First-in-Class, Targeted Nucleic Acid Therapeutics

Gennao Bio is developing first-in-class, targeted nucleic acid therapeutics utilizing its proprietary, non-viral GMAB platform technology. GMAB is an adaptive technology that uses a cell-penetrating antibody to non-covalently bind to and deliver therapeutic levels of a wide variety of nucleic acid payloads to select cells. We are developing this delivery system with an initial focus in oncology and rare monogenic skeletal muscle diseases.

Technology

Our gene monoclonal antibody (GMAB) technology has several unique attributes that enable it to systemically deliver multiple types of nucleic acids, directly into the cell, while preserving therapeutic function.

1

Adaptive and flexible

Our cell-penetrating antibody non-covalently binds to a broad range of nucleic acid payloads including single-stranded/double-stranded RNA, mRNA, synthetic RNA, DNA, RNAi and ASOs. Additionally, GMAB complexes have demonstrated the ability to be delivered into both the cytoplasm and the nucleus.

2

Targeted delivery

GMAB complexes target the nucleoside transporter ENT2, which is highly expressed on tumors cells and skeletal muscle. Specificity for ENT2 allows for better targeting toward relevant cell types while minimizing off-target effects.

3

Preserves therapeutic function

In preclinical studies, GMAB complexes have demonstrated the ability to protect a wide range of nucleic acid payloads including mRNA after systemic delivery thereby preserving structural integrity and biological function of the payload.

GMAB Technology Advantages

Our GMAB technology has the potential to transform genetic medicine by offering meaningful advantages over viral & other antibody-targeted nucleic acid delivery systems.

Broad-based, Systemic Delivery

Non-covalently binds to multiple types of nucleic acid payloads

Delivery of gene-editing molecules

Targeted Cell Transduction

Selective targeting of ENT2

Cell penetration into cytoplasm and nucleus

Dosing Flexibility

Repeat dosing with ability to escalate and titrate dose

Dosing possible regardless of age, weight and medical condition

Improved Safety Profile

Devoid of complement-mediated immune response

Clinical data suggest GMAB is safe and well tolerated

Utilizes mAb Manufacturing Process

Established, cost efficient, scalable and reproducible

No linkers needed

Team

Stephen Squinto, Ph.D.

Position: Co-Founder, Chief Executive Officer, Chair of the Board

Dr. Squinto is an entrepreneur and biotechnology industry veteran with more than 30 years of experience in drug development. He is an executive partner at OrbiMed Advisors, and most recently served as interim chief executive officer at Passage Bio, Inc. Prior to that, he co-founded Alexion Pharmaceuticals, Inc. and served as its executive vice president and chief global operations officer. Earlier in his career, Dr. Squinto held various positions at Regeneron Pharmaceuticals, Inc. and joint academic positions at the Tulane University and LSU Medical Schools.  He is a recipient of numerous honors and awards from academic and professional organizations for his scientific work. Dr. Squinto received his B.A. in Chemistry and Ph.D. in Biochemistry and Biophysics from Loyola University of Chicago.

Dr. Squinto is an entrepreneur and biotechnology industry veteran with...

Stephen Squinto, Ph.D.

Co-Founder, Chief Executive Officer, Chair of the Board

Chris Duke

Position: Chief Operating Officer

Mr. Duke is an accomplished biotech executive with more than 15 years of global commercial, operational and clinical development experience. He has held leadership positions at several rare disease and immune-oncology companies including Amicus Therapeutics, Advaxis, Inc., and NPS Pharma. Most recently, Mr. Duke led operations and the commercial team in Japan for Amicus Therapeutics, after previously serving as the company’s vice president of global commercial operations. He also was chief operating officer for Advaxis, Inc., and executive director of international commercial operations for NPS Pharma. Earlier in his career, Mr. Duke held clinical and scientific positions at Merck & Co., and The Schering-Plough Corporation. He received a MBA from the Wharton School at the University of Pennsylvania, and an MPH and B.S. in Chemical Engineering from Rutgers University.

Mr. Duke is an accomplished biotech executive with more than...

Chris Duke

Chief Operating Officer

Dale L. Ludwig, Ph.D.

Position: Chief Scientific Officer

Dr. Ludwig is a recognized leader in the biopharmaceutical industry, having supported the development and successful launch of several biologic oncology products including Erbitux®, Cyramza™, Portrazza®, and Lartruvo™, as well as the clinical advancement of a number of other therapeutic antibodies over the course of his career. Most recently, he served as the chief science and technology officer for Actinium Pharmaceuticals. Prior to Actinium, Dr. Ludwig served as the chief scientific officer, vice president, of oncology discovery research – biologics technology and a member of the oncology research senior leadership team at Eli Lilly and Company. Prior to the acquisition of ImClone by Eli Lilly, he held the position of head of molecular & cellular engineering at ImClone Systems Incorporated. Dr. Ludwig trained as a postdoctoral associate in the DNA Damage and Repair Group of the Los Alamos National Laboratory and as a postdoctoral fellow in the Department of Molecular Genetics, Biochemistry and Microbiology at the University of Cincinnati College of Medicine. He holds a B.S. in biology with a concentration in microbiology from James Madison University and received his Ph.D. in Microbiology from East Carolina University.

Dr. Ludwig is a recognized leader in the biopharmaceutical industry,...

Dale L. Ludwig, Ph.D.

Chief Scientific Officer

Claudine Prowse, Ph.D.

Position: Chief Financial Officer

Dr. Prowse is an accomplished leader in the healthcare industry with more than 20 years of experience at small, mid and large cap biotechnology companies and investment banks. Most recently, Dr. Prowse served as senior vice president of strategy and corporate development for Rocket Pharmaceuticals, Inc., where she provided leadership and execution across financing transactions and other strategic initiatives contributing to the company’s successful growth. Prior to this role, she was vice president of strategy, corporate development and investor relations officer at Inotek Pharmaceuticals Corporation, where she was instrumental in driving the strategy and leading the merger transaction with Rocket Pharmaceuticals, Inc. Dr. Prowse served in similar strategy and investor relations roles at Biogen, Inc., Human Genome Sciences and Ionis Pharmaceuticals, Inc., as well as vice president at Leerink Swann. She holds a Ph.D. in Biomedical Sciences from the University of California, San Diego (UCSD) School of Medicine and a B.S. in Biomedical Engineering from the UCSD School of Engineering.

Dr. Prowse is an accomplished leader in the healthcare industry...

Claudine Prowse, Ph.D.

Chief Financial Officer

Bruce Turner, M.D., Ph.D.

Position: Co-Founder, Chief Clinical Advisor

Dr. Turner is a medically trained radiation oncologist with deep experience transcending genetic research, drug development and healthcare portfolio management. Since 2017, Dr. Turner has served as Managing Director at Boxer Capital of Tavistock Group. He also spent five years at Hoffman-La Roche in various positions and helped lead the re-organization of the cardiovascular franchise among other responsibilities. Prior to joining Roche, Dr. Turner was vice president of Ionis Pharmaceuticals, Inc. working in cardiovascular drug development and business development. Earlier in his career, he served as a healthcare equities portfolio manager at Bank of America Merrill Lynch and Diker Management. Dr. Turner received a M.D. from the University of Pennsylvania School of Medicine, Ph.D. in Molecular Biology from the University of Pennsylvania School of Medicine, and B.A. degree in biology from the University of Chicago. He completed his postgraduate medical training at Yale-New Haven Hospital in Radiation Oncology and performed postdoctoral research at Yale University School of Medicine in the Department of Genetics.

Dr. Turner is a medically trained radiation oncologist with deep...

Bruce Turner, M.D., Ph.D.

Co-Founder, Chief Clinical Advisor

Peter Glazer, M.D., Ph.D.

Position: Co-Founder, Clinical Advisor

Dr. Glazer is chair of the department of therapeutic radiology, professor of genetics, and the Robert E. Hunter professor of therapeutic radiology at the Yale School of Medicine. His research focuses on elucidating pathways of DNA repair in cancer and developing novel agents for cancer therapy and for gene therapy. In addition to Gennao Bio, Dr. Glazer is a co-founder of Cybrexa Therapeutics. He earned a B.A. in Chemistry from Harvard, M.S. in Biochemistry from the University of Oxford, and M.D. and Ph.D. in Genetics from Yale.

Dr. Glazer is chair of the department of therapeutic radiology,...

Peter Glazer, M.D., Ph.D.

Co-Founder, Clinical Advisor

Elias Quijano, M. Phil.

Position: Co-Founder, Clinical Advisor

Elias Quijano is an M.D. and Ph.D. student at the Yale School of Medicine, working with Dr. Glazer in the department of therapeutic radiology and genetics. He has over 10 years of experience in non-viral drug and gene delivery using polymer nanoparticles, drug eluting stents, biodegradable implants, and monoclonal antibodies. In addition to co-founding Gennao Bio and helping to develop its platform, Elias is a Blavatnik Associate at Yale where he works to commercialize other academic inventions.

Elias Quijano is an M.D. and Ph.D. student at the...

Elias Quijano, M. Phil.

Co-Founder, Clinical Advisor

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