BAG3

Chr 10AD

BAG cochaperone 3

Also known as: BAG-3, BIS, CAIR-1, CMD1HH, CMT2JJ, HMND15, MFM6

BAG proteins compete with Hip for binding to the Hsc70/Hsp70 ATPase domain and promote substrate release. All the BAG proteins have an approximately 45-amino acid BAG domain near the C terminus but differ markedly in their N-terminal regions. The protein encoded by this gene contains a WW domain in the N-terminal region and a BAG domain in the C-terminal region. The BAG domains of BAG1, BAG2, and BAG3 interact specifically with the Hsc70 ATPase domain in vitro and in mammalian cells. All 3 proteins bind with high affinity to the ATPase domain of Hsc70 and inhibit its chaperone activity in a Hip-repressible manner. [provided by RefSeq, Jul 2008]

GeneReviewsOMIMResearchGenerating clinical summary…
MultiplemechanismADLOEUF 0.454 OMIM phenotypes
Clinical SummaryBAG3
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Gene-Disease Validity (ClinGen)
myofibrillar myopathy · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.62) — some intolerance to loss-of-function variants.
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ClinVar Variants
160 unique Pathogenic / Likely Pathogenic· 750 VUS of 1397 total submissions
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Clinical Trials
6 active or recruiting trials — potential therapeutic options may be available
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GeneReview available — BAG3
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint?
0.45LOEUF
pLI 0.617
Z-score 3.37
OE 0.20 (0.100.45)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint?
-0.74Z-score
OE missense 1.11 (1.021.21)
376 obs / 337.9 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios?
LoF OE?0.20 (0.100.45)
00.351.4
Missense OE?1.11 (1.021.21)
00.61.4
Synonymous OE?1.01
01.21.6
LoF obs/exp: 4 / 20.5Missense obs/exp: 376 / 337.9Syn Z: -0.13
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveBAG3-related myofibrillar myopathyOTHERAD
definitiveBAG3-related dilated cardiomyopathyOTHERAD

This gene — mechanism propensity

DN
0.4389th %ile
GOF
0.4183th %ile
LOF
0.52top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to loss-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOF1 literature citation · 90% of P/LP variants are LoF · LOEUF 0.45 · ClinGen HI: Sufficient evidence for dosage pathogenicity
GOF1 literature citation

Literature Evidence

GOFMutations in either the IPV or BAG domain of BAG3 cause a dominant form of myopathy, characterized by protein aggregation in both skeletal and cardiac muscle tissues.1
LOFGenome-wide studies of copy number variation and exome sequencing identify rare variants in BAG3 as a cause of dilated cardiomyopathy2

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

1397 submitted variants in ClinVar

Classification Summary

Pathogenic108
Likely Pathogenic52
VUS750
Likely Benign354
Benign27
Conflicting100
108
Pathogenic
52
Likely Pathogenic
750
VUS
354
Likely Benign
27
Benign
100
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
94
6
8
0
108
Likely Pathogenic
50
2
0
0
52
VUS
22
685
41
2
750
Likely Benign
0
18
45
291
354
Benign
0
1
24
2
27
Conflicting
100
Total1667121182951,391

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

33 pathogenic / likely-pathogenic (of 41) ClinVar copy-number / structural variants overlap BAG3 — these span large chromosomal regions, not the gene specifically, and are excluded from the counts above. Explore in CNV tools →

Protein Context — Lollipop Plot

BAG3 · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Dilated Cardiomyopathy (DCM)

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

RECRUITING
NCT07137338Phase PHASE1Rocket Pharmaceuticals Inc.Started 2026-06
RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
BAG3 Mutation Associated Dilated Cardiomyopathy

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

RECRUITING
NCT07218887Phase PHASE1, PHASE2Alexion Pharmaceuticals, Inc.Started 2025-10-24
ALXN2350
Cardiomyopathy, DilatedBcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

ACTIVE NOT RECRUITING
NCT05981092Alexion Pharmaceuticals, Inc.Started 2022-10-14
Dilated Cardiomyopathy (DCM)BAG3 Mutation Associated Dilated Cardiomyopathy

An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)

RECRUITING
NCT07426419Phase PHASE1, PHASE2Affinia TherapeuticsStarted 2026-06
AFTX-201
Arrhythmogenic Cardiomyopathies

Tissue and Metabolic Characterization of Arrhythmogenic Cardiomyopathies by Hybrid PET-MRI Imaging, Impact of the Observed Profiles on the Phenotype and on the Evolution of Cardiomyopathy

RECRUITING
NCT05450783Nantes University HospitalStarted 2022-09-01
Biocollection
Dilated Cardiomyopathy

Observational Study of Natural History of BAG3 Gene Mutation-Associated Dilated Cardiomyopathy in Chinese Adults

NOT YET RECRUITING
NCT07646600AstraZenecaStarted 2026-06-29
Observational, None intervention