Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on
changing the possible for patients through engineered cells, today
announced preclinical data from six presentations, including two
oral presentations, at the International Society for Stem Cell
Research (ISSCR) 2023 Annual Meeting.
“Our leading presence at ISSCR showcased key preclinical data
generated from our programs using our hypoimmune and fusogen
platforms,” said Doug Williams, Ph.D., Sana’s President of Research
and Development. “The ability to transplant allogeneic cells
engineered to evade immune detection without immunosuppression with
durable cell persistence and functionality has the potential to
transform the field of cell therapy, as well as medicine as a
whole. At this conference, much of our data focused on the
hypoimmune platform’s ability to avoid immune detection in various
preclinical models as well as the potential of incorporating this
platform into pancreatic islet cells for the treatment of type 1
diabetes. We also shared data demonstrating in vivo delivery of
various genetic payloads to human hematopoietic stem/progenitor
cells, highlighting an important capability with the fusogen
platform. Later this year, we look forward to sharing initial
clinical data from our hypoimmune platform which should help us
understand the translatability of these and other preclinical data
to humans, including data for our allogeneic CD19-directed CAR T
cells for the treatment of B-cell cancers and data for primary
human pancreatic islet cells for the treatment of type 1
diabetes.”
Oral PresentationsOn Thursday, June 15, an oral presentation
titled “Human Hypoimmune Primary Pancreatic Islets Evade Allogeneic
and Autoimmune Rejection Without Immunosuppression and Alleviate
Diabetes in Humanized Mice” featured data from in vitro and in vivo
studies of human hypoimmune (HIP) islet cells. The data
demonstrated that HIP islet cells were similar in size, cell type
composition, and in vitro insulin secretion as wild-type (wt) islet
cells, showing that HIP engineering itself does not impact islet
cell morphology or endocrine function. In vivo studies assessed the
survival of HIP islet cells in immunocompetent, diabetic allogeneic
humanized mice as well as in Sana’s proprietary humanized
autoimmune diabetes mouse model. In the diabetic allogeneic
humanized mouse study, the results demonstrated that HIP islet
cells survived and functioned to control glucose levels while wt
islet cells were rejected with no glucose control observed. The
study in the humanized autoimmune diabetes mouse model showed that
HIP islet cells also survived autoimmunity and alleviated diabetes
while wt islet cells were rejected with no glucose control
observed.
On Thursday, June 15, a second oral presentation titled “In Vivo
Delivery of Genetic Payloads to Human Hematopoietic Stem/Progenitor
Cells” featured data demonstrating the ability of Sana’s fusogen
platform to deliver genetic payloads to resting human hematopoietic
stem/progenitor cells (HSPCs) and access human HSPCs in both the
peripheral blood and bone marrow of humanized mice. The
presentation included data demonstrating the ability of
HSPC-targeted fusosomes to achieve high transduction efficiency and
specificity for HSPCs in vivo, avoiding off-target cellular
“sinks.” Highlights included a demonstration of in vivo nuclease
delivery for efficient editing of HSPCs, as well as specific gene
delivery to cells harboring a target HSPC receptor with >100x
selectivity.
Poster PresentationsOn Wednesday, June 14, poster #2082 titled
“Hypoimmune Rhesus Macaque Induced Pluripotent Stem Cells Achieve
Long-Term Survival in Fully Immunocompetent Allogeneic Recipients”
detailed data on the ability of Sana’s HIP-modified allogeneic
cells to escape immune detection in non-human primates (NHPs) in
the absence of immune suppression. HIP-modified induced pluripotent
stem cells (iPSCs) and wt iPSCs were transplanted into fully
immunocompetent NHPs without immunosuppression in a crossover
design, whereby after 6 weeks, NHPs initially administered one type
of iPSCs were injected with the other type of iPSCs. In all
instances, HIP iPSC grafts survived the entire study period. The
administration of HIP iPSCs did not generate de novo antibodies,
and no antibody-related killing of HIP iPSCs was observed,
regardless of the order of administration. In contrast, all wt iPSC
grafts were rejected within 2-3 weeks after transplantation, and
administration of wt iPSCs provoked a vigorous antibody and killing
response against these wt cells.
On Wednesday, June 14, poster #2034 titled “Engineered
Hypoimmune CAR T Cells Survive, Function, and Persist in
Immunocompetent Allogeneic Humanized Mice” outlined data evaluating
HIP CD19-directed CAR T cells versus unmodified CD19-directed CAR T
cells in three-month persistence studies with allogeneic humanized
mice. In all mice treated, tumor control was initially rapidly
achieved with both the unmodified CAR T cells and HIP CAR T cells.
However, in mice treated with unmodified CAR T cells, tumor control
did not last throughout the study or respond to rechallenge with
tumor cells. In contrast, in HIP CAR T cell-treated mice, tumor
control was maintained, including following a rechallenge with
tumor cells over 80 days after administration of the HIP CAR T
cells, demonstrating that the cells persist and remain functional
over multiple months in an allogeneic immune system.
On Thursday, June 15, poster #1122 titled “Standing Out From the
Crowd: Stem Cell-Derived Islet Cells Function Independent of
Clustering When Transplanted Intramuscularly” outlined data from
mice that received stem cell-derived islet cells (SC-islets) that
were intramuscularly implanted using standard clusters, standard
clusters disaggregated prior to implantation, or cells
differentiated without aggregation into clusters. SC-islets
implanted intramuscularly secreted C-peptide (a proxy for insulin)
and secretion, which increased over time, was independent of the
SC-islets’ initial clustering status. In addition, SC-islets
implanted intramuscularly as single cells effectively controlled
blood glucose levels, including after glucose challenge, and looked
histologically similar to SC-islets implanted as clusters.
On Friday, June 16, poster #166 titled “Stem Cell Derived Islet
Cells Show Robust Survival and Function When Transplanted in the
Muscle Without Need for Additional Bioscaffolding” presented data
on the effectiveness of stem cell-derived islet cells (SC-islets)
using intramuscular implant sites in immunodeficient diabetic mice.
The studies showed that these fully differentiated SC-islets can be
delivered intramuscularly with robust function and without the need
for bioscaffolding. Reversal of hyperglycemia was cell dose
dependent, and efficacy was observed with all tested doses.
About Sana’s Hypoimmune PlatformSana’s
proprietary hypoimmune platform is designed to create cells ex vivo
that can “hide” from the patient’s immune system to enable the
transplant of allogeneic cells without the need for
immunosuppression. We are applying hypoimmune technology to both
donor-derived allogeneic T cells, with the goal of making potent
and persistent CAR T cells at scale, and pluripotent stem cells,
which can then be differentiated into multiple cell types at scale.
Preclinical data from a variety of cell types demonstrate that
these transplanted allogeneic cells can evade both the innate and
adaptive arms of the immune system while retaining their function.
Our most advanced programs using hypoimmune technology include our
allogeneic CAR T program targeting CD19+ cancers (SC291), our
allogeneic CAR T program targeting CD22+ cancers (SC262), our
allogeneic CAR T program targeting BCMA+ cancers (SC255), and our
stem-cell derived pancreatic islet cell program for patients with
type 1 diabetes (SC451).
About Sana’s Fusogen PlatformSana’s proprietary
fusogen platform is designed to optimize in vivo cell specific
delivery of genetic material. Our goal is to be able to repair and
control genes in cells. Engineering cells in vivo requires the
development of both an appropriate delivery vehicle, as well as an
active component to effectively modify the target cell. Fusogens
are naturally occurring cell-targeting proteins. Sana reengineers
these proteins to target specific cell surface receptors, enabling
cell-specific delivery to many different types of cells. The
platform was developed to deliver an active component to any cell
in a specific, predictable, and repeatable way. This technology is
the backbone of Sana’s in vivo delivery platform and is
incorporated into various product candidates, including SG299, a
CD8-targeted fusosome that delivers a CD19 CAR to target CD19+
cancer 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, Cambridge, South San Francisco, and Rochester. 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 pre-clinical,
clinical, and regulatory development plans and timing expectations,
including regarding clinical data and the potential impact thereof;
the potential ability of HIP-modified cells to serve as a universal
off-the-shelf therapy that provides long-term durability of
response without immunosuppression; the potential ability of
intramuscular administration to serve as a preferred administration
route for patients; and the potential capabilities, benefits, and
impact of the hypoimmune platform, including the potential ability
to create cells ex vivo that can “hide” from the patient’s immune
system to enable the transplant of allogeneic cells without the
need for immunosuppression, and the fusogen platform, including the
potential ability to repair and control genes in cells and deliver
an active component to any cell in a specific, predictable, and
repeatable way. 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,” “intend,”
“may,” “objective,” “plan,” “positioned,” “potential,” “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, including due to the COVID-19
public health crisis. 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, 2023. 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)
Gráfica de Acción Histórica
De Abr 2024 a May 2024
Sana Biotechnology (NASDAQ:SANA)
Gráfica de Acción Histórica
De May 2023 a May 2024