We believe better cells lead to better medicines™ and that the key to making better cells is precisely controlling the differentiation of human pluripotent stem cells, which are the building blocks of all cells of the human body. For over 15 years, our stem cell research and development teams and collaborators have developed proprietary methods for controlling the differentiation of pluripotent stem cells to produce multiple types of mature, functional adult human cells, with emphasis on human heart and liver cells for our drug rescue programs. We believe drug rescue is the highest-value, near-term commercial application of the human cells we produce and our novel in vitro bioassay systems, CardioSafe 3D™ and LiverSafe 3D™, the foundation of our Human Clinical Trials in a Test Tube™ platform.
“Drug rescue” refers to research and development using small molecule drug candidates (Drug Rescue Candidates™) previously discovered and validated in efficacy studies by pharmaceutical companies, but terminated during development prior to FDA approval due to unexpected heart or liver safety concerns. Our drug rescue model leverages publicly available information and substantial prior investment by pharmaceutical companies and others in Drug Rescue Candidates to generate Drug Rescue Variants™. These are proprietary, new, safer small molecule variants of Drug Rescue Candidates validated internally in our bioassay systems. We anticipate that each validated lead Drug Rescue Variant will be a proprietary, new molecular entity (NME), suitable as a promising drug development program, either internally or in collaboration with a strategic partner. Through our stem cell technology-based drug rescue programs, we intend to become a leading source of proprietary, small molecule NMEs to the global pharmaceutical industry.
Using mature cardiomyocytes (heart cells) differentiated from human pluripotent stem cells,
we have developed CardioSafe
3D, as a novel, in vitro
bioassay system used to assess new drug candidates for potential cardiac toxicity before they are tested in animals or humans. We believe CardioSafe
3D is capable of predicting the in vivo
cardiac effects, both toxic and non-toxic, of small molecule drug candidates with greater speed and precision than the long-established, surrogate safety models most often used in drug development, including models using animal cells or live animals, and cellular assays using cadaver, immortalized or transformed cells.
Using mature, hepatocytes (liver cells) derived from human pluripotent stem cells,
with adult functional properties, we are currently validating LiverSafe
3D, our second novel stem cell technology-based bioassay system. We believe LiverSafe
3D will enable us to assess new drug candidates for potential liver toxicity and metabolism-based safety issues resulting in adverse drug-drug interactions, early in development, long before human testing. Drug-related liver toxicity and adverse drug metabolism, as a group, represent one of the top-two reasons for safety-related drug failure during clinical development. We plan to use LiverSafe
3D, and the clinically predictive liver biology insight we believe it provides, to expand the scope of our commercial opportunities related to drug rescue.
With $8.8 million of grant funding awarded from the U.S. National Institutes of Health (NIH),
we have successfully completed Phase 1 development of AV-101, also known as “L-4-chlorokynurenine” and “4-Cl-KYN”. AV-101 is an orally available small molecule prodrug candidate aimed at the multi-billion dollar neurological disease and disorders market, including neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, epilepsy and depression.