October 18, 2022
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Press Release: ingenious targeting laboratory Receives $3M Phase II Small Business Innovation Research (SBIR) Award

Ronkonkoma, NY October 18, 2022: ingenious targeting laboratory announced today that it has received a $2,995,545 Phase II Small Business Innovation Research (SBIR) in conjunction with New York University Langone Health. Funding was awarded from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH) in order to create and optimize a highly versatile suite of mouse platforms for isolating heavy-chain only Abs (HCAbs). The research will be led by Dr. Milen Kirolov, Scientific Director for ingenious targeting laboratory and Dr. Sergei Koralov, Principal Investigator/Associate Professor of Pathology NYU Langone Health.

The Phase II grant was awarded based on the data we have already generated from a triple-hybrid HCAb platform model which utilizes diverse camelid VHHs and human D and J segments due to a targeted replacement in the IgH locus. As part of this Phase II application, our team will provide flexibility to those who wish to avoid complications with immunogenicity by inserting human VHs with select mutations that confer camelid-like properties. One goal of the program is to demonstrate positive HCAb discovery and functionality based on usage of a minimum of 3 model antigens (HIV-1 gp120, SARS CoV2 Spike, and a novel Enterobacter antigen). This will be accomplished with the guidance from Dr. Sergei Koralov, who is partnering with his NYU colleagues Dr. Victor Torres, C.V. Starr Professor of Microbiology, and Dr. Xiangpeng Kong, Professor Biochemistry and Molecular Pharmacology to perform these studies.

ingenious targeting laboratory will then proceed with offering custom HCAb services and licensing of these novel mouse platforms to multiple pharmaceutical and small biotechnology companies, while also developing single-domain antibodies for therapeutic and diagnostic purposes in house.

ingenious targeting laboratory maintains an exclusive license and option agreement with NYU Langone for access to the relevant intellectual property.

Paul Sheiffele, President of ingenious targeting laboratory, stated, “We are working to successfully commercialize these platforms by offering custom antibody services to academic and small biotechnology early adopters while simultaneously building toward licensing the model for non-exclusive use to established pharmaceutical companies who have already expressed interest in the technology should we produce validation data.” He added, “This award from the NIAID provides an infusion of research dollars that will allow us to demonstrate positive HCAb discovery and functionality.”

“I look forward to continuing to work closely with ingenious targeting laboratory on further development of new small animal platforms for generation of a diverse repertoire of heavy-chain only antibodies and on testing the many exciting downstream applications for these models,” said Dr. Sergei Kovalov. “We are excited to see how single-domain antibodies produced by these new mouse models perform for neutralization of pathogens and for other therapeutic and diagnostic applications”.

Dr. Torres commented, “I am excited to evaluate the therapeutic potential of single-domain antibodies that ingenious targeting laboratory and Dr. Koralov have generated against emerging and antimicrobial-resistant pathogens.” He also added, “It is exciting to work closely with a new venture to develop a promising technology invented both at NYU and ingenious targeting laboratory”.

Antibodies (Abs), the pathogen-neutralizing proteins produced by immune cells, are critical for the humoral immune response against bacteria, viruses and cancer. While playing this important role in immune defense, they also have vast applications for therapeutics, diagnostics and research.

Unlike conventional Abs, heavy-chain-only Abs (HCAbs) found in camel species contain a single variable binding domain (VHH) that is necessary and sufficient for antigen recognition. Notably, even as an isolated single-domain antibody (sdAb), the VHH retains full antigen-binding capacity and displays favorable physicochemical properties that conventional Abs made up of light and heavy immunoglobulin (Ig) chains do not possess, including minimal complexity, higher hydrophilicity and better conformational stability when exposed to high temperatures, solvents and pH ranges.

The unique structure and smaller size of VHH domains (derived from HCAbs) allows them (as well as entire HCAbs) to be modified and combined with other functional partners with ease when targeting restricted antigenic epitopes (e.g. active sites of enzymes, binding sites of receptors or virus/host interaction sites). Consequently, HCAbs and their smallest functional units, VHH domains, make sites previously inaccessible by means of conventional Abs now “druggable”.

Fields that can benefit from HCAb properties include infectious diseases, cancer, inflammation, neurodegeneration (such as Alzheimer’s disease and Parkinson’s disease), and imaging/diagnostics.

About ingenious targeting laboratory
ingenious targeting laboratory (ingenious) is a biotechnology company that provides custom genetically modified mouse, rat, and rabbit models. Our Company also develops mouse platforms that can benefit a plethora of disease areas. Our trusted gene editing service is built on over two decades’ worth of successful animal model creation for investigators, organizations, and companies in 44 states and 28 countries.

Visit genetargeting.com for more information.

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