On April 10, 2022 Gennao Bio, a privately held genetic medicines company developing first-in-class, targeted nucleic acid therapeutics, reported promising preclinical results of its proprietary, non-viral gene monoclonal antibody (GMAB) platform in multiple solid tumor models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting (Press release, Gennao Bio, APR 10, 2022, View Source [SID1234611851]). The findings from these studies were reported and discussed in an oral presentation by Elias Quijano, M.D./Ph.D. candidate in the laboratory of Dr. Peter Glazer at the Yale School of Medicine, and co-founder of Gennao Bio, entitled, "Systemic targeting of therapeutic RNA to cancer via a novel, cell-penetrating and nucleic acid binding, monoclonal antibody."

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The preclinical results demonstrated GMAB’s ability to form non-covalent complexes with and systemically target and deliver 3p-hpRNA, a potent activator of the immune signaling RIG-I pathway, to solid tumors, including orthotopic mouse models of human pancreatic cancer (KPC) and medulloblastoma (DAOY). GMAB’s highly specific delivery into tumors is independent of the endocytic pathway and is uniquely enabled by targeting ENT2, a nucleoside transporter that is overexpressed in many tumors. In vitro studies of GMAB/3p-hpRNA demonstrated that delivery of a RIG-I agonist to tumor cells triggers an immune stimulating type-1 interferon response and triggers direct tumor cell death.

"These positive results further reinforce our strong belief in the broad therapeutic potential and diverse application of our GMAB platform in treating cancers with substantial unmet need," said Stephen Squinto, Ph.D., chief executive officer and chair of the board of Gennao Bio. "We expect to advance the humanized version of GMAB/3p-hpRNA, GMAB-7001, into Investigational New Drug (IND)-enabling studies in the second half of 2022 and will continue to assess additional oncology pipeline programs."

In the KPC pancreatic cancer model, multiple doses of GMAB/3p-hpRNA resulted in a significant survival benefit, driven in part by long-term increases in tumor-infiltrating lymphocytes, including CD45+, CD8+, CD4+, and CD19+ cells. GMAB/3p-hpRNA treatment also showed a statistically significant increase in tumor cell necrosis compared to the control group. Previous studies of a single dose administration of GMAB/3p-hpRNA in an orthotopic model of medulloblastoma demonstrated its ability to penetrate the central nervous system, reduce intracranial tumor burden by 50%, and prevent spinal metastases.

"The GMAB platform has the potential to address the challenges faced by alternative methods of delivery of immunostimulatory nucleic acids to tumors, which have been associated with systemic toxicities or rely on suboptimal intra-tumoral injections. Studies of single and multiple intravenous doses of GMAB/3p-hpRNA have shown targeted payload tumor delivery and resultant tumor growth suppression in several preclinical models of difficult-to-treat forms of cancer," said Mr. Quijano. "These promising monotherapy results, and the new data generated in a difficult-to-treat pancreatic cancer model, warrant continued research of the GMAB platform and development of this new class of targeted nucleic acid therapeutics for cancer."

A copy of the AACR (Free AACR Whitepaper) presentation can be found under the "News" section on the Company’s website, www.gennao.com.