SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses
TARGETgenehuman

ROCK2Rho-associated protein kinase 2

Description

ROCK2 — Rho-associated kinase, druggable by Fasudil (approved in Japan/China for stroke). ROCK1/2 expression elevated in SMND7 mice. Fasudil Phase 2 in ALS (NCT03792490). Part of actin dynamics cascade: SMN-PFN1-RhoA-ROCK-LIMK-CFL2. Nobody has tested ROCK inhibitors in SMA mouse models — first-in-field.

Linked hypotheses (20)

  • MN25_ROCK2 binds LIMK2_aC (Boltz-2 iPTM=0.921)
    targetconf 92%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%
  • [Survival] LIMK1 is implicated in SMA pathogenesis based on 3 converging claims
    targetconf 90%

Top compound hits (10)

Compound IDSMILESScoreTier
FasudilO=C1CCN(Cc2cccc3cnccc23)CC10.488ready
CHEMBL128467O=S(=O)(O)CCNc1cc2ncnc(Nc3cccc(Br)c3)c2cn11.276promising
CHEMBL308645Cc1cc(CCC(=O)O)[nH]c1/C=C1\C(=O)Nc2ncnc(Nc3ccc(F)c(Cl)c3)c211.250promising
RiluzoleOc1nc2ccc(OC(F)(F)F)cc2s11.206promising
GenMol_100O=C1CCN(Cc2csc(-c3ccccc3)n2)CC11.198promising
RipasudilO=S(=O)(NCC1CC1)c1ccc2[nH]c3c(c2c1)CNC31.194promising
CHEMBL409082O=C(Nc1nnc(SCc2ccccc2)s1)c1ccc(F)cc11.191promising
H-1152CC(C)C(=O)N1CCC(C(F)(F)F)CC1c1ccncc11.185promising
GenMol_103O=C1CCN(Cc2cccnc2-c2ccccc2)CC11.184promising
MW150Cc1nnc(-c2cccc(-c3cc(C)nn3C)c2)o11.150promising

Linked claims (50)

TypePredicateConfSource
pathway membershipThe RHO-1/Rho GTPase-LET-502/ROCK-MLC-4 phosphorylation signaling pathway regulates axon regeneration (observed in C. elegans)90%W2887003161
protein interactionaf3_iptm83%
neuroprotectionBlockade of motor neuron K channels by 4-aminopyridine may prolong the action potential and introduce a significant delay and slowing of evoked synaptic currents (observed in zebrafish model)80%W2120280034
gene expressionfasudil (30 μM) specifically increased glial cell line-derived neurotrophic factor (GDNF) among the secreted proteins at the transcription and secretion levels (in vitro).69%25185536
drug efficacyThe dual ROCK1/2 inhibitor, fasudil (10 mg/kg), reduced infarct size (mouse model).69%41704339
drug efficacyOral administration of a selective ROCK2 inhibitor, KD025, reduces psoriasis area and severity index scores by 50% from baseline in 46% of patients with psoriasis vulgaris (clinical).69%28389592
pathway membershipActivation of the Rho kinase (ROCK) pathway appears to be instrumental for the modulation of the microglial phenotype.65%28420986
drug efficacyROCK inhibition with Y-27632 produced a genotype-specific response, restoring β-catenin nuclear localization exclusively in FlncKO-C2C12 without affecting WT-C2C12 cells (in vitro).65%41724730
pathway membershipThe endoplasmic reticulum-to-Golgi transport of SREBP cleavage-activating protein requires the actin-based cytoskeleton and is enhanced by the Rho-ROCK-LIMK-cofilin pathway.65%12775580
gene expressionKD025 downregulated the expression of lipogenic factors FABP4 and Glut4 during adipogenesis (in vitro).65%29410516
pathway membershipFluid shear stress activates the Rho downstream effectors ROCK, LIM kinase (LIMK), and cofilin.65%12775580
pathway membershipThe ROCK/LIMK/Cofilin pathway is suppressed in rat fibroblasts upon expression of activated Ras (in vitro).65%16007212
drug efficacyInhibiting ROCK by Y-27632 treatment at the zygote stage resulted in most embryos failing to develop to the 8-cell stage (mouse model).65%24562870
drug efficacyKD025 dose-dependently reduced infarct volume after transient middle cerebral artery occlusion (mouse model).65%24466563
drug efficacyY27632 (a specific ROCK inhibitor) (in vitro) reduced the internalization of A. fumigatus and the level of phosphorylated cofilin.65%26268695
pathway membershipActivation of the Rho-associated kinase (ROCK) pathway participates in the dopaminergic neuron degeneration and possibly in Parkinson's disease (PD).65%PMID:25908255
drug efficacySpecific ROCK2 inhibitors (e.g. belumosudil) could be repurposed for cystic kidney disease treatment (in vitro).65%40253509
drug efficacyTreatment with Y-27632, a specific ROCK inhibitor, dysregulation of ROCK/cofilin pathway decreased p-cofilin expression and induced cell migration.65%21769918
drug efficacyOral administration of KD025, a selective ROCK2 inhibitor, to healthy human subjects down-regulates the ability of T cells to secrete IL-21 and IL-17 by 90% and 60%, respectively, but not IFN-γ in res...65%25385601
drug efficacyFasudil treatment significantly reduced α-Syn aggregation in vitro in a H4 cell culture model as well as in a cell-free assay.65%27101974

Showing 20 of 50.

Login → Command Center