COLUMBUS, Ohio, July 10,
2024 /PRNewswire/ -- Andelyn Biosciences, Inc.,
a leading and patient-focused cell and gene therapy Contract
Development and Manufacturing Organization (CDMO), has been
selected by UMass Chan Medical School to use its suspension AAV
Curator™ Platform to manufacture clinical grade AAV9-CSA
vector to treat Cockayne syndrome.
The nonprofit patient-advocacy organization, Riaan Research
Initiative, has provided the largest known donation ever made
toward the development of a treatment for Cockayne syndrome.
The gift from Riaan Research Initiative and the partnership with
Andelyn brings the start of clinical trials for the fatal autosomal
recessive disorder one step closer. Andelyn's extensive
experience in gene therapy development and manufacturing will be
leveraged to industrialize the production process and drive program
success.
Miguel Sena-Esteves, PhD,
Associate Professor of Neurology and Director of the Translational
Institute for Molecular Therapeutics said, "Our dream of making a
difference for Cockayne patients is coming closer every day, and
signing the contract with Andelyn Biosciences to make the clinical
material is a major step in that direction. Developing a gene
therapy for fatal pediatric neurological diseases is challenging in
many ways, but together with Riaan Research Initiative we have an
outstanding team to bring our program to a first-in-human gene
therapy clinical trial for Cockayne syndrome".
Matt Niloff, Chief Commercial
Officer of Andelyn, adds, "We are honored to have been selected to
be a key contributor in the advancement of this critical therapy
for Cockayne syndrome. With more and more organizations
across the industry adopting our data-driven and configurable AAV
Curator™ Platform, we are excited to add UMass as an important partner leveraging the
platform to drive the acceleration of the Cockayne syndrome
program."
With exceptional quality and scalable end-to-end development and
manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn continues
to partner with premier innovator organizations to enable the
progression of life-altering cell and gene therapies to cure rare
and prevalent diseases.
About Andelyn Biosciences, Inc.
Andelyn Biosciences is
a full-service cell and gene therapy CDMO focused on the
development, characterization and production of viral vectors for
gene therapy. With more than 20 years of experience, Andelyn's deep
scientific expertise has resulted in the production of cGMP
material for more than 450 clinical batches and 75 global clinical
trials. Operating out of three Columbus,
Ohio facilities, Andelyn supports its clients in developing
cell and gene therapies from concept through plasmid engineering
and manufacturing, process and analytical development, and cGMP
clinical and commercial manufacturing. Andelyn accelerates programs
and delivers high quality products by manufacturing product on its
configurable, data-driven AAV Curator™ Platform, or through a
tech transfer in of established client programs. Capabilities
include cGMP manufacturing for both adherent and suspension
processes up to a 2,000-liter capacity. A rigorous quality system,
regulatory support and supply chain vertical integration further
support clients in bringing their critical therapies to market. For
more information, visit andelynbio.com.
About UMass Chan Medical School
UMass Chan Medical
School, one of five campuses of the University
of Massachusetts system, comprises the T.H. Chan School of Medicine; the Morningside
Graduate School of Biomedical Sciences; the Tan Chingfen Graduate
School of Nursing; ForHealth Consulting of UMass Chan Medical
School, a public service health care consulting division; and
MassBiologics, the only nonprofit, FDA-licensed manufacturer of
vaccines, biologics and viral vector gene therapies in the United States. At UMass Chan, we
are advancing together to improve the health and wellness
of our diverse communities throughout Massachusetts and across the world by leading
and innovating in education, research, health care delivery and
public service. UMass Chan has built a reputation as a world-class
destination for biomedical research, with more than $300 million in annual funding and more than 500
active clinical trials and as a leader in primary care education,
perennially ranked in the top 10 percent of medical schools for
primary care by U.S. News and World Report. In 2021, the Medical
School received a $175 million
donation from The Morningside Foundation and was renamed UMass
Chan Medical School. Learn more at www.umassmed.edu.
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SOURCE Andelyn Biosciences