SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
1,145Targets
453Trials
60Drugs
7Datasets
34,514Sources
43,071Claims
46,973Evidence
29,625Hypotheses

Lab-OS

EXPLORATORY
8 assays · 3 cloud labs

Phase 10.4 — Open-source experiment design automation. 8 standardized SMA assays with timeline and protocol specifications. 3 cloud lab integrations (Emerald Cloud Lab, Strateos, Opentrons). Generates complete experiment designs from hypothesis text.

SMA Assay Library

AssayTypeTargetReadoutThroughputCostTimeAuto
SMN2 Splicing Reporter (RT-qPCR)cell_basedSMN2 exon 7 inclusion ratioFL-SMN / delta7-SMN mRNA ratiohigh$15-253dYES
SMN Protein ELISAbiochemicalSMN protein levelsSMN protein (ng/mL) by sandwich ELISAhigh$20-352dYES
Motor Neuron Survival (iPSC-MN)cell_basedMotor neuron viability% surviving MNs (HB9+/ChAT+ cells) at day 28medium$100-20028d
NMJ Formation Assay (co-culture)cell_basedNeuromuscular junction formation and functionAChR cluster count + co-localization with SV2 (presynaptic)low$200-40021d
SMA Mouse Survival Study (delta7)animalSurvival and motor functionMedian survival (days), righting reflex time, body weightlow$500-100060d
Ion Channel Electrophysiology (patch clamp)cell_basedIon channel function in motor neuronsAction potential parameters, firing frequency, resting Vmemlow$300-5007d
RNA-Binding Fluorescence PolarizationbiochemicalCompound-RNA binding affinityKD (dissociation constant) by fluorescence polarizationhigh$5-101dYES
Mitochondrial Function (Seahorse XF)cell_basedMitochondrial respiration and glycolysisOCR (oxygen consumption rate), ECAR (extracellular acidification rate)medium$30-502dYES

8 assays cover the full SMA validation pipeline: from RNA binding → splicing → protein → cell survival → NMJ → animal model. 4 are cloud-lab compatible for automated screening.

Cloud Lab Integrations

Python SDK (Symbolic Lab Language) · 7d turnaround
Capabilities: Liquid handling (multichannel, 96/384-well), qPCR (QuantStudio 7), ELISA (plate reader), Cell culture (incubators, media exchange), Flow cytometry, Mass spectrometry
SMA assays: 4
Pay-per-experiment + monthly platform fee
REST API + Web UI · 14d turnaround
Capabilities: High-throughput screening (384/1536-well), Compound management, Automated cell culture, Imaging (Opera Phenix), Compound cherry-picking
SMA assays: 3
Subscription + per-run fees
Python API (opentrons package) · 1d turnaround
Capabilities: Liquid handling (OT-2 or Flex), PCR prep, ELISA prep, Serial dilution, Plate reformatting
SMA assays: 3
$10,000 hardware + consumables only
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