Protein / target

RAF proto-oncogene serine/threonine-protein kinase

RAF1P04049Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
4
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Protein serine/threonine kinase activity

Primary system

Nervous system

Strongest disease association

RASopathy

Genetic evidence · score 0.96

Therapeutic maturity

Clinically validated target

4 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

4 approved · 5 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Serine/threonine-protein kinase that acts as a regulatory link between the membrane-associated Ras GTPases and the MAPK/ERK cascade, and this critical regulatory link functions as a switch determining cell fate decisions including proliferation, differentiation, apoptosis, survival and oncogenic transformation. RAF1 activation initiates a mitogen-activated protein kinase (MAPK) cascade that comprises a sequential phosphorylation of the dual-specific MAPK kinases (MAP2K1/MEK1 and MAP2K2/MEK2) and the extracellular signal-regulated kinases (MAPK3/ERK1 and MAPK1/ERK2). The phosphorylated form of RAF1 (on residues Ser-338 and Ser-339, by PAK1) phosphorylates BAD/Bcl2-antagonist of cell death at 'Ser-75'. Phosphorylates adenylyl cyclases: ADCY2, ADCY5 and ADCY6, resulting in their activation. Phosphorylates PPP1R12A resulting in inhibition of the phosphatase activity. Phosphorylates TNNT2/cardiac muscle troponin T. Can promote NF-kB activation and inhibit signal transducers involved in motility (ROCK2), apoptosis (MAP3K5/ASK1 and STK3/MST2), proliferation and angiogenesis (RB1). Can protect cells from apoptosis also by translocating to the mitochondria where it binds BCL2 and displaces BAD/Bcl2-antagonist of cell death. Regulates Rho signaling and migration, and is required for normal wound healing. Plays a role in the oncogenic transformation of epithelial cells via repression of the TJ protein, occludin (OCLN) by inducing the up-regulation of a transcriptional repressor SNAI2/SLUG, which induces down-regulation of OCLN. Restricts caspase activation in response to selected stimuli, notably Fas stimulation, pathogen-mediated macrophage apoptosis, and erythroid differentiation

Subcellular location

CytoplasmCell membraneMitochondrionNucleus
Domains and Gene Ontology detail (39)

Domains & features

RBDProtein kinase

Gene Ontology

  • Ccytoplasm
  • Ccytosol
  • CGolgi apparatus
  • Cmitochondrial outer membrane
  • Cmitochondrion
  • Cnucleus
  • Cplasma membrane
  • Cpseudopodium
  • Fadenylate cyclase activator activity
  • FATP binding
  • Fenzyme binding
  • Fidentical protein binding

648 aa · 73 kDa · 2 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Kinase signallingUniProt · GO · ReactomeApoptosis & cell deathUniProt · GOImmune signallingUniProtTranscriptional regulationUniProt
View supporting evidence

Kinase signalling

  • ·Serine/threonine-protein kinase that acts as a regulatory link between the membrane-asso…
  • ·MAP kinase kinase kinase activity
  • ·protein kinase activity
  • ·protein serine kinase activity

Apoptosis & cell death

  • ·Serine/threonine-protein kinase that acts as a regulatory link between the membrane-asso…
  • ·apoptotic process
  • ·negative regulation of apoptotic process
  • ·regulation of apoptotic process

Immune signalling

  • ·Serine/threonine-protein kinase that acts as a regulatory link between the membrane-asso…

Transcriptional regulation

  • ·Serine/threonine-protein kinase that acts as a regulatory link between the membrane-asso…
View underlying pathways (17)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

RAP1AYWHAZHRASMAP2K1BRAFKSR1NRASKRASYWHAHYWHABRAF1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

1

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

regorafenib
ApprovedInhibitor

Serine/threonine-protein kinase RAF inhibitor

Appears in clinical studies involving colorectal cancer, metastatic colorectal cancer, colorectal neoplasm, neoplasm

Direct interaction with this protein · 1 of 18 recorded protein targets — broad pharmacology

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

RASopathy0.96

Genetic · overall 0.68

LEOPARD syndrome 20.94

Genetic literature · overall 0.71

Noonan syndrome 50.93

Genetic · overall 0.76

Noonan syndrome0.93

Genetic · overall 0.87

dilated cardiomyopathy0.85

Genetic · overall 0.69

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

hepatocellular carcinoma0.97

Clinical · overall 0.67

renal cell carcinoma0.95

Clinical · overall 0.58

colorectal cancer0.94

Clinical · overall 0.58

cancer0.18

Clinical · overall 0.63

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

Noonan syndrome with multiple lentigines0.78

Genetic literature

Show all associations
Noonan syndrome0.87
Noonan syndrome with multiple lentigines0.78
Noonan syndrome 50.76
LEOPARD syndrome 20.71
dilated cardiomyopathy0.69
RASopathy0.68
hepatocellular carcinoma0.67
cancer0.63
renal cell carcinoma0.58
colorectal cancer0.58

Open Targets ranks 3,149 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 9 total

BELVARAFENIBPhase 2

central nervous system neoplasm · central nervous system cancer · melanoma

LY-3009120Phase 1

cancer

XL-281Phase 1 2

colorectal cancer · melanoma

SORAFENIB TOSYLATEApproval

renal cell carcinoma · thyroid gland carcinoma · hepatocellular carcinoma

REGORAFENIBApproval

colorectal cancer · metastatic colorectal cancer · colorectal neoplasm

ARQ-736Phase 1

neoplasm

SORAFENIBApproval

renal cell carcinoma · hepatocellular carcinoma · renal cell carcinoma

TOVORAFENIBApproval

glioma · low grade glioma · low grade glioma

NAPORAFENIBPhase 3

melanoma · non-small cell lung carcinoma · non-small cell lung carcinoma

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC high confAB · UniProt loc med confAB · Human Protein Atlas locPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Safety liabilities

regulation of catalytic activityheart disease

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ACTIVE_NOT_RECRUITING · via regorafenib · NCT05395741

TERMINATED · via regorafenib · NCT02402036

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

Noonan syndromeWell supported
0.97
agreement 0.861.00
Genetic59%Pathway24%Animal model13%Literature5%Genetic literaturedup

Open Targets aggregate 0.87 · 4 independent evidence families · 1 not counted as duplicate

RASopathyWell supported
0.96
agreement 0.821.00
Genetic97%Literature3%Genetic literaturedup

Open Targets aggregate 0.68 · 2 independent evidence families · 1 not counted as duplicate

Noonan syndrome 5Well supported
0.94
agreement 0.821.00
Genetic85%Animal model14%Literature1%Genetic literaturedup

Open Targets aggregate 0.76 · 3 independent evidence families · 1 not counted as duplicate

LEOPARD syndrome 2Well supported
0.91
agreement 0.791.00
Genetic87%Animal model13%Literature1%Genetic literaturedup

Open Targets aggregate 0.71 · 3 independent evidence families · 1 not counted as duplicate

dilated cardiomyopathyWell supported
0.88
agreement 0.760.99
Genetic84%Animal model15%Literature1%Genetic literaturedup

Open Targets aggregate 0.69 · 3 independent evidence families · 1 not counted as duplicate

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

7

Recent activity around this target, drawn from one canonical event stream. Every item is reached through a drug that targets this protein, so each event is news about that drug rather than about the protein directly.

  1. New publication2025-01-06
    Efficacy of pembrolizumab in microsatellite-stable, tumor mutational burden-high metastatic colorectal cancer: genomic signatures and clinical outcomes.

    ESMO open · 2025 · 10 citations · Europe PMC · via regorafenib

  2. New publication2024-06-04
    Lenvatinib Plus Pembrolizumab Versus Standard of Care for Previously Treated Metastatic Colorectal Cancer: Final Analysis of the Randomized, Open-Label, Phase III LEAP-017 Study.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2024 · 69 citations · Europe PMC · via regorafenib

  3. New publication2020-08-11
    Targeted therapy for hepatocellular carcinoma.

    Signal transduction and targeted therapy · 2020 · 565 citations · Europe PMC · via regorafenib

  4. New publication2019-11-04
    Molecular targeted and immune checkpoint therapy for advanced hepatocellular carcinoma.

    Journal of experimental & clinical cancer research : CR · 2019 · 162 citations · Europe PMC · via regorafenib

  5. New publication2019-04-23
    Randomized Double-Blind Phase II Study of Regorafenib in Patients With Metastatic Osteosarcoma.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2019 · 199 citations · Europe PMC · via regorafenib

  6. New publication2016-12-06
    Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial.

    Lancet (London, England) · 2017 · 2,767 citations · Europe PMC · via regorafenib

  7. Regulatory approval2013-08-26

    Approval: Stivarga (EMA)

    ema · regulatory · ema · via regorafenib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.