GenEditBio Co-Founder and Chairman Shares Expert Insights on Therapeutic Genome Editing in Exclusive PharmaBoardroom Interview: The Journey from Technology Innovator to Therapeutic Pioneer
[June 4, 2025] – GenEditBio Limited (“GenEditBio”), a biotechnology start-up company focusing on genome-editing therapeutic solutions through the discovery of novel Cas nucleases and the development of safe and efficient cargo delivery platforms, today announced that its Co-Founder and Chairman, Zongli ZHENG, PhD, was featured in an exclusive, in-depth interview with PharmaBoardroom, a global digital publishing platform specializing in healthcare insights for leaders.
In the interview, Dr Zheng traced his scientific journey in therapeutic genome editing from Boston to Hong Kong, and explained how this experience has shaped the company’s transformative evolution from technology innovator to therapeutic pioneer. He further highlighted the company's strengths on in vivo genome editing, and outlined the company's strategies to provide fundamentally safe, efficacious and affordable in vivo genome editing therapies to treat genetic diseases with unmet needs.
An excerpt from the interview is reproduced below with permission:
What advantages does in vivo genome editing offer over traditional methods, and why is it central to your therapeutic approach?
In vivo genome editing holds transformative clinical potential due to its simplicity, versatility, and ability to address disease at its genetic root. Unlike ex vivo techniques, which require harvesting, modifying, and reinfusing cells, in vivo approaches deliver editing components directly into the patient’s body, allowing targeted correction within the desired tissue. While this streamlines treatment, it also raises specific regulatory considerations. Most importantly, ensuring that edits are confined to somatic cells and do not affect the germline. Agencies such as the FDA remain particularly vigilant about these safety parameters.
From a scientific perspective, in vivo editing offers two major advantages. First, the platform is inherently programmable. By altering a short guide sequence, the same technology can be tailored to target different genes, enabling potential applications across thousands of rare genetic disorders with high unmet need. Just as significantly, it supports a “one-and-done” paradigm in which a single intervention corrects the underlying mutation at the DNA level, eliminating the need for ongoing treatment. For many chronic or life-limiting conditions, this capacity to resolve disease at its source represents a profound shift in how therapeutics are conceived and delivered.
Also, I would like to emphasise that confidence in the field was significantly bolstered by the FDA approval of the first CRISPR-based therapy in 2023, manufactured by Vertex Pharmaceuticals for the treatment of sickle cell disease. Notably, a key safety assay referenced during the FDA’s evaluation was GUIDE-seq, a genome-wide off-target detection method that I co-developed while in Boston. Its incorporation into the regulatory framework underscored both the maturity of the field and the value of robust analytical tools in supporting clinical translation.
What are your lead therapeutic programmes, and how are you approaching early clinical development?
Our lead candidate, currently advancing toward clinical evaluation, targets an ocular disease and represents the most mature programme within a pipeline of five therapeutic assets. The initial trial will be conducted in China under the investigator-initiated trial mechanism – a regulatory pathway that enables early-stage safety and efficacy testing ahead of formal Investigational New Drug filings with global agencies such as the FDA. The study will enrol approximately ten patients in its first year. Ocular indications provide a practical and clinically relevant entry point for in vivo genome editing, given the accessibility of the tissue and the ability to directly observe therapeutic effects.
This programme leverages a proprietary delivery vehicle: a non-replicative viral vector engineered to transport a ribonucleoprotein editing complex directly to the target tissue. Unlike mRNA-based approaches, which require intracellular translation before therapeutic action can occur, our protein-based system is active immediately upon delivery. This eliminates translational delays, reduces molecular instability, and removes the need for chemically modified guide RNAs, which can pose manufacturing and regulatory challenges. By correcting the genetic defect responsible for pathogenic protein accumulation, we aim to achieve a durable, one-time intervention capable of restoring and preserving vision.
What long-term strategy is guiding GenEditBio’s evolution from a technology platform to a therapeutic developer?
GenEditBio is intentionally evolving as both a technology platform company and a therapeutic developer, reflecting a model successfully pursued by several pioneering biotech firms in the US. With a proprietary genome editing system at its core and a growing portfolio of therapeutic programmes, the company is well-positioned to pursue multiple strategic avenues. While no singular exit strategy has yet been defined, we are carefully weighing options that include out-licensing select assets as well as advancing internal candidates through early and mid-stage clinical development. Our discussions with leadership and investors have centred on demonstrating the platform’s translational potential, and to that end, we are particularly focused on progressing our lead ocular programme into Phase I/II trials. This asset, which combines high potency with no observable off-target effects in preclinical animal studies, has the potential to serve as a benchmark for in vivo genome editing, a space that remains largely clinically unvalidated despite increasing momentum globally. In this context, GenEditBio aims not only to develop novel disease-modifying therapies but also to help define the standards for safety and precision that will shape the future of the field.
The full interview transcript is available on PharmaBoardroom:
https://pharmaboardroom.com/interviews/zongli-zheng-co-founder-and-chair-geneditbio/
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 other investors.
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