AS² Bio is a drug development accelerator established by the Foundation for Angelman Syndrome Therapeutics (FAST), a premier patient advocacy group known for disrupting traditional research models and spearheading innovative drug development in the neurogenetic space. 

 

Since its inception, FAST has been funding what would become over 90 translational research grants, resulting in 13 different therapeutic programs, each focused on different mechanisms to potentially address Angelman syndrome (AS), a monogenic non-degenerative neurologic disorder.

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After an influx of funding to expand its drug discovery pipeline and development capabilities, FAST took a venture philanthropy approach, launching the company GeneTx Biotherapeutics, to advance an 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 an effort to launch the first clinical trial of a potential disease modifying therapy in AS.

 

In 2022, GeneTx was acquired by Ultragenyx Pharmaceuticals after interim data from the Phase 1/2 open-label clinical trial data was reported, supporting continued clinical development of the program and representing a critical milestone in the advancement of potential therapies for AS.

 

Capitalizing on this success, FAST and AS² Bio are creating an integrated approach to drug development, leveraging collective expertise, resources, data, and vital networks to provide a “bridge” for new technologies so they can move, quickly and safely, from proof-of-concept to early stage clinical trials. Each AS program will be part of a structured and supportive ecosystem with the purpose of leveraging knowledge of the AS landscape and resources .

 

With FAST financing, companies are being created to further evaluate the therapeutic modalities that show promise in AS, ensuring priorities for these programs do not waiver and their potential human application is seen through, if deemed scientifically appropriate.

 

Nearly all of the learnings from these various therapeutic modalities can be leveraged to enable advancement beyond AS. In fact, the entire platform model—from the scientific modalities themselves to the organizational infrastructure that supports it—can support development of potential therapeutics for other neurodevelopmental disorders.

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To invest with us, or learn more, write to us at 
info@as2bio.com