The encoded protein is a hepatokine that regulates lipid and glucose metabolism by inhibiting lipoprotein lipase activity and endothelial lipase, thereby affecting plasma triglyceride clearance and HDL cholesterol levels. Biallelic mutations cause familial hypobetalipoproteinemia type 2, inherited in an autosomal recessive pattern. This gene shows very low constraint against loss-of-function variants in the general population.

OMIMResearchSummary from RefSeq, OMIM, UniProt
DNmechanismARLOEUF 1.301 OMIM phenotype
Clinical SummaryANGPTL3
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Gene-Disease Validity (ClinGen)
familial hypobetalipoproteinemia 2 · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.30LOEUF
pLI 0.000
Z-score 0.43
OE 0.90 (0.641.30)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-0.09Z-score
OE missense 1.02 (0.911.13)
232 obs / 228.0 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.90 (0.641.30)
00.351.4
Missense OE1.02 (0.911.13)
00.61.4
Synonymous OE1.09
01.21.6
LoF obs/exp: 21 / 23.2Missense obs/exp: 232 / 228.0Syn Z: -0.63
DN
0.6939th %ile
GOF
0.5955th %ile
LOF
0.3066th %ile

The highest-scoring mechanism for this gene is dominant-negative.

DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

ANGPTL3 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Hypocholesterolemia

Physiological Effects of ANGPTL3 Variants in Humans

RECRUITING
NCT04234724Phase NAUniversity of PennsylvaniaStarted 2020-12-01
Kinetics test, oral fat tolerance test, and an oral glucose tolerance test.
Diabetic Kidney Disease (DKD)

Study to Assess the Effects of Angiopoietin-like Protein 3 (ANGPTL3) Inhibition in Adult Participants With Diabetic Kidney Disease

RECRUITING
NCT07271186Phase PHASE2Regeneron PharmaceuticalsStarted 2026-01-09
ALN-ANG3EvinacumabALN-ANG3 placebo
CardiovascularMetabolic DiseaseDyslipidemias

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

RECRUITING
NCT07491172Phase PHASE1CRISPR Therapeutics AGStarted 2024-06-21
CTX310
Homozygous Familial Hypercholesterolemia

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

NOT YET RECRUITING
NCT07473843Phase PHASE3Arrowhead PharmaceuticalsStarted 2026-03
Zodasiran
Homozygous Familial Hypercholesterolemia

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

RECRUITING
NCT07037771Phase PHASE3Arrowhead PharmaceuticalsStarted 2025-06-17
zodasiran InjectionPlacebo
Severe Hypertriglyceridemia

A Study of Solbinsiran (LY3561774) in Participants With Severe Hypertriglyceridemia

RECRUITING
NCT07269210Phase PHASE2Eli Lilly and CompanyStarted 2025-12-08
SolbinsiranPlacebo
Non-alcoholic Fatty LiverAtherogenic DyslipidemiaInsulin Resistance

Genetic Pathways Leading to Fatty Liver and Atherogenic Dyslipidemia

ENROLLING BY INVITATION
NCT04209816Marja-Riitta TaskinenStarted 2019-12-01
Lipoprotein kinetics
Homozygous Familial Hypercholesterolemia (HoFH)Coronary Computed Tomography Angiography

Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

RECRUITING
NCT07447648Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)Started 2026-04-30
Homozygous Familial Hypercholesterolemia

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

ACTIVE NOT RECRUITING
NCT05217667Phase PHASE2Arrowhead PharmaceuticalsStarted 2022-04-22
ARO-ANG 3 Injection
Homozygous Familial Hypercholesterolemia (HoFH)

A Dose-exploration Study of EDP167 in HoFH

RECRUITING
NCT07489209Phase PHASE2Eddingpharm (Zhuhai) Co., Ltd.Started 2026-02-06
EDP167
HypercholesterolemiaHypertriglyceridemiaRefractory Hyperlipidemia

Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

RECRUITING
NCT06451770Phase PHASE1Verve Therapeutics, Inc.Started 2024-10-30
VERVE-201
Pancreatic Ductal AdenocarcinomaPancreatic CancerPancreas Cancer

Cholesterol Disruption in Combination With the Standard of Care in Patients With Advanced Pancreatic Adenocarcinoma

ACTIVE NOT RECRUITING
NCT04862260Phase EARLY_PHASE1CHU de Quebec-Universite LavalStarted 2021-10-04
Cholesterol metabolism disruption
Clinical Literature
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