Quiver Bioscience (“Quiver”), a discovery technology and therapeutics company advancing programs for treatment of serious central nervous system (CNS) disorders and chronic pain, today announced receipt of a Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH). The Phase II grant, awarded by the National Institute of Neurological Disease and Stroke (NINDS) is titled “Safe-OPTION: Optical Physiology To Interrogate Oligonucleotide Neurotoxicity.” It will provide Quiver with $2.15 million over three years to continue development of an integrated platform for improved prediction of safety and tolerability of CNS-targeted antisense oligonucleotide (ASO) therapeutics.

ASO therapeutics are increasingly being developed for the treatment of neurological conditions, as they offer precision regulation of disease target activity. Despite demonstrated clinical success of ASOs for neurological disorders, ASO drug development in this area faces the challenge of ensuring that candidates avoid modality-specific neurotoxicity, requiring lengthy preclinical safety studies in several animal species. Incorporating improved earlier predictors of ASO-induced acute and delayed onset neurotoxicity will allow for more efficient ASO drug development. Quiver has built an exceptional drug discovery platform which combines disease relevant human neuronal models, all-optical electrophysiology-based functional readouts, and AI/ML-enabled analytics to provide unique insights into neurological disorders and accelerate drug development. With the new SBIR funding, Quiver will further augment their existing ASO drug development capabilities by applying their platform to better prediction of ASO CNS toxicity using a combination of machine learning (ML)-based in silico ASO design tools and a series of all-optical electrophysiology platform-based in vitro neuronal functional assays, benchmarking their predictive algorithm with empirical data from rodent studies. This enhanced platform will accelerate the ASO drug development process by rapidly identifying lead molecules with better probability of clinical success while also reducing costs and time associated with preclinical animal studies.

Quiver’s objectives in this newly funded project are aptly aligned with the FDA’s push towards replacement of preclinical animal safety studies with New Approach Methodologies (NAMs), including in vitro human cellular systems and artificial intelligence (AI) / machine learning (ML)-based in silico models. While animal models have been historically valuable in understanding disease biology and facilitating drug development, they do have limitations including the fact that animal studies are not scalable, and they can be prohibitively expensive and time consuming. In addition, due to species-specific variations in biology, animal models may not accurately predict performance of a drug compound in humans. Advanced human cellular models have transformed the field of drug discovery in neurological disorders by allowing for the study of biological mechanisms and evaluation of drug targets in live functional neurons carrying the same genetic basis as neurons in human subjects affected by the disease. Quiver’s platform uniquely probes neuronal biology at a scale that is not possible with alternative technologies and fills a gap in the ability to directly target fundamental neurophysiological mechanisms for therapeutic discovery. With the added capabilities built partly with this new funding, Quiver now addresses the outstanding challenges associated with predicting and mitigating neurotoxicity related to ASO therapeutics.

Quiver has already leveraged their unique ASO design and in vitro screening platform to advance internal precision gene-targeted therapy programs including their lead asset, an ASO candidate targeting the genetically and clinically validated pain target Na_v1.7, a voltage gated sodium channel implicated in several neuropathic pain disorders.

In addition to NIH SBIR awards, Quiver has recently received grant support from key patient advocacy and research groups who are focused on advancing precision medicines for neurological disorders. These include grants from the Dup15q Alliance, the FRAXA Research Foundation, and the KCNT1 Epilepsy Foundation in partnership with CURE Epilepsy and are all intended to support Quiver’s drug discovery programs related to each of the three CNS disorders (Dup15q syndrome, Fragile X syndrome (FXS), and KCNT1-related epilepsy, respectively). Quiver recently announced a FXS-focused research collaboration with QurAlis, which will be partially supported by the new FRAXA Research Foundation grant. These partnerships are instrumental to Quiver’s mission to accelerate transformative therapeutic discovery for both rare and common disorders of the CNS.

About Quiver Bioscience

Quiver Bioscience is a technology-driven company established to create transformational medicines for the brain while simultaneously uncovering new biology and novel, effective drug targets. Using advanced single-cell imaging and multi-omics, we are building the world's most information-rich neuronal insight map via our "Genomic Positioning System." Our approach integrates cutting-edge scalable human models, state-of-the-art technology and proprietary engineering, and learning and surrogate AI/ML models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients. For more information, including partnerships and publications describing application of Quiver’s GPS to drug discovery, visit www.quiverbioscience.com or follow us on LinkedIn.

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