Therapeutics Pipeline
Dup15q Syndrome is a neurodevelopmental disorder generally characterized by hypotonia, motor delays, intellectual disability, epilepsy, and autism spectrum disorder. Overexpression of the UBE3A gene is thought to be the predominant cause of Dup15q Syndrome.
DEE13 is characterized by seizures, developmental delay, and intellectual disability caused by mutations in the SCN8A gene.
DEE4 is characterized by seizures, developmental delay, intellectual disability, and speech delays caused by mutations in the STXBP1 gene.
DEE associated with mutations in the SYNGAP gene presents with a spectrum of symptoms including intellectual disability, hypotonia, developmental delay, and epilepsy, among others. The SYNGAP gene encodes SynGAP, a synaptically-enriched protein that plays an important role in synaptic physiology and plasticity.
One of the most prevalent known monogenic epilepsy disorders associated with mutations to either TSC1 or TSC2 genes, which are critical to the control of cell growth and division. Patients develop benign tumors centrally and systemically in different tissues. Seizures and neurocognitive impairment are key clinical symptoms.
Neurodevelopmental disorder associated with autism and caused by hypermethylation of expanded CGG repeats (>200) in the FMR1 gene leading to gene silencing and loss of Fragile X Messenger Ribonucleoprotein (FMRP). FMRP plays important roles in neuronal function and synaptic signaling.
Nav1.7 is a genetically validated pain target outside of the opiate signaling pathway. Candidate ASOs are currently being considered for intractable cancer pain, small fiber neuropathy, and moderate-to-severe lumbosacral radiculopathy.
Nav1.8 is a genetically validated pain target outside of the opiate signaling pathway. Candidate ASOs are currently being considered for intractable cancer pain, small fiber neuropathy, and moderate-to-severe lumbosacral radiculopathy.
Nav1.7 and Nav1.8 are genetically validated pain targets outside of the opiate signaling pathway. Candidate ASOs are currently being considered for intractable cancer pain, small fiber neuropathy, and moderate-to-severe lumbosacral radiculopathy.
Nav1.7 is a genetically validated pain target outside of the opiate signaling pathway. Candidate small molecules are currently being considered for osteoarthritis.
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Select Technology Pipeline
Cutting-edge technologies in development support therapeutic discovery
Human Biology
Proprietary spiking HEK cell models are scalable, synthetically excitable cells that will have robust utility for high-throughput, optogenetically-driven, target-based screening on select ion channels.
Inhibitory neuron models will support the study of genetic epilepsies and neurodevelopmental disorders where more standard excitatory neuron models inadequately capture disease-relevant biology.
Novel sensory neuron models will augment research in pain and other peripheral nervous system disorders to help bridge the translational gap where other models typically fail to reproduce human biology.
Measurement Engineering
Firefly™ 3.0 will advance throughput and capacity of the original instrument while maintaining the same exquisite sensitivity in the capture of single-cell and single action potential activities.
Swarm™ 1.5 will enhance operational performance of the original instrument to support the capture and measurement of electrophysiology data with whole-well parallelized readouts.
Swarm™2.0 will advance throughput, capacity, and enable multiplexed assay formats from the original instrument to generate electrophysiology insights at a uniquely fast scale beyond conventional approaches.
Assays will assemble a detailed network map of in vitro interconnected neurons and associated synaptic strengths and kinetics to reinforce the evaluation of complex disease states and the development of relevant therapeutics.
Advanced assays and associated neuro-analytics will combine the power of transcriptomics with the multi-parameter quantitative characterization of neuronal activity to a support target evaluation and therapeutic screening efforts.
Algorithmic Analysis
Computational neuro-analytics will quantify synaptic activity at an unprecedented scale by coordinating high-content imaging of synaptic protein localization with synaptic functional data from our cutting-edge optical electrophysiology technology.
Sophisticated AI/ML algorithms will identify and recognize archetypal effects of compounds and compound classes as “functional drug fingerprints” using in-house datasets, and a reference database will span hundreds of critical, druggable disease targets to support phenotyping and mechanism-of-action analyses.
Custom AI/ML algorithms will leverage the massive data capture from our proprietary optogenetic electrophysiology engineering to provide deeper insights into neuronal phenotypes and the effects of candidate therapeutics.
To support preclinical wet lab activities, AI/ML algorithms will automate experimental design parameters in the discovery process to speed planning, tracking, and execution efforts as well as enable new phenotyping and data mining capabilities.
Next-generation analytics will augment ASO design and enriched sequence selection through toxicity predictions based on internally-generated transcriptomic and optical electrophysiology data.
* Programs in validation assumed to reach deployment in 6 to 12 months
† Supported by NIH SBIR funding
NIH Awarded Grants
Recently earned grants highlight the value of internal efforts
We have garnered exciting collaborations with a variety of industry leaders.
We welcome partnership opportunities with other organizations seeking to develop novel therapeutics for serious diseases.
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