GenEditBio Scientists Unveil GenomePAM: An Innovative and Scalable Platform Designed with AI Integration to Accelerate the Development of Next-Generation Genome Editors for Therapeutic Use
[August 13, 2025] – GenEditBio Limited (“GenEditBio”), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic solutions through the discovery of novel and precise Cas nucleases and the development of safe and efficient cargo delivery platforms, today announced GenomePAM, an innovative and scalable platform with artificial intelligence (AI) integration, designed to accelerate the discovery, characterization and engineering of novel CRISPR-Cas—based genome editors for therapeutic applications. The research, led by GenEditBio scientists, was conducted in collaboration with the City University of Hong Kong, Karolinska Institutet, St. Jude Children’s Research Hospital, and Massachusetts General Hospital & Harvard Medical School. The findings have been published online in the prestigious international biomedical journal Nature Biomedical Engineering.
CRISPR-Cas systems, nature’s antiviral defence mechanism in bacteria, have been repurposed as powerful genome-editing tools with far-reaching therapeutic applications in genome editing therapy. However, discovering new CRISPR-Cas enzymes and optimizing them for therapeutic use remains slow and labour-intensive. A key hurdle is characterizing the short DNA sequences called protospacer adjacent motifs, or PAM in short, that these enzymes require to recognize and bind their target DNA.
"CRISPR-Cas genome editing involves engagement before commitment, but traditional methods to identify PAMs (the first step engagement) are slow, costly, and laborious, yielding only 5- to 6-bit width. Our innovative GenomePAM platform overcomes these limitations, achieving at least 10-bit width using naturally occurring and highly repetitive DNA sequences in human cells," said Zongli ZHENG, PhD, the corresponding author of the study and Co-Founder and Chairman of GenEditBio. "By integrating GenomePAM with the AI model AlphaFold 3, we discovered an extensive collection of new Cas nucleases with enhanced PAM selectivity. This breakthrough offers a new strategy for developing customizable next-generation genome editors."
“GenomePAM represents a transformative leap in CRISPR-Cas characterization, born from years of pioneering work by our world-class team of scientists and bioinformaticians," added Tian ZHU, PhD, Co-Founder and CEO of GenEditBio. "This platform uniquely combines high-fidelity cellular data with AI-powered protein engineering to do more than just streamlining workflows—we’re fundamentally redefining what's possible in therapeutic genome editing. GenomePAM cuts development timelines dramatically while simultaneously enhancing the precision and safety of our genome editors."
Read full research article (open access): https://www.nature.com/articles/s41551-025-01464-y
About GenEditBio
Established in 2021 and headquartered in Hong Kong, China, GenEditBio is a gene therapy start-up company with an overarching strategic goal of providing potentially curative, once-and-done and programmable in vivo genome editing-based therapeutic solutions (dubbed “DNA surgery”) with high safety profile, unmatched precision, and affordable access for genetic diseases with unmet needs. The Company’s core areas of focus include novel Cas nuclease discovery and safe and efficient cargo delivery utilizing lipid nanoparticle (LNP) and engineered protein delivery vehicle (PDV). We have research laboratories and supporting offices in Hong Kong, Beijing, and Boston. GenEditBio is financially backed by top-tier life science investors, including Qiming Venture Partners, Fangyuan Capital, Center Biotherapeutics, Lumosa Therapeutics, HKSTP Venture Fund, and others. For more information, please visit www.geneditbio.com.
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Featured are research team members including (from left) Mr Miao YU, the first author of the study and a doctoral candidate at the Department of Biomedical Sciences of City University of Hong Kong (CityUHK) and previously a Research Assistant at Ming Wai Lau Centre for Reparative Medicine (MWLC) of Karolinska Institutet (KI); Dr Bang WANG, Principal Scientist of GenEditBio and previously a postdoctoral fellow at MWLC of KI; Ms Limei AI, a doctoral candidate at the Department of Biomedical Sciences of CityUHK and previously a Research Assistant at MWLC of KI; and Prof Zongli ZHENG, the corresponding author of the study and Co-Founder and Chairman of GenEditBio.
