- Collaboration to focus on gene therapy
research for the treatment of ultra-rare disease
NORTHBROOK, Ill., June 20,
2024 /PRNewswire/ -- Astellas Pharma US, Inc.
("Astellas") announced today the company has entered into a
sponsored research agreement with the UMass Chan Medical School to
proceed with research for an adeno-associated virus (AAV) vector
mediated gene therapy for the treatment of Alexander disease, a fatal, ultra-rare disease
for which the only current treatment option is supportive care.
This collaboration is part of a focused initiative to deliver value
to patients with rare disease through innovative R&D models
including investigator-led research.
Under the terms of the agreement, UMass Chan will lead research
activities to accelerate the program toward clinical study while
Astellas will support UMass Chan activities by providing drug
discovery research expertise. The current agreement lasts for one
year with an opportunity for Astellas to extend to two years.
Alexander disease is an
ultra-rare and progressive central nervous system (CNS) disorder
caused by mutations in the glial fibrillary acidic protein (GFAP)
gene that usually has a fatal outcome. Most affected patients do
not live beyond their first few years. Alexander disease affects astrocytes, a type
of cell that makes up the majority of cells in the CNS and leads to
the destruction of white matter in the brain, causing progressively
worsening severe intellectual and physical disabilities; and
eventually affected individuals will lose life-sustaining
abilities.1
Yoshitsugu Shitaka, Ph.D., Chief Scientific Officer at
Astellas
"We are pleased to partner with UMass Chan, a leading medical
academic center in Massachusetts,
and help foster innovation in this important ecosystem. At
Astellas, we strive to find innovative ways to treat patients with
the highest unmet medical needs, who have traditionally been left
behind, including rare and ultra-rare diseases.
Gene therapy is a complex breakthrough area where traditional
approaches might not be sufficient or optimal, and ultra-rare
diseases like Alexander disease
cannot be appropriately addressed with the usual pharma models.
Only by working alongside all the key players such as patients,
physicians, academic centers, can we advance pioneering work to
challenge the status quo, by turning innovative science into
meaningful treatments for those patients who are waiting."
The sponsored research agreement supports investigations being
done by Jun Xie, PhD, associate
professor of microbiology & physiological systems, and
Guangping Gao, PhD, the Penelope
Booth Rockwell Professor in Biomedical Research, professor of
microbiology & physiological systems, director of the Horae
Gene Therapy Center and co-director of the Li Weibo Institute for
Rare Diseases Research at UMass Chan, who have developed an
adeno-associated virus vector that can express a small RNA silencer
that suppresses the mutant proteins that cause AxD in mice.
Guangping Gao, PhD,
the Penelope Booth Rockwell Professor in Biomedical
Research, professor of microbiology & physiological systems,
director of the Horae Gene Therapy Center and co-director of the Li
Weibo Institute for Rare Diseases Research, UMass Chan
"We are enthused to be working with Astellas. Their drug
discovery research expertise is a critical component in translating
this work from bench to bedside, and bringing gene therapy to
patients in the clinic who are struggling with this deadly
disease."
Jun Xie, Ph.D., associate
professor of microbiology & physiological system, UMass
Chan
"Silencing GFAP, the mutant protein that causes AxD, with our
artificial miRNA could be a promising therapeutic approach to
treatment. Collaborating with Astellas will bring us one step
closer to our goal of helping patients with this disease."
About Astellas Pharma US
Astellas Pharma US, Inc. is a U.S. affiliate of Tokyo-based Astellas Pharma Inc., a
pharmaceutical company conducting business in more than 70
countries around the world. Astellas stands on the forefront of
healthcare change to turn innovative science into value for
patients. Keeping our focus on addressing unmet medical needs and
conducting our business with ethics and integrity enables us to
improve the health of people throughout the U.S. and around the
world. For more information on Astellas, please visit:
https://www.astellas.com/us. You can also follow us on Twitter at
@AstellasUS, Facebook at www.facebook.com/AstellasUS or LinkedIn at
www.linkedin.com/company/astellas-pharma-us.
For more information about UMass Chan Medical School,
please visit https://www.umassmed.edu/.
Cautionary Notes
In this press release, statements made with respect to current
plans, estimates, strategies and beliefs and other statements that
are not historical facts are forward-looking statements about the
future performance of Astellas. These statements are based on
management's current assumptions and beliefs in light of the
information currently available to it and involve known and unknown
risks and uncertainties. A number of factors could cause actual
results to differ materially from those discussed in the
forward-looking statements. Such factors include, but are not
limited to: (i) changes in general economic conditions and in laws
and regulations, relating to pharmaceutical markets, (ii) currency
exchange rate fluctuations, (iii) delays in new product launches,
(iv) the inability of Astellas to market existing and new products
effectively, (v) the inability of Astellas to continue to
effectively research and develop products accepted by customers in
highly competitive markets, and (vi) infringements of
Astellas' intellectual property rights by third parties.
Information about pharmaceutical products (including products
currently in development) which is included in this press release
is not intended to constitute an advertisement or medical
advice.
- Kuhn J, Cascella M. Alexander Disease. 2023 Sep 4. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing;
2024 Jan–. PMID: 32965913.
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SOURCE Astellas US, LLC