure, FAST took a venture philanthropy approach, launching the company GeneTx Biotherapeutics, to advance the first investigational antisense oligonucleotide (ASO) through early stage research, IND-enabling studies, and eventually into a Phase 1/2 clinical trial. The GeneTx team was able to file an IND application and partner the program with Ultragenyx Pharmaceutical in under 10 months from the declaration of a clinical candidate for the first clinical trial of a disease modifying therapy in AS.
In 2022, GeneTx was acquired by Ultragenyx Pharmaceuticals after preliminary Phase 1/2 open-label clinical data was disclosed, supporting continued clinical development of the program and representing a critical milestone in the advancement of potential therapies for AS.
This led to the establishment of an associated drug development accelerator called AS²Bio Inc. The company is creating a drug development ecosystem to bring transformative therapeutics to those living with Angelman syndrome (AS).