Envisagenics Validates AI/ML Approach for RNA Target Identification and SSO Therapeutic Development

Results from evaluation of the company's SpliceCore^® AI/ML platform in Triple Negative Breast Cancer published in Molecular Systems Biology

NEW YORK, April 25, 2024 /PRNewswire/ -- Envisagenics, an AI-driven biotechnology company, today announced the publication in the journal Molecular Systems Biology of study results evaluating the company's SpliceCore AI/ML platform in Triple Negative Breast Cancer (TNBC). This study demonstrates the efficacy of artificial intelligence and machine learning (AI/ML) for target discovery in triple negative breast cancer (TNBC) and for identifying functional and verifiable splice-switching oligonucleotides (SSOs) crucial for the development of RNA therapeutics. The results also validate its potential to tackle a challenging disease such as TNBC, a particularly aggressive cancer affecting approximately 200,000 patients annually, with a dismal five-year survival rate of only 20%. Detailed results from the study, titled "Development and Validation of AI/ML Derived Splice-Switching Oligonucleotides," are available here.

SSOs are synthetic antisense oligonucleotide compounds that directly act on pre-mRNA to regulate the expression of alternatively spliced isoforms unique to cancer cells, which are key drivers of cancer progression and metastasis. While holding immense promise as a therapeutic approach for impeding cancer growth, the identification of functional SSOs using traditional methods is high cost and requires extensive time and labor.

"This study bridges the gap between computational predictions and experimental validation, positioning AI/ML as a critical force in validating RNA targets and advancing SSO therapeutic development," said Martin Akerman, Ph.D., Envisagenics' CTO and Co-Founder.

In the study, Envisagenics achieved the following milestones:

• Utilized its proprietary platform, SpliceCore, to identify novel therapeutic targets along with their corresponding modulatory SSOs, and the specific Splicing Factors affected in pre-mRNA by these SSOs.
• Conducted a retrospective validation of the SpliceCore algorithm using known functional SSOs.
• Validated a previously unidentified target in triple negative breast cancer (TNBC), NEDD4L exon 13 (NEDD4Le13), discovered through the SpliceCore platform.
• Illustrated the efficacy of targeting NEDD4Le13 with an AI/ML-designed SSO, showcasing its ability to attenuate the proliferative and migratory tendencies of TNBC cells, through downregulation of the transforming growth factor beta (TGFβ) pathway, a pivotal player in tumor invasion and metastasis.
• Discovered a novel mechanism of TGFβ pathway regulation through alternative splicing in cancer.

"For patients with TNBC and other diseases that are difficult to treat, this study illustrates the usefulness of SpliceCore to discover novel therapeutic targets from patient's RNA sequencing data," said Dr. Akerman. "Our findings affirm the robustness and reliability of the platform and shed light on previously unrecognized avenues for therapeutic intervention."

About SpliceCore

SpliceCore is Envisagenics' proprietary AI/ML platform that enables the translation of vast amounts of RNA sequencing data into novel therapeutics. SpliceCore's versatility extends to both the discovery of novel SSOs and splicing-derived neoepitopes for advanced immunotherapy applications.

About Envisagenics

Envisagenics is an AI-driven biotechnology company harnessing the therapeutic potential of RNA splicing. Over 95% of human genes undergo alternative splicing, a process generating multiple RNA isoforms from a single gene. Alternative splicing holds the key to understanding and targeting over 370 diseases, including cancer and neurodegenerative disorders. SpliceCore boasts an impressive 400-fold increase in transcriptomic search space compared to conventional tools for target identification. Leveraging our extensive map of over 14 million RNA splicing events, SpliceCore enables the discovery of disease-specific targets, paving the way for the rational design and development of RNA therapeutics and immunotherapies.

Envisagenics collaborates closely with biopharmaceutical companies and academic institutions to elevate their drug discovery capabilities and have previously partnered with the Lung Cancer Initiative at Johnson & Johnson, Biogen, and BMS. Founded in 2014 as a spin-out from Cold Spring Harbor Laboratory, we take pride in being a woman- and minority-led organization, with several grants from esteemed institutions like the National Institute of General Medical Sciences and the National Cancer Institute.

For more information on our groundbreaking work, connect with us on Twitter and LinkedIn or visit https://www.envisagenics.com

Media Contact

Kathryn Morris, Ph.D.

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SOURCE Envisagenics, Inc.