Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on
changing the possible for patients through engineered cells, today
highlighted that Nature Biotechnology has published a paper titled
“Young glial progenitor cells competitively replace aged and
diseased human glia in the adult chimeric mouse brain.” This paper
showed that when healthy wild-type (WT), pluripotent stem
cell-derived glial progenitor cells (hGPCs) – the cells that give
rise to the glial support cells of the brain – were transplanted
into adult mice that had been neonatally transplanted with mutant
Huntingtin (mHTT)-expressing hGPCs, the healthy cells outcompeted
and eliminated the diseased glia, ultimately repopulating the brain
with the healthy transplanted cells. These data establish an
additional proof-of-concept for the development of SC379, Sana’s
pluripotent stem cell-derived GPC product candidate, as a potential
therapy to deliver healthy allogeneic GPCs to patients with certain
central nervous system disorders.
“It is remarkable to see that healthy human glial cells can
engraft and function in vivo, and succeed in outcompeting resident
glial cells, thereby eradicating the diseased cells from the brain
of the mice,” said Steve Harr, Sana’s President and Chief Executive
Officer. “We have designed SC379 as a potential glial cell
replacement therapy, and these findings support its possible
application in a large number of neurological indications, such as
secondary progressive multiple sclerosis and the childhood myelin
disorders like Pelizaeus-Merzbacher disease, as well as
Huntington’s disease and other diseases of glial cells. Our goal is
to begin clinical testing of SC379 as early as 2025.”
Steve Goldman, MD, PhD, Sana’s Head of CNS Therapy and lead
author on the paper, added, “Diseases of glial cells are among the
most prevalent and debilitating conditions in neurology, as they
include both the myelin-producing oligodendrocytes and the
astrocytic support cells of neurons. We have developed the methods
for producing and isolating glial progenitor cells – which produce
both oligodendrocytes and astrocytes – from pluripotent stem cells,
and for delivering them in the purity and quantity necessary to
replace diseased cell populations.”
In the study, human glial chimeric mice were used to model the
impact of competition between healthy and diseased human glia in
vivo. Genetically tagged WT hGPCs derived from human embryonic stem
cells (hESCs) were engrafted into the striata of adult mice, which
were neonatally chimerized with hGPCs derived from a sibling line
with Huntington’s disease (HD). Over time, the group found that the
healthy WT cells overtook the HD cells and repopulated the striatum
with healthy cells (54 weeks, P < 0.0001; 72 weeks, P <
0.0001).
The study also evaluated the impact of engrafting younger hGPCs
into adult mice that had been neonatally engrafted with separately
tagged older glia derived from the same healthy cell line. The data
demonstrated that the younger hGPCs rapidly infiltrated the
striatum and eventually replaced the older hGPCs, leading to a
substantial recolonization of the tissue (P < 0.0001). This was
associated with the apoptotic death of the older cells, which was
triggered by the introduction of the younger hGPCs and enabled a
repopulation of the mouse striatum with the younger cells.
About Sana BiotechnologySana Biotechnology,
Inc. is focused on creating and delivering engineered cells as
medicines for patients. We share a vision of repairing and
controlling genes, replacing missing or damaged cells, and making
our therapies broadly available to patients. We are a passionate
group of people working together to create an enduring company that
changes how the world treats disease. Sana has operations in
Seattle, WA, Cambridge, MA, South San Francisco, SF, Bothell, WA
and Rochester, NY. For more information about Sana Biotechnology,
please visit https://sana.com/.
Cautionary Note Regarding Forward-Looking
StatementsThis press release contains forward-looking
statements about Sana Biotechnology, Inc. (the “Company,” “we,”
“us,” or “our”) within the meaning of the federal securities laws,
including those related to the company’s vision, progress, and
business plans; expectations for its development programs, product
candidates and technology platforms, including its preclinical,
clinical and regulatory development plans and timing expectations;
the potential for pluripotent stem cell-derived hGPC
transplantation as a treatment for Huntington’s Disease and other
glial-based neurodegenerative conditions; and expectations
regarding the potential significance and impact of the data,
including regarding the potential of SC379 as a potential therapy
to deliver healthy allogeneic GPCs to patients with certain central
nervous system disorders, including secondary progressive multiple
sclerosis, Pelizaeus-Merzbacher disease, Huntington’s disease, and
other diseases of glial cells. All statements other than statements
of historical facts contained in this press release, including,
among others, statements regarding the Company’s strategy,
expectations, cash runway and future financial condition, future
operations, and prospects, are forward-looking statements. In some
cases, you can identify forward-looking statements by terminology
such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,”
“continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,”
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“predict,” “seek,” “should,” “target,” “will,” “would” and other
similar expressions that are predictions of or indicate future
events and future trends, or the negative of these terms or other
comparable terminology. The Company has based these forward-looking
statements largely on its current expectations, estimates,
forecasts and projections about future events and financial trends
that it believes may affect its financial condition, results of
operations, business strategy and financial needs. In light of the
significant uncertainties in these forward-looking statements, you
should not rely upon forward-looking statements as predictions of
future events. These statements are subject to risks and
uncertainties that could cause the actual results to vary
materially, including, among others, the risks inherent in drug
development such as those associated with the initiation, cost,
timing, progress and results of the Company’s current and future
research and development programs, preclinical and clinical trials,
as well as economic, market, and social disruptions. For a detailed
discussion of the risk factors that could affect the Company’s
actual results, please refer to the risk factors identified in the
Company’s Securities and Exchange Commission (SEC) reports,
including but not limited to its Quarterly Report on Form 10-Q
dated May 8, 2024. Except as required by law, the Company
undertakes no obligation to update publicly any forward-looking
statements for any reason.
Investor Relations & Media:Nicole
Keithinvestor.relations@sana.commedia@sana.com
Sana Biotechnology (NASDAQ:SANA)
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