Dilated Cardiomyopathy Drug Pipeline — PatSnap Eureka
Dilated Cardiomyopathy Drug Pipeline: Gene Therapy, SERCA2a & Emerging Targets
DCM affects approximately 1 in 250–2,500 adults and represents a leading cause of heart failure and cardiac transplantation. PatSnap Eureka maps the full patent landscape—from AAV-based RBM20 gene replacement and PLN/SERCA2a axis modulation to HDAC6 inhibitors and non-viral AOC delivery platforms.
Key Genetic Drivers & Therapeutic Targets in DCM
Patent and literature records from this dataset identify a heterogeneous set of genetic and molecular mechanisms driving DCM. The targets below represent the highest-signal opportunities based on assignee convergence and IP activity.
RBM20 — Three Orthogonal Intervention Strategies
RBM20 (RNA-binding motif protein 20) governs titin (TTN) splicing; loss-of-function mutations cause aberrant TTN isoform expression and DCM. PatSnap Eureka identifies filings from AAVantiBio, University of Florida, University of Texas, and Stanford collectively addressing this target via AAV gene replacement, CRISPR/Cas9 editing, and small molecule upregulation—a convergence of modalities that signals increasing competitive intensity.
AAV · CRISPR · Small Molecule — all activePLN / SERCA2a — Most Convergent Mechanistic Opportunity
Phospholamban (PLN) is a critical regulator of the SR Ca²⁺-ATPase SERCA2a pump. PLN mutations including Arg14del, Arg9Cys, and Arg25Cys are specifically cited as DCM-associated alleles. Both life sciences IP leaders Ionis Pharmaceuticals (ASO) and Avidity Biosciences (AOC-siRNA) are pursuing PLN knockdown. Reducing PLN expression is described as improving diastolic calcium uptake, ejection fraction, and LV volumes.
ASO + AOC — dual platform convergenceHDAC6 — Broadest Multi-Jurisdictional Small Molecule Coverage
Tenaya Therapeutics, Inc. has the broadest active multi-jurisdictional patent coverage for an oral small molecule DCM therapy in this dataset. HDAC6 inhibitor filings span WO, US, CA, AU, BR, and JP jurisdictions. Patent language references treating human patients with reduced ejection fraction, suggesting IND-stage development intent. The mechanism involves cytoskeletal/microtubule remodeling.
6 jurisdictions · oral administration routeMyosin Activators — Broadest Small Molecule Patent Family
MyoKardia, Inc. (now part of Bristol Myers Squibb) has the most frequently appearing assignee position for small molecule DCM approaches. 4-methylsulfonyl piperidine urea compounds are described as myosin-activating agents targeting left and/or right ventricular systolic dysfunction. Active grants span IL, SG, NZ, CA, and PH jurisdictions, with a priority date tracing to a 2015 US provisional application.
5 active grant jurisdictions · 2015–2024LMNA & LINC Complex — Mechanistically Distinct DCM Subtype
LMNA mutations cause secondary force generation deficits and nuclear envelope dysfunction in DCM. Cornell University's US patent describes treating laminopathies by combining DDR inhibitors with microtubule stabilizing agents and LINC complex disruptors. NuevoCor Pte. Ltd. has separately filed a 2025 WO patent for LINC complex inhibitors in DCM not associated with laminopathy, representing an emerging structural biology-based intervention.
LINC inhibition · non-laminopathy DCMCD36 — Genotype-Guided Therapeutic Stratification
The Broad Institute, Inc. and The General Hospital Corporation have filed patents describing CD36 loss-of-function mutations as predictors of differential treatment response in DCM/HF. This positions CD36 genotyping as a companion diagnostic rather than a direct therapeutic target, signalling a movement toward genotype-stratified therapeutic enrollment that could reshape clinical trial design for DCM gene therapies.
Companion diagnostic · Broad InstituteFrom AAV Gene Delivery to Non-Viral AOC Platforms
The DCM therapeutic landscape is undergoing a significant mechanistic shift. AAV-based gene delivery remains the most patent-active modality in this dataset. AAVantiBio, Inc. and the University of Florida Research Foundation have filed patents across US, WO, CA, and AU jurisdictions claiming rAAV-mediated delivery of full-length human RBM20. Some embodiments describe co-administration of an rAAV silencing construct alongside the replacement construct—an allele-silencing plus replacement dual approach that signals a maturation of DCM gene therapy beyond simple gene addition.
CRISPR/Cas9 genomic editing of pathogenic RBM20 mutations is being pursued by the University of Texas System using sgRNA/Cas9 systems in human cells and mouse models. This approach targets the causative mutation rather than providing a gene replacement, with filings pending in both US and AU jurisdictions.
RNA-targeting approaches represent a rapidly evolving modality. Ionis Pharmaceuticals' PLN-targeting ASO filing explicitly lists DCM caused by TTN, LMNA, RBM20, SCN5A, MYH7, TNNT2, and TPM1 mutations as target indications—suggesting that PLN axis modulation may provide functional benefit across genetically heterogeneous DCM as a disease-modifying co-therapy. According to WIPO filing data, RNA-targeting cardiac patents have accelerated significantly since 2020.
The AOC platform (Avidity Biosciences) uses an anti-transferrin receptor antibody conjugated to PLN siRNA for cardiomyocyte-selective delivery without viral vectors. If clinically validated, this could unlock a broadly applicable delivery scaffold for cardiac RNA therapeutics. The PatSnap analytics platform tracks delivery modality differentiation across all active DCM assignees.
Peptide biologics include HMGB1 fragment peptides (Osaka University/Genomix Co.) that improve cardiac function and reduce myocardial fibrosis in DCM animal models, neuregulin-1 fusion proteins (Salbris Biotherapeutics) targeting ErbB4-mediated cardiac fibrosis signalling, and elamipretide (Stealth BioTherapeutics) for DMD-associated DCM in combination with exon-skipping ASOs.
The NIH and international cardiovascular research bodies continue to fund foundational DCM mechanistic work that underpins many of these patent strategies. For a broader view of the PatSnap platform's capabilities across cardiac indications, see the full product suite.
Patent Activity & Assignee Landscape in the DCM Pipeline
Inline analysis derived from PatSnap Eureka patent and literature records. All values reflect the retrieved dataset snapshot.
DCM Patent Families by Therapeutic Modality
AAV gene therapy leads patent activity in the DCM pipeline, with small molecules second and RNA/ASO/AOC approaches growing rapidly as non-viral delivery matures.
Leading Assignee Jurisdiction Coverage
Tenaya Therapeutics leads with 6 active patent jurisdictions for HDAC6 inhibitors; MyoKardia holds 5 active grants; AAVantiBio covers 3 jurisdictions for RBM20 gene therapy.
Commercial Biotech & Academic IP Holders in DCM
Retrieved results reveal a predominantly patent-driven dataset with commercial IP from biopharma companies and academic spinouts, supported by a smaller number of academic papers.
Track every DCM patent filer in real time
PatSnap Eureka monitors new filings, grants, and jurisdiction expansions across all DCM assignees automatically.
Emerging Directions & IP Strategy Signals
Retrieved results signal several combination strategies and emerging directions that will shape competitive positioning in DCM drug development.
Dual AAV Strategy: Replace + Silence
The University of Florida and AAVantiBio RBM20 filings explicitly describe co-administration of an rAAV silencing construct alongside an RBM20 replacement construct. This allele-replacement-plus-silencing design may address dominant-negative RBM20 mutations and signals maturation of the DCM gene therapy paradigm beyond simple gene addition. Tracking this via PatSnap analytics reveals no direct competitor filings with identical dual-construct designs.
Non-Viral Delivery Challenging AAV Hegemony
Avidity Biosciences' AOC platform and Icahn School of Medicine's modRNA approach are actively challenging AAV dominance in cardiac gene therapy. The AOC platform's use of transferrin receptor targeting for cardiomyocyte selectivity, if clinically validated, could unlock a more broadly applicable delivery scaffold for cardiac RNA therapeutics beyond PLN, avoiding AAV-related immunogenicity concerns entirely. The PatSnap customer base includes teams tracking this delivery platform shift.
Multi-Genotype PLN Coverage via Single ASO Platform
The Ionis PLN-targeting ASO filing explicitly lists DCM caused by TTN, LMNA, RBM20, SCN5A, MYH7, TNNT2, and TPM1 mutations as target indications—suggesting PLN axis modulation may provide functional benefit across genetically heterogeneous DCM as a disease-modifying co-therapy to genotype-specific correction. This multi-genotype coverage claim is strategically significant for IP freedom-to-operate analysis.
Academic Institutions as Foundational IP Sources
The Broad Institute, Stanford, University of Texas, and VU Amsterdam continue to generate foundational IP in DCM genetic architecture and mechanistic biology. Early-stage licensing or collaboration strategies focused on these institutions may provide access to genotype-specific treatment claims that will underpin precision DCM medicine. According to EPO filing data, academic cardiac gene therapy filings have increased year-on-year since 2020.
From Preclinical to Phase 1: What the Data Shows
Retrieved results contain limited direct clinical evidence. The dataset is predominantly patent-driven, with commercial IP from biopharma companies and academic spinouts. No regulatory submissions, Phase 2/3 trial outcomes, or approved gene therapy/protein replacement products for DCM are identifiable in the retrieved dataset.
AZD3427 (AstraZeneca) represents the strongest clinical translation signal: a CN pending filing describes a dosing regimen study with AZD3427 (heterodimer relaxin fusion protein) in a Phase 1 setting, including PK profiles from non-human primates and early human cohorts (single and multiple ascending dose).
Tenaya Therapeutics HDAC6 inhibitors: Patent language references "treating a human patient" with reduced ejection fraction, suggesting IND-stage development. The multi-jurisdictional patent activity (WO, US, CA, AU, BR, JP) and oral administration route indicate advanced development intent, though no clinical trial data are present in the retrieved texts.
Longeveron allogeneic MSCs: The AU filing (2025) references immune system modulation—specifically exhausted B-cell reduction—in non-ischemic DCM subjects as a described therapeutic mechanism, which may reflect clinical observations.
Cardiac contractility modulation (CCM): Two retrieved papers describe CCM as an adjunct device therapy for heart failure with narrow QRS, including a case of TTN-variant noncompaction cardiomyopathy that responded favorably. The WHO classifies DCM as a significant global cardiovascular disease burden warranting novel therapeutic approaches.
For comprehensive clinical trial tracking alongside patent intelligence, the PatSnap life sciences platform integrates both patent and clinical data streams. Researchers can also access raw data via PatSnap's open API for custom pipeline analysis.
Dilated Cardiomyopathy Drug Pipeline — key questions answered
Among the most prominently featured targets in this dataset are RBM20, the PLN/SERCA2a axis, TTN (titin), LMNA (Lamin A/C), HDAC6, and CD36. RBM20 in particular is addressed by three orthogonal strategies: AAV gene replacement, CRISPR/Cas9 editing, and small molecule upregulation, signalling increasing competitive intensity around this genetically defined DCM subtype.
Phospholamban (PLN) is a critical regulator of the SR Ca²⁺-ATPase SERCA2a pump. PLN mutations (e.g., Arg14del, Arg9Cys, Arg25Cys) are specifically cited as DCM-associated targets. Reducing PLN expression via ASO or AOC strategies is described as improving diastolic calcium uptake, ejection fraction, and LV volumes. Both Ionis Pharmaceuticals (ASO) and Avidity Biosciences (AOC-siRNA) are pursuing PLN knockdown, making this the most convergent mechanistic opportunity in the dataset.
AAVantiBio, Inc. and the University of Florida Research Foundation have filed patents across multiple jurisdictions (US, WO, CA, AU) claiming rAAV-mediated delivery of full-length human RBM20 to treat DCM caused by RBM20 mutations. Some embodiments describe co-administration of an rAAV silencing construct alongside the replacement construct, suggesting an allele-silencing plus replacement dual approach. Evidence is patent-based; stage appears preclinical/IND-enabling.
MyoKardia, Inc. (now part of Bristol Myers Squibb) is the most frequently appearing assignee for small molecule DCM approaches, with active patent grants in IL, SG, NZ, CA, and PH jurisdictions covering 4-methylsulfonyl piperidine urea myosin activators. Tenaya Therapeutics, Inc. is the second-most prolific DCM-specific assignee, with HDAC6 inhibitor filings in WO, US, CA, AU, BR, and JP, with a development profile suggesting proximity to clinical stage.
Avidity Biosciences' anti-transferrin receptor antibody conjugated to PLN siRNA (AOC platform) represents an emerging non-viral, receptor-mediated delivery approach for the SERCA2a axis. Icahn School of Medicine at Mount Sinai has also filed a WO patent claiming modified mRNA (modRNA) encoding pip4k2c for DCM treatment via non-viral mRNA delivery to cardiac tissue. These approaches could compete with or complement AAV-based strategies and avoid AAV-related immunogenicity concerns.
AZD3427 (heterodimer relaxin fusion protein, AstraZeneca) represents the strongest clinical translation signal in the dataset. A CN pending filing describes a dosing regimen study with AZD3427 in a Phase 1 setting, including PK profiles from non-human primates and early human cohorts (single and multiple ascending dose). No regulatory submissions, Phase 2/3 trial outcomes, or approved gene therapy/protein replacement products for DCM are identifiable in the retrieved dataset.
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References
- Methods and compositions for treating RBM20 related cardiomyopathy with a viral vector — AAVantiBio, Inc., 2025, US [Patent]
- Methods and compositions for treating RBM20 related cardiomyopathy with a viral vector — University of Florida Research Foundation, 2023, WO [Patent]
- Methods and compositions for treating RBM20 related cardiomyopathy with a viral vector — AAVantiBio, Inc., 2024, AU [Patent]
- Gene delivery systems for treatment of heart failure — Icahn School of Medicine at Mount Sinai, 2021, WO [Patent]
- Beneficial effects of intramyocardial mesenchymal stem cells and VEGF165 plasmid injection in rats with furazolidone induced dilated cardiomyopathy — Dalian University Affiliated Zhongshan Hospital, 2015 [Paper]
- Genomic editing of RBM20 mutations — The Board of Regents of the University of Texas System, 2024, US [Patent]
- Genomic editing of RBM20 mutations — The Board of Regents of the University of Texas System, 2024, AU [Patent]
- Compounds and methods for reducing PLN expression — Ionis Pharmaceuticals, Inc., 2025, CN [Patent]
- Compositions comprising PLN-targeting anti-transferrin receptor antibody-polynucleotides and methods of use thereof to treat cardiomyopathy — Avidity Biosciences, Inc., 2025, US [Patent]
- Allele-specific RNA silencing for the treatment of hypertrophic cardiomyopathy — President and Fellows of Harvard College, 2015, CA [Patent]
- Improved compound for the treatment of heart failure — Medizinische Hochschule Hannover, 2020, CL [Patent]
- HDAC6 inhibitors for use in the treatment of dilated cardiomyopathy — Tenaya Therapeutics, Inc., 2022, WO [Patent]
- HDAC6 inhibitors for treatment of dilated cardiomyopathy — Tenaya Therapeutics, Inc., 2024, US [Patent]
- 4-methylsulfonyl-substituted piperidine urea compounds useful for the treatment of cardiac disorders such as dilated cardiomyopathy — MyoKardia, Inc., 2023, CA [Patent]
- Treatment of cardiomyopathy not associated with laminopathy — NuevoCor Pte. Ltd., 2025, WO [Patent]
- CILP-1 inhibitors for use in the treatment of dilated cardiomyopathy — National Healthcare Research Institute, 2023, CN [Patent]
- Therapeutic agent for cardiomyopathy, old myocardial infarction and chronic heart failure — Osaka University, 2024, US [Patent]
- Methods for Treating Fibrosis and Arrhythmia with Neuregulin-1 Fusion Proteins — Salbris Biotherapeutics, Inc., 2025, JP [Patent]
- Methods and compositions for the treatment of muscular dystrophy — Stealth BioTherapeutics Inc., 2021, WO [Patent]
- Methods of using human mesenchymal stem cells to effect cellular and humoral immunity — Longeveron Inc., 2025, AU [Patent]
- lncRNA Meg3 for the treatment and diagnosis of cardiac remodeling — Medizinische Hochschule Hannover, 2021, JP [Patent]
- Regression of Electrocardiographic Left Ventricular Hypertrophy and Strain Pattern Using Pharmacotherapy in a Patient With Dilated Cardiomyopathy — Kyushu University, 2021 [Paper]
- Normalization of ventricular function after cardiac contractility modulation in noncompaction cardiomyopathy heterozygous positive for a pathologic TTN gene variant — University of Kentucky, 2022 [Paper]
- Dosing regimen using heterodimer relaxin fusion protein — AstraZeneca (Sweden) Ltd., 2025, CN [Patent]
- World Intellectual Property Organization (WIPO) — Patent filing data and cardiovascular IP trends
- National Institutes of Health (NIH) — Dilated cardiomyopathy research funding and disease background
- European Patent Office (EPO) — Academic cardiac gene therapy patent filing trends
- World Health Organization (WHO) — Cardiovascular disease global burden classification
All data and statistics on this page are sourced from the references above and from PatSnap's proprietary innovation intelligence platform. This report is derived from a limited set of patent and literature records retrieved across targeted searches and represents a snapshot of innovation signals within this dataset only.
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