Eterna Therapeutics Inc. (Nasdaq: ERNA) (“Eterna”), a life science
company committed to realizing the potential of mRNA cell
engineering to provide patients with transformational new
medicines, and its discovery partner Factor Bioscience (“Factor”)
today announced new data on an iPS cell-derived multi-cell-type
therapeutic strategy for solid-tumor targeting, as well as advances
in nucleic-acid delivery and targeted gene insertion, in eight
poster presentations at the American Society of Gene & Cell
Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023
at the Los Angeles Convention Center.
“We are pleased to highlight what we believe is
the first iPS cell-derived multi-cell-type therapeutic strategy
designed to mimic how the human immune system naturally fights
disease,” said Matt Angel, Ph.D., Chief Executive Officer and
President of Eterna. “This approach has the potential to form the
foundation of an entirely new class of cell therapies that could
play an important role in treating cancer. We believe that the data
we are presenting this week, combined with our recent acquisition
of Exacis Biotherapeutics’ immuno-oncology platform, position
Eterna to play a leading role in the development of next-generation
cell therapies to treat cancer.”
The study (poster #616) demonstrated a scalable
platform for generating cell therapies comprising multiple immune
cell types, such as NK cells and macrophages, derived from induced
pluripotent stem (iPS) cells reprogrammed with mRNA. In vitro
studies with lymphoid and myeloid cells showed synergistic killing
of SKOV3 ovarian tumor cells. Furthermore, in an effort to improve
how the iPSC-derived myeloid cells could target and engage solid
tumors, the cells were transfected with mRNA encoding a humanized
ROR1-CAR protein. Notably, the ROR1-targeted myeloid cells
demonstrated engagement of ROR1 and cytotoxicity toward SKOV3
ovarian cancer cells.
Christopher Rohde, Ph.D., Co-Founder and Chief
Technology Officer of Factor and member of the Eterna Scientific
Advisory Board, commented, “Our research teams are focused on
developing new approaches to overcome the limitations of existing
cell therapies. The data presented at ASGCT, including potentially
the first iPS cell-derived multi-cell-type therapeutic strategy to
treat cancer, highlights the capabilities of our patented
technology, in-licensed by Eterna, and the potential of this
approach to inform the development of therapies using mRNA cell
engineering.”
Eterna and Factor are presenting additional
research highlighting advances in nucleic-acid delivery and
targeted gene insertion:
Rational Design of Multivalent Ionizable
Lipid Delivery Systems for mRNA Delivery to Blood Cells
(#563) The study aimed
to identify a lipid formulation capable of efficiently transfecting
blood cells, believed to be an ideal target cell for in vivo mRNA
cell engineering. The data presented demonstrates efficient
delivery of mRNA to target blood cells. This platform has the
potential to support numerous ex vivo and in vivo applications of
mRNA delivery, including cell reprogramming, gene editing, and
protein replacement.
Chemically Modified Single-Stranded DNA
Donors Enable Efficient mRNA Gene Editing-Mediated Knock-In in
Human iPS Cells (#1065)
This presentation describes a method for high-yield synthesis of
single-stranded DNA potentially suitable for targeted insertion
applications, including generation of knock-in cell lines. This
platform may have applications in the development of both ex vivo
and in vivo gene-editing therapies.
Efficient Transgene Knock-In in Human
iPS Cells Combined With Small Molecule-Mediated “On-Switch” Yields
Clonal Populations of Engineered Tissue-Specific Cells
(#1103)The study
aimed to identify methods of differentiating engineered iPS cells
to tissue-specific cell populations that uniformly and stably
express a desired protein from a transgene inserted by mRNA gene
editing. The results presented show temporal control of transgene
expression using small molecules during directed differentiation of
iPS cells. This methodology has the potential to support the
development of engineered cell therapies designed to express
therapeutic proteins.
Novel Ionizable Lipids Derived from
2-Hydroxypropylamine and Spermine for mRNA-LNP Delivery
(#1262)The study aimed
to identify novel ionizable lipids capable of formulation in lipid
nanoparticles (LNPs) exhibiting lower mean particle sizes, higher
loading efficiencies, and enhanced protein expression relative to
LNPs incorporating the established lipids ALC0315 or DLin-MC3-DMA.
A library of ionizable lipids containing elements related to
spermine or 2-hydroxypropylamine was developed and evaluated. The
data presented show that the lipids identified may serve as
components of next-generation, LNP-based mRNA and gene
therapies.
Directed Differentiation of Gene Edited
iPSCs by Small-Molecule Inhibition of a Transgene-Encoded Protein
(#1314) The study aimed
to develop a method for generating transgene-expressing
tissue-specific cells derived from engineered iPS cells through the
use of small-molecule inhibition of the transgene-encoded protein
during differentiation. The results presented suggest that
small-molecule inhibition of transgene-encoding proteins may form a
key element of directed differentiation process development for
knock-in iPS cell lines and may be useful for scaled-up
manufacturing of engineered cells.
Gene Editing Proteins with Nickase
Functionality Enable Scarless Targeted Gene Insertion in Primary
Human Cells (#1516) The
study explored the use of UltraSlice™ gene editing proteins
containing cleavage domain variants with nickase functionality for
targeted insertion of donor sequences into defined genomic loci.
Nickases have the ability to create targeted single-strand breaks,
which can favor high-fidelity DNA repair pathways, compared to
double-strand breaks, which can be more prone to error and have
been associated with cytotoxicity and off-target effects. The data
presented demonstrate that the UltraSlice™ nickases enable scarless
insertion of donor sequences into defined genomic loci. This
methodology may have the potential to improve the safety of in vivo
gene insertion by reducing off-target effects of gene-editing
nucleases.
mRNA Cell Engineering Enables Rapid
Prototyping of Macrophage Gene-Editing Strategies for Cancer
Immunotherapy Applications
(#1563)The study
assessed an mRNA-based platform to rapidly evaluate macrophage
engineering approaches for solid tumor applications. A sequence
encoding a CAR targeting ROR1 was inserted into the AAVS1 safe
harbor locus in iPS cells, which were then differentiated into
macrophages. This technology has the potential to enable the rapid
assessment and validation of novel macrophage gene-editing
strategies.
Abstracts are available on the ASGCT Annual
Meeting website here. Copies of the foregoing poster presentations
are available on Eterna’s website located at www.eternatx.com.
About Eterna TherapeuticsEterna
Therapeutics is a life science company committed to realizing the
potential of mRNA cell engineering and thereby providing patients
with transformational new medicines. Eterna has in-licensed a
portfolio of over 120 patents covering key mRNA cell engineering
technologies, including technologies for mRNA cell reprogramming,
mRNA gene editing, the NoveSlice™ and UltraSlice™ gene-editing
proteins, and the ToRNAdo™ mRNA delivery system from Factor
Bioscience. NoveSlice™, UltraSlice™, and ToRNAdo™ are trademarks of
Factor Bioscience. For more information, please visit
www.eternatx.com.
About Factor BioscienceFounded
in 2011, Factor Bioscience develops technologies for engineering
cells to advance the study and treatment of disease. Factor’s
gene-editing technologies enable the precise deletion, insertion,
and repair of DNA sequences in living cells to correct
disease-causing mutations, make cells resistant to infection and
degenerative disease, modulate the expression of immunoregulatory
proteins to enable the generation of durable allogeneic cell
therapies, and engineer immune cells to more effectively fight
cancer. Factor’s cell-reprogramming technologies enable the
generation of clonal lines of pluripotent stem cells that can be
expanded and differentiated into any desired cell type for the
development of regenerative cell therapies. For more information,
visit www.factorbio.com.
Forward-Looking StatementsThis
press release contains forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933, as amended,
and Section 21E of the Securities Exchange Act of 1934, as amended,
which are intended to be covered by the safe harbor provisions of
the Private Securities Litigation Reform Act of 1995.
Forward-looking statements are any statements that are not
statements of historical fact and may be identified by terminology
such as “believe,” “could,” “estimate,” “anticipate,” “expect,”
“plan,” “possible,” “potential,” “project,” “will” or other similar
words and the negatives of such words. Forward-looking statements
are based on current beliefs and assumptions that are subject to
risks and uncertainties and are not guarantees of future
performance. Actual results could differ materially from those
stated or implied in any forward-looking statement as a result of
various factors, including, but not limited to, uncertainties
related to: (i) the evolution of Eterna’s business model into a
platform company focused on mRNA, iPS cell and gene editing
technologies; (ii) Eterna’s ability to successfully,
cost-effectively and efficiently develop its technology and
products; (iii) Eterna’s ability to successfully commence clinical
trials of any products on a timely basis or at all; (iv) Eterna’s
ability to successfully fund and manage the growth of its
development activities; and (v) Eterna ’s ability to obtain
regulatory approvals of its products for commercialization. You
should not rely upon forward-looking statements as predictions of
future events. The forward-looking statements made in this
communication speak only as of the date on which they were made,
and Eterna does not undertake any obligation to update the
forward-looking statements contained herein to reflect events that
occur or circumstances that exist after the date hereof, except as
required by applicable law. Factors that may cause Eterna’s actual
results to differ from those expressed or implied in
forward-looking statements contained in this press release are more
fully disclosed in Eterna’s periodic public filings with the U.S.
Securities and Exchange Commission, particularly under the heading
“Risk Factors” in Eterna’s Annual Report on Form 10-K for the year
ended December 31, 2022, as well as under similar headings in
Eterna’s subsequently filed Quarterly Reports on Form 10-Q and
Current Reports on Form 8-K.
Eterna Therapeutics
Investorsinvestors@eternatx.com
Eterna Therapeutics
MediaEternaPR@westwicke.com
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