Bionano Announces Publication Demonstrating Utility of OGM to Assess Genome Integrity of CRISPR-Edited Cells as Part of Gene Therapy Development
29 Novembre 2023 - 4:04PM
Bionano Genomics, Inc. (Nasdaq: BNGO) today announced a
publication demonstrating the use of optical genome mapping (OGM)
to identify structural variations (SVs) introduced by CRISPR-Cas9
gene editing of CD4+ T-cells that could potentially limit the
therapeutic use of such edited cells.
The research study authors used OGM in combination
with drop-off digital PCR (ddPCR) to assess genome integrity of
cells that were edited by CRISPR to restore gene function in Hyper
IgM1 (HIGM1), an X-linked combined immunodeficiency caused
by CD40LG mutations which can lead to liver disease,
cancer and infections. The study authors describe that while ddPCR
assays are suitable to characterize certain expected mutations,
they may miss large SVs that are either outside the regions
targeted by ddPCR primers or are too big to be detected by
ddPCR.
The study authors first validated OGM’s performance at high
coverage to capture unexpected gene editing events and found that
it outperformed karyotyping’s resolution and sensitivity. OGM then
revealed the presence of concameters missed by the ddPCR assay,
indicating that 20-50% of the edited cells expressing the rescued
gene did not undergo precise editing. In addition, OGM detected 11
nonrecurring SVs outside of the target locus, demonstrating its
genome-wide ability to detect off-target events. The study authors
concluded that using OGM early in the development of the gene
therapy could enable researchers to reduce the number of ddPCR
assays needed in the final product quality control panel.
“This study highlights how researchers can use OGM when
developing cell and gene therapies. The expansion of gene therapy
faces risks due to both on-target and off-target structural
variations that may be introduced during CRISPR-Cas9 genome
editing. Since genome aberrations caused by CRISPR-Cas9
editing could lead to unforeseen adverse effects, we believe
careful and comprehensive analysis of edited genomes is important
for quality control while developing these therapies and their
manufacture," commented Erik Holmlin, PhD, president and chief
executive officer of Bionano.
The paper is available at:
https://www.embopress.org/doi/full/10.15252/embj.2023114188.
About Bionano
Bionano is a provider of genome analysis solutions that can
enable researchers and clinicians to reveal answers to challenging
questions in biology and medicine. The Company’s mission is to
transform the way the world sees the genome through OGM solutions,
diagnostic services and software. The Company offers OGM solutions
for applications across basic, translational and clinical research.
Through its Lineagen, Inc. d/b/a Bionano Laboratories business, the
Company also provides diagnostic testing for patients with clinical
presentations consistent with autism spectrum disorder and other
neurodevelopmental disabilities. The Company also offers an
industry-leading, platform-agnostic software solution, which
integrates next-generation sequencing and microarray data designed
to provide analysis, visualization, interpretation and reporting of
copy number variants, single-nucleotide variants and absence of
heterozygosity across the genome in one consolidated view. The
Company additionally offers nucleic acid extraction and
purification solutions using proprietary isotachophoresis
technology. For more information, visit www.bionano.com,
www.bionanolaboratories.com or www.purigenbio.com.
Unless specifically noted otherwise, Bionano’s OGM products
are for research use only and not for use in diagnostic
procedures.
Forward-Looking Statements of Bionano
Genomics
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Words such as “believe,” “can,” “could,” and
similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) convey
uncertainty of future events or outcomes and are intended to
identify these forward-looking statements. Forward-looking
statements include statements regarding our intentions, beliefs,
projections, outlook, analyses or current expectations concerning,
among other things: the potential utility of OGM in identifying
on-target and off-target structural variations introduced by
CRISPR-Cas9 gene editing; the utility and ability of OGM to be used
as part of developing cell and gene therapies and the manufacture
thereof; the importance of comprehensive analysis of edited genomes
in order to develop cell and gene therapies and their manufacture;
and our ability to drive adoption of OGM and our technology
solutions to be used as part of developing cell and gene therapies
and the manufacture thereof. Each of these forward-looking
statements involves risks and uncertainties. Actual results or
developments may differ materially from those projected or implied
in these forward-looking statements. Factors that may cause such a
difference include the risks and uncertainties associated with: the
timing and amount of revenue we are able to recognize in a given
fiscal period; the impact of adverse geopolitical and macroeconomic
events, such as recent and potential future bank failures, global
pandemics and the ongoing conflicts between Ukraine and Russia and
Israel and Hamas, on our business and the global economy; general
market conditions; changes in the competitive landscape and the
introduction of competitive technologies or improvements to
existing technologies; changes in our strategic and commercial
plans; our ability to obtain sufficient financing to fund our
strategic plans and commercialization efforts and our ability to
continue as a “going concern”; the ability of medical and research
institutions to obtain funding to support adoption or continued use
of our technologies; study results that differ or contradict the
results mentioned in this press release; failure of OGM to prove
useful in uncovering SVs in CRISPR-Cas9 edited genomes; failure of
OGM to be used as part of developing cell and gene therapies and
their manufacture; failure of and the risks and uncertainties
associated with our business and financial condition in general,
including the risks and uncertainties described in our filings with
the Securities and Exchange Commission, including, without
limitation, our Annual Report on Form 10-K for the year ended
December 31, 2022 and in other filings subsequently made by us with
the Securities and Exchange Commission. All forward-looking
statements contained in this press release speak only as of the
date on which they were made and are based on management’s
assumptions and estimates as of such date. We do not undertake any
obligation to publicly update any forward-looking statements,
whether as a result of the receipt of new information, the
occurrence of future events or otherwise.
CONTACTSCompany Contact:Erik
Holmlin, CEOBionano Genomics, Inc.+1 (858)
888-7610eholmlin@bionano.com
Investor Relations:David HolmesGilmartin
Group+1 (858) 888-7625IR@bionano.com
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