Gene Therapies for Cardiomyopathies Market Poised for Rapid Expansion During the Forecast Period (2025-2034) with Advancements in AAV and CRISPR Platforms | DelveInsight

The gene therapies for cardiomyopathies market is poised for strong growth as advancements in genomic medicine accelerate the development of targeted treatments for inherited and acquired cardiac disorders. Additionally, emerging candidates such as LX2020 and LX2006 from Lexeo Therapeutics and AB-1002 from AskBio exemplify targeted, mechanism-driven strategies aimed at correcting or compensating for underlying genetic defects in hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy. 

LAS VEGAS, Dec. 15, 2025 /PRNewswire/ — DelveInsight’s Gene Therapies for Cardiomyopathies Market Insights report includes a comprehensive understanding of current treatment practices, emerging gene therapies for cardiomyopathies, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). 

Gene Therapies for Cardiomyopathies Market Summary

• The market size for gene therapies for cardiomyopathies in the leading markets is expected to grow significantly by 2034.
• The United States accounted for the highest gene therapies for cardiomyopathies treatment market size in 7MM in 2024, in comparison to the other major markets, i.e., EU4 countries, the United Kingdom, and Japan.
• In the US, the prevalence of cardiomyopathies is significant, with hypertrophic cardiomyopathy affecting approximately 1 in 500 individuals and dilated cardiomyopathy occurring in about 1 in 2,500. Restrictive cardiomyopathy, though less common, accounts for an estimated 2% to 5% of cardiomyopathy cases, underscoring the diverse burden of these heart muscle disorders.
• Leading gene therapies for cardiomyopathies companies developing emerging therapies, such as Lexeo Therapeutics, AskBio, Tenaya Therapeutics, Rocket Pharmaceuticals, and others, are developing new gene therapies for cardiomyopathies that can be available in the gene therapies for cardiomyopathies market in the coming years. 
• The promising gene therapies for cardiomyopathies in clinical trials include LX2020, LX2022, LX2006, AB-1002, TN-201, RP-A701, RP-A501, and others.

Discover the emerging gene therapies for cardiomyopathies @ New Gene Therapies for Cardiomyopathies

Key Factors Driving the Growth of the Gene Therapies for Cardiomyopathies Market 

Growing disease and genetic burden

Cardiomyopathies and heart failure are increasing globally, driven by aging populations, hypertension, diabetes, and obesity, which raises demand for disease-modifying therapies beyond conventional drugs and devices.

Advances in genomic diagnostics and precision medicine

Wider use of next‑generation sequencing, genomic panels, and AI‑enabled variant interpretation makes it easier to identify pathogenic mutations and stratify patients for gene therapy trials.

Technological progress in vectors and editing platforms

Improvements in cardiotropic adeno‑associated virus (AAV) vectors, lipid nanoparticles, and tissue‑specific promoters are enhancing cardiac delivery, expression efficiency, and safety profiles.

Clinical pipeline expansion and upcoming launches

The expected launch of gene therapies for cardiomyopathies such as LX2020, LX2022, and LX2006 (Lexeo Therapeutics), AB-1002 (AskBio), TN-201 (Tenaya Therapeutics), RP-A701 and RP-A501 (Rocket Pharmaceuticals), and others will change the dynamics of the market in the coming years.

Gene Therapies for Cardiomyopathies Market Analysis

Gene therapies for cardiomyopathies are assessed using a thorough clinical evaluation that incorporates patient history, genetic testing, and cardiac function analysis. Because no single diagnostic tool can determine treatment suitability for every individual, advanced molecular tests, especially next-generation sequencing, are critical for detecting pathogenic mutations and informing therapeutic decisions. Imaging modalities such as echocardiography and cardiac MRI play a central role in identifying structural and functional heart abnormalities. In contrast, biomarkers such as NT-proBNP and troponins provide further insight into disease burden. In the end, accurate genetic characterization remains fundamental to selecting candidates and achieving optimal results across hypertrophic, dilated, and restrictive cardiomyopathies.

Gene therapies are tailored to the cardiomyopathy subtype, the underlying genetic defect, and the patient’s clinical presentation, acknowledging that a universal treatment approach is not feasible. These therapies focus on correcting or counteracting defective genes to restore cardiac function and slow or prevent disease progression. Personalized treatment strategies, developed jointly by clinicians and patients and guided by genetic profiles, symptoms, and care goals, are essential. Investigational techniques include adeno-associated viral vectors for gene replacement, gene-silencing approaches to reduce expression of harmful proteins, and gene-editing platforms that directly repair mutations. Whether used independently or in combination, these modalities show strong potential to improve heart performance, mitigate complications, and offer long-lasting, disease-modifying benefits beyond current standard therapies.

New therapeutic candidates continue to broaden the field, reinforcing the promise of precision medicine approaches that aim to treat cardiomyopathies at their genetic origin rather than merely managing symptoms. Supportive interventions, such as lifestyle adjustments, ongoing clinical monitoring, and proactive management of complications, remain vital to achieving the best possible outcomes alongside gene-based treatments. Collectively, these developments mark a major shift in cardiomyopathy care, paving the way toward more effective and durable treatments, and potentially a cure as research progresses.

To know more about gene therapies for cardiomyopathies, visit @ Approved Gene Therapies for Cardiomyopathies 

Gene Therapies for Cardiomyopathies Competitive Landscape

The cardiomyopathy treatment landscape is evolving with gene therapy candidates such as LX2020, LX2022, and LX2006 from Lexeo Therapeutics and AB-1002 from AskBio, which aim to directly target the genetic basis of disease and provide durable, disease-modifying benefits beyond current treatment approaches.

Lexeo Therapeutics’ LX2020 is an experimental gene therapy targeting arrhythmogenic cardiomyopathy driven by PKP2 mutations. It uses an adeno-associated virus (AAV) vector to deliver a healthy PKP2 gene into cardiac muscle cells, restoring desmosomal integrity, slowing or halting disease progression, and offering long-lasting, disease-modifying benefits beyond current standard treatments.

Lexeo Therapeutics’ LX2006 is an investigational gene therapy being evaluated for cardiomyopathies linked to inherited genetic defects. Delivered through an AAV vector, it is engineered to insert a functional version of the faulty gene, directly addressing the root cause of the disease rather than only managing symptoms. By targeting the underlying pathology, it has the potential to restore cardiac function, prevent progression to heart failure, and significantly improve long-term outcomes—marking a shift toward durable, disease-modifying therapeutic strategies.

AskBio’s AB-1002 is another investigational AAV-based gene therapy for cardiomyopathies. It delivers the ubiquitin carboxyl-terminal hydrolase L1 (UBIQLN1) gene to restore heart function by correcting genetic mechanisms that contribute to disease progression. This precise, mechanism-driven approach is designed to produce long-lasting, disease-modifying effects, positioning it as a potentially transformative alternative to conventional therapies that offer only symptomatic relief.

The anticipated launch of these emerging therapies are poised to transform the gene therapies for cardiomyopathies market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the gene therapies for cardiomyopathies market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

Discover more about gene therapies for cardiomyopathies in development @ Gene Therapies for Cardiomyopathies Clinical Trials 

Recent Developments in the Gene Therapies for Cardiomyopathies Market

• In August 2025, Lexeo Therapeutics, in its second quarter 2025 results and operational highlights, reported that eight participants have been dosed in the HEROIC-PKP2 Phase I/II trial across low- and high-dose cohorts, with Cohort 3 still enrolling up to two additional participants.
• In August 2025, Lexeo Therapeutics, in its second quarter 2025 results and operational highlights, reported participation in the FDA’s Development Readiness Pilot (CDRP) program to accelerate  Chemistry, Manufacturing, and Controls (CMC) registration readiness and expects final alignment on the LX2006 registration study in the late third quarter to early fourth quarter 2025.
• In July 2025, Lexeo received Breakthrough Therapy Designation (BTD) from the US FDA for LX2006 based on interim clinical data showing meaningful improvements in cardiac and neurologic outcomes in patients with Friedreich’s ataxia. The therapy was also selected to participate in the FDA’s CMC program.
• In May 2025, Lexeo Therapeutics reported USD 80 million in equity financing to advance further the development of transformative genetic medicines for cardiovascular diseases.
• In May 2025, AskBio presented complete Phase I trial results of AB-1002 gene therapy in participants with congestive heart failure at the European Society of Cardiology Heart Failure Meeting.

What are Cardiomyopathies?

Cardiomyopathies are diseases of the heart muscle that impair its ability to pump blood effectively, leading to shortness of breath, fatigue, irregular heartbeat, and fluid buildup. The main forms include hypertrophic, dilated, restrictive, and arrhythmogenic cardiomyopathy. If not treated appropriately, these conditions may progress to heart failure, dangerous rhythm disturbances, stroke, or even sudden cardiac death. Gene therapy for cardiomyopathies focuses on correcting the underlying genetic defects to restore proper heart function. The goal is to reduce symptoms, halt or slow disease progression toward heart failure, and deliver long-lasting, disease-modifying benefits that surpass those of conventional therapies.

Gene Therapies for Cardiomyopathies Epidemiology Segmentation

The gene therapies for cardiomyopathies epidemiology section provides insights into the historical and current gene therapies for cardiomyopathies patient pool and forecasted trends for the leading markets. In Japan, hypertrophic cardiomyopathy has a prevalence of approximately 11.1 per 10,000 individuals.

The gene therapies for cardiomyopathies market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into:

• Total Prevalent Cases of Cardiomyopathies
• Total Diagnosed Prevalent Cases of Cardiomyopathies 
• Gender-specific Diagnosed Prevalent Cases of Cardiomyopathies
• Type-Specific Diagnosed Prevalent Cases of Cardiomyopathies
• Total Treated Cases of Cardiomyopathies 

Scope of the Gene Therapies for Cardiomyopathies Market Report

• Therapeutic Assessment: Gene Therapies for Cardiomyopathies current marketed and emerging therapies
• Gene Therapies for Cardiomyopathies Market Dynamics: Key Market Forecast Assumptions of Emerging Gene Therapies for Cardiomyopathies Drugs and Market Outlook
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL’s views, Analyst’s views, Gene Therapies for Cardiomyopathies Market Access and Reimbursement

Download the report to understand which factors are driving gene therapies for cardiomyopathies therapeutics market trends @ Gene Therapies for Cardiomyopathies Market Trends

Table of Contents

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