AUSTIN,
Texas, June 4, 2024 /PRNewswire/ --
The Foundation for Angelman Syndrome Therapeutics (FAST)
announced 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).
"The team at FAST brought together expertise, experience and
commitment to patients in order to create a unique platform—one now
poised to make transformative change a reality, as safely and
quickly as possible, for those living with Angelman syndrome," said
Julien de Bournet, CEO of AS²Bio.
"We want to use the success we have had so far to turbocharge that
future."
Since its founding, FAST had a vision to disrupt the traditional
research model and create an innovative drug development culture to
advance therapeutic discovery for Angelman syndrome (AS) and other
like-disorders. In 2016, as FAST's chief science officer, Dr.
Allyson Berent spearheaded a team
that created a focused roadmap that interlaced conventional as well
as new and emerging therapeutic approaches. Based on that vision,
FAST began 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, a monogenic non-degenerative neurologic disorder.
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.
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 first-in-human. Each AS
program will be part of a structured and supportive ecosystem with
the purpose of leveraging managerial talent and resources across
all.
With FAST financing, companies are being created to address 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.
Those investigational development programs currently being advanced
include: in vivo AAV-gene replacement therapy, in-vivo CRISPR gene
editing, and ex vivo hematopoietic progenitor stem cell gene
replacement therapy.
Moreover, nearly all of the learnings from these translatable
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 be transferable to other neurodevelopmental
disorders.
Employees include Julien de
Bournet, chief executive officer; Allyson Berent, chief development officer; and
Jennifer Panagoulias, chief
operations officer.
CONTACT:
FAST
info@cureangelman.org
630-852-3278
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SOURCE Foundation for Angelman Syndrome Therapeutics (FAST)