Our Scientific Approach
At Genexys BioMed, our research is driven by a commitment to innovation and patient impact.
Genexys BioMed is pioneering a next-generation, non-viral, non-LNP gene therapy platform called GeneZips, designed to safely and effectively deliver large genetic payloads while minimizing immune response. This groundbreaking technology enables repeat dosing and long-term gene expression, overcoming the limitations of traditional viral vectors and lipid nanoparticles. Our goal is to develop mutation-agnostic, re-dosable genetic medicines that provide lasting therapeutic benefits without the risks typically associated with current delivery methods, paving the way for transformative treatments for a wide range of genetic disorders.
Why GeneZips?
Next-Generation Delivery – A non-viral, non-LNP polymer-based nucleic acid compaction platform reshaping genetic medicine.
Exceptionally Safe, Redosable, and Long-Lasting – Low immunogenicity enables effective repeat and repeated delivery of large genetic payloads with durable gene expression for sustained efficacy.
Precision Targeting – Platform allows engineering for cell- and tissue-specific delivery.
Unmatched Payload Capacity – Overcomes size limitations that hamper other genetic therapies.
Unparalleled Versatility – Seamlessly delivers mRNA, DNA, and gene editing systems such as CRISPR/Cas9.
Innovative R&D and Strategic Collaborations
At Genexys BioMed, we are pioneering non-viral, non-lipid gene delivery decades of research in non-viral gene delivery, leveraging decades of cutting-edge research and strategic partnerships to accelerate development and prepare for first-in-human studies.
Built on Breakthrough Science – Founded at Cincinnati Children’s Hospital, a global leader in CF research and gene therapy.
Fueled by Innovation – Backed by Orange Grove Bio, a premier biotech investment firm dedicated to transforming academic discoveries into breakthrough therapeutics.
Supported by the NIH – Secured an NIH SBIR Phase I grant to drive preclinical validation.
Strategic Partnerships – Collaborating with leading experts to optimize manufacturing, delivery, and regulatory strategy for rapid clinical advancement.
Our Pipeline
Our lead program, GEN-023, is designed to restore CFTR function in all CF patients, including those unresponsive to current modulators. Preclinical studies have demonstrated:
Sustained CFTR gene expression for up to 40 days.
Strong preclinical safety profile, with no adverse immune reactions.
Scalable platform with potential applications in other large-gene diseases, such as neurological disorders.
With first-in-human trials on the horizon, Genexys BioMed is poised to bring life-changing gene therapies to patients with CF– and beyond.
