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CNS Drug Discovery — From Target to IND

Integrated CNS expertise, from complex biology to confident development decisions. 

ChemPartner’s Neuroscience platform integrates in vitro CNS target screening, multi-electrode array and patch clamp electrophysiology, primary neuron and iPSC cell biology, and 120+ validated in vivo models across Alzheimer’s, Parkinson’s, epilepsy, pain, psychiatric disorders, and rare neurological diseases — all under one roof.

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Platform Overview

Two Integrated Pillars. One Neuroscience Platform.

Drug discovery for CNS diseases is one of the highest-attrition areas in pharma — and one of the most complex to execute in a CRO setting. ChemPartner’s neuroscience platform resolves this by integrating in vitro CNS pharmacology with a deeply validated in vivo program, with shared data infrastructure and direct scientist-to-scientist collaboration between the two divisions.

In Vitro Pillar

CNS target screening (20+ ion channels, GPCRs), primary neuron culture, patch clamp electrophysiology, multi-well MEA (mwMEA), brain slice recording, and iPSC/organoid functional evaluation.

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In Vivo Pillar

120+ validated disease models across Alzheimer’s, Parkinson’s, epilepsy (14 models), pain (21 models), psychiatric disorders (26 models), auditory/ophthalmic (19 models), rare disease, and CNS safety pharmacology — supported by EEG telemetry, IHC/IF pathology, and DMPK integration.

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In Vitro

In Vitro Capability Modules

Four modules spanning CNS target screening, electrophysiology, primary neuron and iPSC biology, and seizure liability — each validated against clinical reference compounds.

Module 1
CNS Target Screening

Comprehensive CNS target screening with full ion channel coverage, GPCR functional assays, and a proprietary validation database with historical raw trace retrieval.

CNS Target Screening
  • High-throughput automated patch clamp (HT-APC): Nav1.1, Nav1.5, Nav1.7, Nav1.8, Kv1.3, Kv11.1 (hERG), KCNQ2/3, SK channels
  • Ligand-gated ion channels: AMPA, NMDA, GABA-A, nAChR
  • Manual patch clamp for secondary characterization and primary neuron work
  • FLIPR calcium flux for voltage-gated and store-operated channels
Module 2
In Vitro Electrophysiology

The broadest in vitro electrophysiology menu in the CNS CRO space — from single-cell patch clamp on primary neurons to network-level mwMEA on cerebral organoids.

Patch Clamp (Primary Neurons & iPSC):
  • AMPA receptor PAM (CX-614 dose-response on cortical neurons)
  • NMDA receptor characterization (AP-5 validated on primary cortical neurons)
  • GABA reversal potential (E_GABA) developmental time-course by gramicidin-perforated patch
  • Nav1.8 selective inhibition (A803467, VX-548/Suzetrigine on DRG neurons)
  • Retigabine KCNQ activation, trigeminal ganglia RMP hyperpolarization
  • hiPSC cortical/motor neuron evoked AP firing from healthy and diseased sources
Module 3
Primary Neuron & Cell Biology

Primary neuron isolation, culture, and functional biology — from cortical, hippocampal, and DRG neurons to microglia — with morphology analysis and disease-relevant biomarker staining.

Primary Neuron, Glial Cell & Disease-Model Capabilities:
  • Primary neuron isolation: cortical, hippocampal, DRG, trigeminal ganglia, astrocytes (monkey)
  • High-content imaging: Nikon and Leica confocal + high-content cell analyzer (neurite morphology)
  • Biomarker staining: TUJ, MAP2, synapsin, target co-localization IHC/IF
  • Microglia culture and activation; phagocytosis assays (trontinemab Aβ clearance validated)
  • Aβ oligomer model: quality-controlled Aβ oligomer prep, CTG viability assay on cortical neurons
  • 2D direct transdifferentiation: human DRG neurons (TTX-sensitive AP, Nav1.7/Nav1.8 functional expression)
Module 4
Seizurogenic Assays & Genetic Disease Models

Purpose-built in vitro seizure liability and CNS genetic disease assays, validated against clinical reference compounds.

Validated assay panel:
  • In vitro seizurogenic panel (mwMEA): PTX, 4-AP, PTZ, High K+ — vs. valproate (VPA) and retigabine (RTG)
  • Dravet Syndrome (DS) model: SCN1A-mutant neurons; comparative spike/burst susceptibility vs. WT
  • 3D brain organoid seizure model: spontaneous firing potentiation (PTX) and suppression (RTG)
  • hiPSC-forebrain, cerebral, and midbrain organoid functional validation (collaboration portfolio)
  • Basal forebrain cholinergic neuron, dopaminergic neuron, and DRG neuron functional validation
Module 5
CNS Target Screening

Comprehensive CNS target screening with full ion channel coverage, GPCR functional assays, and a proprietary validation database with historical raw trace retrieval.

CNS Target Screening
  • High-throughput automated patch clamp (HT-APC): Nav1.1, Nav1.5, Nav1.7, Nav1.8, Kv1.3, Kv11.1 (hERG), KCNQ2/3, SK channels
  • Ligand-gated ion channels: AMPA, NMDA, GABA-A, nAChR
  • Manual patch clamp for secondary characterization and primary neuron work
  • FLIPR calcium flux for voltage-gated and store-operated channels
Module 6
CNS Target Screening

Comprehensive CNS target screening with full ion channel coverage, GPCR functional assays, and a proprietary validation database with historical raw trace retrieval.

CNS Target Screening
  • High-throughput automated patch clamp (HT-APC): Nav1.1, Nav1.5, Nav1.7, Nav1.8, Kv1.3, Kv11.1 (hERG), KCNQ2/3, SK channels
  • Ligand-gated ion channels: AMPA, NMDA, GABA-A, nAChR
  • Manual patch clamp for secondary characterization and primary neuron work
  • FLIPR calcium flux for voltage-gated and store-operated channels
Module 7
CNS Target Screening

Comprehensive CNS target screening with full ion channel coverage, GPCR functional assays, and a proprietary validation database with historical raw trace retrieval.

CNS Target Screening
  • High-throughput automated patch clamp (HT-APC): Nav1.1, Nav1.5, Nav1.7, Nav1.8, Kv1.3, Kv11.1 (hERG), KCNQ2/3, SK channels
  • Ligand-gated ion channels: AMPA, NMDA, GABA-A, nAChR
  • Manual patch clamp for secondary characterization and primary neuron work
  • FLIPR calcium flux for voltage-gated and store-operated channels
Capability Finder

Choose your starting point

Alzheimer's Disease (13 models)
  • 5xFAD mice — licensed (valid 2025–2035). Full phenotype: Aβ ELISA/IHC/IF, neuroinflammation (astrocytes/microglia), cytokines (TNF-α/IL-1β/IL-6), EEG (DSI), cognition (Water Maze/NOR/Y-Maze/Passive Avoidance), LTP in brain slice
  • Case study: Trontinemab (anti-tau/TfR bispecific) — detection of human IgG signal in blood vessels 8h post 10 mg/kg IV in M6 5xFAD mice; IHC confirmed BBB engagement
Biochemistry
  • 6-OHDA rats/mice (classic model); L-DOPA and reserpine-induced dyskinesia; tacrine jaw movement
  • CBE-induced PD: glucocerebrosidase inhibition → mitochondrial dysfunction → PD onset; NFL plasma biomarker; beam walk/rotarod/wire hang endpoints
  • Rotenone-induced PD (2.5 mg/kg s.c. × 28d): behavioural deficits + BMP(22:6) urinary biomarker
  • NHP eyeblink model; harmaline-induced essential tremor (rats/mice)
Cell-Based Assays
  • Chemical: PTZ s.c. (101 study historical database), LiCl-pilocarpine SE (rat), kainate MTLE with EEG SRS monitoring
  • Electrical: 6Hz, MES, MEST, amygdala kindling (ADD/ADT)
  • Genetic: Dravet mice (hyperthermia-induced), AY9944 Smith-Lemli-Opitz model
Functional Assays
  • Inflammatory: CFA, formalin, LPS, MIA (OA); visceral: acetic acid writhing
  • Neuropathic: CCI, SNL, SNI, STZ-diabetic, vincristine/paclitaxel-induced
  • Post-surgical (Brennan incisions); NTG-induced migraine (in development)
  • Readouts: Von Frey, Randall-Selitto, tail flick, hot/cold plate, formalin Laboras
Pain Disease Models

 

  • Inflammatory pain: CFA, formalin, LPS, MIA-induced osteoarthritis, and visceral acetic-acid writhing
  • Neuropathic pain: CCI, SNL, SNI, STZ-diabetic, and vincristine/paclitaxel-induced models
  • Post-surgical pain: Brennan incision model
  • Migraine: NTG-induced migraine model
  • Readouts: von Frey, Randall–Selitto, tail flick, hot/cold plate, and formalin/Laboras
Integrated In Vivo Pharmacology

 

  • Generate decision-ready efficacy, PK/PD, and biomarker data through fit-for-purpose pain studies, including exposure–response analysis, behavioral endpoints, histopathology, and ex vivo pharmacology support.

Explore Translational Pharmacology

Sensory &
Specialized Models

Supporting two growing CNS-adjacent modalities — gene therapy and complement-targeted biologics — with dedicated preclinical infrastructure.

Auditory / Hearing (19 Models)
  • Drug-induced hearing loss (aminoglycoside, cisplatin)
  • Noise-exposure hearing loss
  • Genetic deafness models (DPOAE + ABR endpoints)
  • Local drug delivery: intratympanic (IT), intra-labyrinthine (IL/PSCC), transtympanic (TT), round window
  • Vestibular function evaluation (head bobbing, circling, righting reflex)
  • Ex vivo cochlea: whole-mount + cross-section confocal (stereocilia, dendritic spine, myosin, TUJ staining)
Case Study — AAV Gene Therapy:

AAV-DJ CMV-GFP expressed in hair cells and supporting cells at 2E+10 vg, 4 wks post intra-labyrinthine injection in adult C57 mice. Dose-dependent IHC expression confirmed.

  • Dry eye: BAC-induced (mouse)
  • Cataract: sodium selenite-induced (rat)
  • Dry AMD: sodium iodate-induced (mouse) with ERG readout; N-acetylcysteine rescue validation confirmed retinal layer thickness recovery
  • Optic nerve crush model + NF-L plasma biomarker
  • Intravitreal AAV injection (mouse); scraping corneal endothelial cells (rabbit)
Case Study — Pegcetacoplan (Complement C3 Inhibitor):

ERG waveform peak recovery in complement C3-inhibitor treated eyes vs. AMD-model controls — pilot completed, formal study ongoing. Validates ChemPartner’s complement AMD platform for biologics testing.

Why It Matters

Translational Relevance & Key Differentiators

ChemPartner’s neuroscience platform is anchored in clinical translation — every model and assay has been validated against clinically relevant reference compounds or established against approved drugs.

01
Trontinemab
(Roche, Phase II AD)
ChemPartner 5xFAD BBB engagement validated — human IgG IHC in blood vessels 8h post IV
02
Pegcetacoplan
(complement C3 inhibitor)
ChemPartner dry AMD model showed ERG recovery — ongoing formal efficacy study
03
AAV gene therapy
(auditory)
IL injection GFP expression in IHCs at clinical-relevant dose range
04
Emraclidine
(M4 mAChR, schizophrenia, Phase 1b)
ChemPartner fEPSP assay supports this target class
05
Suzetrigine
(VX-548, Nav1.8, approved 2024)
ChemPartner DRG Nav1.8 inhibition assay validated against clinical compound
Platform Differentiators
Licensed 5xFAD colony on site — no import delays. 10-year license (2025–2035)
Organoid electrophysiology (mwMEA): industry-leading capability for rare CNS diseases and iPSC human-relevant models
101 historical PTZ epilepsy studies + 56 MEST studies — the deepest historical dataset of any CRO in the region
NHP pharmacology: pharmaco-EEG, eyeblink, and ECG safety pharmacology in cynomolgus monkeys
Combined in vitro–in vivo workflow: patch clamp hit confirmed in 5xFAD or 6-OHDA within the same project
EXPLORE THE PLATFORM

Explore the Full Range of Development and Manufacturing Capabilities

CNS Target Screening

Ion channels, GPCRs, HT-APC, manual patch, FLIPR — with validated reference compound database.
In Vitro Electrophysiology

Patch clamp, mwMEA (Maestro Pro), and MED64 brain slice recording across multiple CNS targets.
Primary Neuron & iPSC Biology

Primary culture, IHC/IF morphology, Aβ models, microglia activation, transdifferentiated human neurons.
In Vivo Neuropharmacology

120+ validated models: AD, PD, epilepsy, pain, psychiatric, rare disease — AAALAC-accredited.
CNS Safety Pharmacology

sLMA, modified Irwin, pharmaco-EEG (rodent/dog/NHP), respiratory, cardiovascular safety.
Auditory & Ophthalmology

Hearing loss models (DPOAE/ABR), AAV inner ear delivery, retinal degeneration, ERG, NF-L.
Neuroscience

Ready to accelerate your CNS program?

From first-in-class target validation to full IND-ready preclinical packages, ChemPartner’s integrated neuroscience platform delivers the models, the data quality, and the translational relevance your program demands.

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