Bionano Announces Three Publications Demonstrating OGM’s Utility for Cell and Gene Therapy
Bionano Genomics, stock symbol BNGO, announced three new studies that support the use of Optical Genome Mapping (OGM) in cell and gene therapy development. These studies demonstrate OGM's ability to detect genome integrity and structural variations in therapeutic cell lines like CAR-T cells, stem cells, and hiPSCs. The first study from Niño Jesús University Children's Hospital showed OGM's efficacy in assessing genotoxicity in CAR-T cells. The second study from Ruhr-University Bochum demonstrated OGM's utility in evaluating genome integrity post-CRISPR/Cas9 editing for Alzheimer's research. The third study from Janssen explored OGM's role in confirming the absence of off-target effects in stem cells edited for the APOE genotype, associated with Alzheimer's disease.
- OGM shows high sensitivity and resolution in detecting genome integrity.
- Three new studies support OGM's application in cell and gene therapy.
- OGM can reveal important structural variations that other techniques might miss.
- Niño Jesús University Children's Hospital study confirmed OGM's utility in evaluating CAR-T cell safety.
- Ruhr-University Bochum study highlighted OGM's effectiveness in assessing CRISPR/Cas9-edited hiPSCs.
- Janssen study demonstrated OGM's role in confirming gene edits without off-target effects.
- OGM is valuable for comprehensive genome-wide quality assessment in genetic engineering.
- Potential pathogenic implications were found in one donor source using OGM.
- OGM revealed multiple aberrations affecting numerous genes in edited hiPSCs.
- Edited hiPSCs showed potential neuroectodermal differentiation impairment despite mild phenotypic changes.
Insights
The recent studies show optical genome mapping (OGM)'s valuable role in ensuring genome integrity in cell and gene therapy, a critical consideration as genetic treatments become more sophisticated. OGM’s ability to detect structural variants (SVs) with higher sensitivity and resolution compared to traditional methods is noteworthy. For instance, in the Niño Jesús University Children’s Hospital study, OGM identified a potential pathogenic variant in CAR-T cell products, which could impact the therapy’s safety profile.
In the case of the Ruhr-University Bochum study, OGM effectively confirmed on-target edits and detected genomic alterations post-CRISPR/Cas9 editing, suggesting its utility in quality control processes. This is important as gene editing techniques, while promising, can introduce unintended genomic changes that may affect cell functionality.
Finally, the Janssen study demonstrates OGM’s capability in confirming the absence of off-target effects in newly created stem cell lines, ensuring the reliability of the research conclusions. This contributes to a more robust understanding of how genetic modifications could potentially influence disease outcomes, specifically in Alzheimer’s research.
Overall, these studies underscore OGM’s potential to enhance the safety and efficacy of cell and gene therapies by providing a more comprehensive genomic landscape. For investors, this strengthens Bionano's positioning in the genomics sector, potentially driving future growth as the demand for precise genetic analysis tools increases.
The publication of these studies bolsters Bionano Genomics' (BNGO) strategic positioning in a competitive market. With the demonstrated utility of OGM in high-stakes applications such as CAR-T cell therapy and Alzheimer’s disease research, Bionano is aligning itself with emerging, high-growth sectors in biotechnology.
From a financial perspective, these findings could positively impact Bionano’s revenue streams by attracting more academic and commercial clients who require high-precision genome mapping. Additionally, the involvement of reputable institutions like Niño Jesús University Children’s Hospital and Janssen adds credibility to Bionano's technology, potentially leading to more partnerships and collaborations.
For retail investors, this data suggests a strong growth trajectory for Bionano, driven by market demand for advanced genomic tools. However, investors should also be aware of the broader market dynamics and competitive landscape, where other technologies might emerge with similar or superior capabilities.
- Three peer-reviewed publications collectively illustrate the building support for optical genome mapping (OGM) as a tool for cell and gene therapy development that allows researchers in academic medical centers and biopharmaceutical companies to assess genome integrity in therapeutic cell lines like chimeric antigen receptor-modified T cells (CAR-T), stem cells, and cell lines used for translational research in unraveling genetic causes of Alzheimer’s disease
- Taken together, the publications illustrate that OGM can be highly sensitive for genome-wide detection of on and off target effects that may limit the utility of stem cell lines and CAR-T cell products
SAN DIEGO, May 21, 2024 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (BNGO), today announced the publication of three studies which collectively illustrate the continued development of data supporting the utility of optical genome mapping (OGM) in cell and gene therapy applications. In all three publications, the authors note OGM’s potential to provide a comprehensive landscape of genome structure and to assess genome integrity due to the workflow’s ability to detect structural variants (SVs) with higher sensitivity and resolution than standard cytogenetic techniques.
Key Findings and Takeaways
In a publication from Niño Jesús University Children's Hospital (García-García et al.), researchers analyzed different strategies to improve the efficacy of chimeric antigen receptors (CARs) in solid tumors, focusing on the characterization of a new strategy to target neuroblastoma, based on the combination of a monoclonal antibody and fluorescein isothiocyanate (FITC) and anti-FITC CAR-T cells. Following an FDA announcement of an investigation into the serious risk of T-cell malignancy following CAR-T cell immunotherapies, the authors then evaluated the risk of genotoxicity of the CAR-T cell products, using OGM to study genomic integrity and confirm the safety of ex vivo culture and transduction of T cells. The authors noted that OGM offered a comprehensive landscape of genome structure in CAR-T cells, due to the higher sensitivity and resolution that the workflow can provide when compared to traditional methods of analysis.
- OGM analyzed PB, 45RA and CB CAR-T products after 8 days of culture and compared with T cells from donor samples at day 0 to look for new variants, including aneuploidies
- OGM revealed a single event with potential pathogenic implications in one of the analyzed donor sources, as well as additional variants of uncertain significance
The publication from Ruhr-University Bochum (Gallego Villarejo et al.) covered the use of OGM following gene editing to assess the genome integrity of two CRISPR/Cas9-edited hiPSC lines used for studying Alzheimer’s disease. Researchers used OGM to identify on-target editing and to detect genomic alterations that might have neuroectodermal differentiation impairment. Study results confirmed the vulnerability of genomic DNA to gene editing and highlighted the utility of OGM for genome-wide quality assessment of genetic engineering.
- OGM revealed multiple aberrations that affected a large number of genes but were found to have mild impact on the ability of hiPSCs to develop cerebral organoids
- Edited hiPSCs were not found to have major phenotypic changes but one edited cell line showed potential neuroectodermal differentiation impairment
- OGM confirmed on-target edits and did not detect off-target edits
The publication from Janssen (Haidar et al.) details research into the Apolipoprotein E (APOE) genotype, which is the strongest risk factor for late-onset Alzheimer’s disease (AD). A rare version of the APOE gene, called Christchurch, may help protect against Alzheimer’s in people with a strong genetic risk for the disease. In the study, researchers describe the creation of new stem cell lines, available via the European Bank of iPSCs, where the Christchurch mutation was added to three common versions of the APOE gene to study how APOE variants impact AD and other genetic conditions. OGM was used to assess genome integrity and the data were used by researchers to support their conclusions that gene edits did not result in off-target effects.
- OGM confirmed the absence of off-target effects above 500 bp in the cell lines
“These studies highlight 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 genome editing. Since genome aberrations caused by gene editing could obscure the true conclusions in translational research studies or even 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.
To learn more about OGM’s utility in cell and gene therapy applications, please visit this website.
The publication from García-García et al. can be found here; the publication from Gallego Villarejo et al. can be found here; the publication from Haidar et al. can be found here.
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, and nucleic acid extraction and purification solutions using proprietary isotachophoresis (ITP) technology. Through its Lineagen, Inc. d/b/a Bionano Laboratories business, the Company also provides OGM-based testing for certain laboratory developed tests. 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. For more information, visit www.bionano.com and www.bionanolaboratories.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
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believe," “can,” “may,” “offer," "potential,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances and the negatives thereof) 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 performance of OGM compared to traditional cytogenetic methods in applications for cell and gene therapy development to measure genome integrity in therapeutic cell lines including, chimeric antigen receptor-modified T cells (CAR-T), stem cells, and cell lines used for translational research in unraveling genetic causes of Alzheimer’s disease; the utility of OGM in detecting on and off target events in cell and gene therapy applications described in this press release; the ability and utility of OGM to detect SVs introduced during gene editing; the ability and utility of OGM to assess genome integrity; and other statements that are not historical facts.
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 impact of geopolitical and macroeconomic developments, such as the ongoing conflicts between Ukraine and Russia and Israel and Hamas, and related sanctions, global and regional pandemics, on our business and the global economy; general market conditions, including inflation and supply chain disruptions; challenges inherent in developing, manufacturing and commercializing products; our ability to further deploy new products and applications and expand the markets for our technology platforms; failure of OGM to detect SVs compared to traditional cytogenetic methods in applications for cell and gene therapy development to measure genome integrity in therapeutic cell lines including, chimeric antigen receptor-modified T cells (CAR-T), stem cells, and cell lines used for translational research in unraveling genetic causes of Alzheimer’s disease; the failure of OGM to detect on and off target events in cell and gene therapy applications described in this press release; the failure of OGM to detect SVs introduced during gene editing; the failure of OGM to properly assess genome integrity; the failure of OGM to detect SVs consistent with the study results described in this press release; future study results that contradict the study results described in this press release; future study results that do not support the study results described in this press release; our expectations and beliefs regarding future growth of the business and the markets in which we operate; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts and to continue as a "going concern”; and 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, 2023 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 are under no duty to update any of these forward-looking statements after the date they are made to conform these statements to actual results or revised expectations, except as required by law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date the statements are made. Moreover, except as required by law, neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements contained in this press release.
CONTACTS
Company Contact:
Erik Holmlin, CEO
Bionano Genomics, Inc.
+1 (858) 888-7610
eholmlin@bionano.com
Investor Relations:
David Holmes
Gilmartin Group
+1 (858) 888-7625
IR@bionano.com
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