SEATTLE and SAN RAFAEL, Calif., April 28, 2021 /PRNewswire/ -- The Allen
Institute and BioMarin Pharmaceutical Inc. announced a
collaboration today that will use technologies developed at the
Allen Institute to create new gene therapies aimed at rare genetic
diseases of the central nervous system. BioMarin will receive an
exclusive license to each program for research, development and
commercialization. The organizations did not disclose financial
terms.
It is increasingly understood in the scientific research
community that many central nervous system disorders affect
individual brain circuits or cell types rather than the entire
brain. Gene therapies are emerging as a promising approach to
improving outcomes for patients with these conditions. The Allen
Institute for Brain Science, a division of the Allen Institute, has
developed modified adeno-associated viruses, or AAVs, that have
been engineered to impact specific classes of cells in the
brain.
A key component of each engineered AAV is a unique molecular
enhancer, which ensures that viral gene expression is restricted to
only a precise cell type relevant in a particular disease. The cell
type specific enhancers enable a similar control over gene
expression in central nervous system tissues from several species,
strongly suggesting their potential for contributing to a new class
of precision gene therapies in patients. Scientists from BioMarin
and the Allen Institute will collaborate to establish whether these
novel AAVs can enable the creation of a new class of gene therapies
displaying a higher level of precision to treat diseases of the
central nervous system.
"The technology behind these viral tools could have incredible
impact for both basic neuroscience and clinical research," said
Allan Jones, Ph.D., President and
CEO of the Allen Institute. "On the basic research side, the
biggest impact will come from the Allen Institute's traditional
model of releasing data, tools and knowledge to the public for
broader scientific use, as we have with versions of these tools
used in basic research. BioMarin's industry leading expertise in
gene therapy and drug development will allow for the transformation
of the basic research to clinical therapy candidates."
"BioMarin is committed to bringing transformative therapies to
patients with rare genetic diseases. This collaboration offers us
great opportunities to partner with scientific leaders to develop
therapies in the CNS therapeutic area," said Lon Cardon, Ph.D., Senior Vice President, Chief
Scientific Strategy Officer. "Combining the Allen Institute's
leadership in large-scale genomic science in the central nervous
system with BioMarin's proven experience in developing
transformational therapies for rare genetic diseases, lays the
foundation to potentially deliver multiple investigational gene
therapies to the clinic."
Background on the Technology
The licensed technology was originally developed by researchers
at the Allen Institute for Brain Science toward the goal of
studying and classifying individual brain cell types. The tools are
built by engineering AAVs to carry genes that switch on in specific
types of neurons or other cells in the brain. A key component of
these engineered AAVs is a molecular "zip code" or enhancer, which
ensures that gene expression is restricted only to the correct
cellular address in the brain.
Neuroscientists at the Allen Institute have been using these
tools to ferry fluorescent labels to the brain, lighting up single
brain cell types or subclasses of brain cells under the microscope
to enable better studies of individual cell types. The researchers
have also shown that the same tools can often selectively label
comparable cell types. The viral tools for labeling brain cell
types were described in two recent publications led by research
teams at the Allen Institute for Brain Science, which were
published March 30, 2021 in the
journals Neuron and Cell Reports.
About the Allen Institute
The Allen Institute is an independent, 501(c)(3) nonprofit
research organization founded by philanthropist and visionary, the
late Paul G. Allen. The Allen Institute is dedicated to answering
some of the biggest questions in bioscience and accelerating
research worldwide. The Institute is a recognized leader in
large-scale research with a commitment to an open science model.
Its research institutes and programs include the Allen Institute
for Brain Science, launched in 2003, the Allen Institute for Cell
Science, launched in 2014, the Allen Institute for Immunology,
launched in 2018, and the MindScope Program, launched in 2020. In
2016, the Allen Institute expanded its reach with the launch of The
Paul G. Allen Frontiers Group, which identifies pioneers with new
ideas to expand the boundaries of knowledge and make the world
better. For more information, visit alleninstitute.org.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening rare genetic diseases. The Company's portfolio
consists of six commercialized products and multiple clinical and
pre-clinical product candidates. For additional information, please
visit www.biomarin.com. Information on BioMarin's website is
not incorporated by reference into this press release.
Media Contact:
Rob
Piercy, Director, Media Relations, Allen Institute
206.548.8486 | press@alleninstitute.org
Debra Charlesworth
BioMarin Pharmaceutical Inc.
415.455.7451
Investor Contact:
Traci McCarty
BioMarin Pharmaceutical Inc.
415.455.7558
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SOURCE BioMarin Pharmaceutical Inc.