Pipelines

Therapeutics Pipeline

16 internal proprietary and partnered collaboration programs

Internal

Disorder / Target
Discovery
Candidate ID
Preclinical
Clinical
Seizure and Neurodevelopment Disorders
Dup15q Syndrome - UBE3A (ASO)
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DEE13 - SCN8A (ASO)
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DEE4 - STXBP1 (ASO)
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Rett Syndrome - MECP2
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DEE7 - KCNQ2
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DEE - SYNGAP
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Phelan-McDermid Syndrome - SHANK3
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Pain
Nav1.7 (ASO)
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Nav1.8 (ASO)
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Dual Nav1.7 / 1.8 (ASO)
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Nav1.7 (Small Molecule)
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Partnered

Disorder / Target
Partner
Candidate ID
Preclinical
Clinical
Spastic Paraplegia Type 49 (ASO)
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TECPR2 Foundation
ALS Target (Small Molecule/ASO)
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QurAlis
Parkinson's Disease Target (ASO)
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Chamishi Therapeutics
Select Genetic Epilepsy (Small Molecule)
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UCB
Undisclosed Genetic Disorder
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Vertex

Select Technology Pipeline

Cutting-edge technologies in development support therapeutic discovery

Human Biology

Technology
Concept
Development
Validation*
Deployment
New Spiking HEK Cell Models †
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Next-Gen Human Inhibitory Neuronal Models †
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Next-Gen Human Sensory Neuronal Models †
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Measurement Engineering

Technology
Concept
Development
Validation*
Deployment
Firefly™ Instrument 3.0 (500,000 neurons/day) †
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Swarm™ Instrument 1.5 (10,000 wells/day) †
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Swarm™ Instrument 2.0 (100,000 wells/day)
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Synaptic Neuronal Network Assays †
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Integrated Optogenetics/Transcriptomics Assays
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Algorithmic Analysis

Technology
Concept
Development
Validation*
Deployment
Multimodal Synaptic Quantification †
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AI/ML-driven Drug Fingerprinting †
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AI/ML-driven Optogenetic Trace-based Phenotyping
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AI/ML-directed Experimental Design Syntax
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ASO Design Tool 2.0
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* 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

Research Subject
Agency
Type
Progress
Anti-sense oligonucleotide platform for rare genetic diseases of the nervous system
NIMH
Phase I
Targeting ALS-linked FUS with allele-specific antisense oligonucleotides
NINDS
Phase I
Optopatch: high-throughput all-optical electrophysiology
NIMH
Phase II
An all-optical electrophysiology platform for the discovery of pain therapeutics
NINDS
Fast Track Phase II
Scalable, all-optical assays of synaptic function and plasticity
NIMH
Phase II
Functional drug fingerprinting with all-optical electrophysiology
NIMH
Phase I
A phenotypic screen for osteoarthritic pain therapeutics using all-optical electrophysiology
NIAMS
Fast Track Phase II
Channelopathy-associated epilepsy research
NINDS
Phase I
An antisense oligonucleotide therapy for KCNT1 based epileptic encephalopathies
NINDS
Phase I
A phenotypic screen for compounds that differentially affect excitatory and inhibitory signaling
NIMH
Fast Track Phase II
Development of anti-sense oligonucleotides as a therapeutic for cancer pain through selective block of sodium channel pain targets
NCI
Fast Track Phase II