Secures $2.15M NIH Grant to Advance AI-Driven Platform for CNS Drug Safety Prediction
Quiver Bioscience, a discovery-stage biotechnology company focused on the treatment of serious central nervous system (CNS) disorders and chronic pain, has received a prestigious Phase II Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH). The award, granted by the National Institute of Neurological Disorders and Stroke (NINDS), will provide $2.15 million over three years to support further development of it’s cutting-edge CNS drug discovery platform.
The project, titled “Safe-OPTION: Optical Physiology To Interrogate Oligonucleotide Neurotoxicity,” aims to enhance the safety and tolerability prediction of antisense oligonucleotide (ASO) therapeutics using an integrated suite of AI, in vitro assays, and in silico modeling tools.
Addressing the Safety Challenges in ASO Therapeutics
ASO-based therapeutics have emerged as powerful tools in treating neurological disorders due to their precision in modulating gene expression. Several ASO therapies have demonstrated clinical success; however, neurotoxicity remains a major development hurdle, particularly in the early stages of drug development.
Traditional ASO development requires lengthy and costly preclinical safety evaluations, often conducted across multiple animal models. These studies aim to assess both acute and delayed-onset neurotoxic effects, which are difficult to predict using conventional methods. Without reliable early-stage predictors of neurotoxicity, promising ASO candidates may be delayed or discarded, slowing progress in treating neurological diseases.
Quiver’s Unique Solution: A Fully Integrated Predictive Platform
It’s approach to overcoming these challenges lies in its advanced drug discovery platform, which blends human biology, high-throughput functional screening, and data science. Their platform integrates:
- Human neuronal models derived from patient-relevant cells
- All-optical electrophysiology, enabling non-invasive, high-speed functional readouts of neuronal activity
- AI and machine learning (ML) algorithms for analyzing large-scale data and modeling toxicity risk
With the new NIH SBIR funding, it will expand this platform to enhance its predictive power for ASO-related CNS toxicity. Specifically, the funding will support the refinement of in silico ASO design tools and validation of in vitro toxicity predictions using empirical data from rodent studies. This integrated system is designed to rapidly identify safer and more effective ASO drug candidates, ultimately reducing reliance on animal testing and improving clinical translation.
Supporting FDA-Aligned New Approach Methodologies (NAMs)
it’s efforts align closely with the U.S. FDA’s push for New Approach Methodologies (NAMs)—innovative tools that reduce or replace the use of animals in drug development. NAMs include in vitro systems based on human cells and AI-powered in silico models, both of which form the foundation of it’s platform.
While animal studies have long been the standard in preclinical testing, they have several limitations:
- Scalability issues due to cost and time constraints
- Species-specific biology that may not reflect human responses
- Ethical concerns related to animal use in research
By leveraging human-derived neuronal models and computational modeling, it’s platform addresses these limitations and delivers a more human-relevant, scalable, and cost-effective approach to CNS drug safety testing.
Advancing Internal Therapeutic Programs
It has already applied its ASO discovery and screening platform to advance its own precision gene-targeted therapy pipeline. The company’s lead candidate targets Nav1.7, a voltage-gated sodium channel that plays a key role in various neuropathic pain disorders. This ASO therapeutic is designed to modulate pain signaling pathways in a highly specific and personalized manner.
Nav1.7 is a genetically and clinically validated target, and it’s data-driven platform enables rapid and precise candidate selection based on functional neuronal responses and safety profiles—reducing the development timeline and increasing the likelihood of success in clinical trials.
Broadening Impact Through Strategic Collaborations and Grants
In addition to the NIH grant, it has secured funding from several patient advocacy and research foundations dedicated to advancing therapies for rare CNS disorders. Recent awards include:
- Dup15q Alliance: Supporting drug discovery efforts for Dup15q syndrome
- FRAXA Research Foundation: Funding work on Fragile X syndrome (FXS)
- KCNT1 Epilepsy Foundation (in partnership with CURE Epilepsy): Backing efforts related to KCNT1-related epilepsy
These grants not only validate the promise of it’s platform but also provide critical resources to support the development of disease-specific therapies for underserved patient populations.
Additionally, it recently announced a research collaboration with QurAlis, focused on Fragile X syndrome. This project will be partially supported by the FRAXA grant and will leverage both companies’ strengths in precision neuroscience and ASO design.
Transforming the Future of CNS Drug Discovery
Quiver Bioscience is at the forefront of a paradigm shift in CNS drug discovery. By integrating AI, machine learning, human neuron-based assays, and scalable electrophysiological tools, the company is creating a new standard for evaluating ASO safety and efficacy.
With this new funding from the NIH and support from patient-driven organizations, it is well-positioned to:
- Accelerate the development of novel ASO therapies
- Reduce the time and cost of preclinical testing
- Improve patient safety outcomes
- Support the broader transition toward human-relevant, animal-free drug development
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.
