Gene Therapies for Cardiomyopathies - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights:

• Cardiomyopathies are heart muscle disorders that weaken the heart's pumping ability, causing symptoms like breathlessness, fatigue, palpitations, and swelling. Major subtypes include hypertrophic, dilated, restrictive, and arrhythmogenic cardiomyopathy. Without proper management, they can lead to heart failure, arrhythmias, stroke, or sudden cardiac death.
• Gene therapies for cardiomyopathies target faulty genes to restore heart function, aiming to reduce symptoms, prevent progression to heart failure, and provide long-term disease-modifying benefits beyond standard treatments.
• Our secondary research indicates that in France, cardiomyopathies affect around 809.5 per million people, with dilated cardiomyopathy being the most common at 428.4 per million, followed by other cardiomyopathies at 253.4 per million, hypertrophic cardiomyopathy at 100.8 per million, and restrictive cardiomyopathy at 26 per million. This distribution underscores the heterogeneity of these disorders and highlights the disproportionate burden of dilated cardiomyopathy within the overall landscape.
• 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.
• 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.
• A key unmet need in cardiomyopathies is the absence of approved gene therapies, leaving patients without targeted options that address the underlying genetic causes of disease and limiting treatment to symptom management rather than long-term, disease-modifying solutions.

DelveInsight's comprehensive report titled "Gene Therapy for Cardiomyopathies - Market Insights, Epidemiology, and Market Forecast - 2034" offers a detailed analysis of gene therapies of cardiomyopathies. The report presents historical and projected epidemiological data covering 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, and total treated cases of cardiomyopathies. In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements gene therapies of cardiomyopathies. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Gene Therapy for Cardiomyopathies Overview

Gene therapies for cardiomyopathies represent a transformative approach targeting the genetic defects that weaken the heart muscle and impair its ability to pump blood effectively. These therapies are designed to address inherited forms of hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy by correcting or compensating for faulty genes. Clinical manifestations of cardiomyopathies vary, ranging from mild symptoms such as fatigue, palpitations, or shortness of breath to severe complications including heart failure, arrhythmias, stroke, or sudden cardiac death. While each subtype arises from distinct genetic mechanisms, disrupted sarcomere function, mitochondrial dysfunction, and abnormal myocardial remodeling are central to disease progression. Gene therapies aim to intervene at the molecular level, offering the potential for durable, disease-modifying effects. Early identification of eligible patients and timely intervention are critical, as untreated or progressive cases may result in advanced heart failure or life-threatening outcomes.

Gene therapies of Cardiomyopathies Diagnosis and Treatment Overview

Gene therapies for cardiomyopathies are evaluated through a comprehensive clinical assessment, supported by detailed patient history, genetic testing, and cardiac function studies. While no single test can determine eligibility for all patients, molecular diagnostics such as next-generation sequencing help identify disease-causing mutations and guide therapeutic targeting. Cardiac imaging, including echocardiography and cardiac MRI, is essential for assessing structural and functional abnormalities, while biomarkers such as NT-proBNP and troponins provide additional insights into disease severity. Ultimately, precise genetic characterization remains the cornerstone for determining suitability and optimizing outcomes with gene therapy in hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy.

Gene therapy for cardiomyopathies is tailored to disease subtype, causative mutation, and clinical severity, recognizing that no single approach suits all patients. These therapies aim to correct or offset faulty genes responsible for hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy, with the goal of restoring heart muscle function and preventing progression. Personalized treatment strategies are essential, developed through collaboration between patients and clinicians and guided by genetic profiling, clinical features, and individual goals of care. Investigational approaches include adeno-associated virus vectors to deliver functional genes, gene silencing to suppress harmful protein production, and gene editing to directly repair mutations. Applied individually or in combination, these strategies hold promise to enhance cardiac performance, limit complications, and deliver durable, disease-modifying benefits well beyond conventional management.

Cardiomyopathies Epidemiology

The epidemiology section of the gene therapies of cardiomyopathies market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the total prevalence rate of cardiomyopathies, supported by relevant tables and graphs to provide a clear and concise understanding of the data. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

• Hypertrophic cardiomyopathy is the most common inherited heart disease in Germany, with reported prevalence estimates ranging from 1 in 500 to 1 in 3,000 individuals, reflecting variability between population-based screening data and clinically diagnosed cases, and underscoring its significant impact on cardiac health and outcomes.
• In France, cardiomyopathies show a gender-specific distribution, with approximately 62% of cases occurring in males, underscoring a notable disparity that highlights the importance of tailored approaches in early detection, gene therapy development, and long-term disease management.
• In the UK, hypertrophic cardiomyopathy has a prevalence of approximately 4.15 per 10,000 individuals, underscoring its clinical significance and highlighting the need for improved early detection, advancement of gene therapy approaches, and optimized long-term disease management.
• In Japan, hypertrophic cardiomyopathy has a prevalence of approximately 11.1 per 10,000 individuals, underscoring its clinical significance and highlighting the need for improved early detection, advancement of gene therapy approaches, and optimized long-term disease management.

Gene Therapy for Cardiomyopathies Market Outlook

The gene therapies of cardiomyopathies market is further expected to increase by the major drivers, such as the rising prevalent population, technological advancements, and upcoming therapies in the forecast period (2025-2034).

Gene therapies for cardiomyopathies are focused on directly addressing the underlying genetic mutations that drive disease progression, aiming to restore normal heart muscle function and prevent long-term complications. Current investigational strategies include adeno-associated virus-based vectors delivering functional gene copies, gene-silencing approaches to reduce harmful protein expression, and gene-editing platforms designed to repair pathogenic mutations. These innovative modalities are being explored across subtypes such as hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy, offering the potential for durable, disease-modifying outcomes. Emerging candidates continue to expand the therapeutic landscape, underscoring the promise of precision medicine approaches that move beyond conventional management. Complementary measures such as lifestyle modifications, clinical monitoring, and management of associated complications remain essential to optimize outcomes alongside gene therapy interventions. Together, these advancements highlight a paradigm shift in the treatment of cardiomyopathies, from symptom control to targeting disease at its genetic root.

With ongoing research and continued dedication, the future holds promise for even more effective treatments and, ultimately, a potential cure for this challenging condition. According to DelveInsight, the gene therapies of cardiomyopathies market in the 7MM is expected to change significantly during the forecast period (2025-2034).

Gene Therapy for Cardiomyopathies Drug Chapters

Emerging Drugs

LX2020: Lexeo Therapeutics

LX2020, developed by Lexeo Therapeutics, is an investigational gene therapy for arrhythmogenic cardiomyopathy caused by PKP2 mutations. Using an adeno-associated virus vector to deliver a functional PKP2 gene to heart muscle cells, it aims to restore desmosomal function, prevent disease progression, and provide durable, disease-modifying benefits beyond conventional therapies.

• 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 plans to share an interim clinical data update from the HEROIC-PKP2 Phase I/II trial in the second half of 2025.
• In January 2025, Lexeo Therapeutics reported its participation at the 43rd Annual J.P. Morgan Healthcare Conference.
• In December 2023, Lexeo Therapeutics reported that the US FDA granted both Orphan Drug Designation (ODD) and Fast Track Designation (FTD) to LX2020, its AAV-based gene therapy candidate for PKP2-arrhythmogenic cardiomyopathy.

LX2006: Lexeo Therapeutics

LX2006 is an investigational gene therapy being studied for the treatment of cardiomyopathies caused by genetic mutations. Delivered using an adeno-associated virus vector, it is designed to introduce a functional copy of the defective gene, directly addressing the underlying cause of disease rather than just alleviating symptoms. This targeted approach has the potential to restore heart muscle function, prevent progression to heart failure, and significantly improve long-term patient outcomes, representing a shift toward durable, disease-modifying treatment strategies.

• 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 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 equity financing to further advance the development of transformative genetic medicines for cardiovascular diseases.
• In April 2025, Lexeo reported positive interim data of LX2006 across both the Lexeo-sponsored SUNRISE-FA Phase I/II clinical trial and the Weill Cornell Medicine investigator-initiated Phase Ia trial.
• In April 2024, Lexeo Therapeutics was granted FTD by the US FDA for LX2006, an AAV-based gene therapy candidate for the treatment of Friedreich's ataxia cardiomyopathy.
• In June 2021, Lexeo Therapeutics received Rare Pediatric Disease Designation (RPDD) and ODD from the US FDA for LX2006, an investigational gene therapy for the treatment of Friedreich's ataxia cardiomyopathy.
• Lexeo Therapeutics reported that LX2006 received FDA Regenerative Medicine Advanced Therapy (RMAT) designation along with Orphan Medicinal Product designation from the European Commission (EC).
• Lexeo Therapeutics reported that it expects to initiate a registration study in early 2026 with a potential efficacy readout in 2027.

AB-1002: AskBio

AB-1002, developed by AskBio, is an investigational gene therapy for the treatment of cardiomyopathies. It utilizes an adeno-associated virus (AAV) vector to deliver the ubiquitin carboxyl-terminal hydrolase L1 (UBIQLN1) gene, aiming to restore cardiac function by addressing the underlying genetic mechanisms driving disease progression. This targeted, mechanism-based approach is designed to provide durable, disease-modifying benefits, offering a transformative alternative to conventional symptomatic treatments.

• 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.
• In February 2025, AskBio reported that the first participant was randomized in Europe in GenePHIT, its Phase II clinical trial of AB-1002 for the treatment of congestive heart failure.
• In April 2024, AskBio received FDA FTD for AB-1002, its investigational gene therapy program for the treatment of congestive heart failure.
• In January 2024, AskBio reported the initiation of the Phase II GenePHIT trial evaluating AB-1002, its investigational gene therapy for congestive heart failure.

Gene Therapy for Cardiomyopathies Market Segmentation

DelveInsight's "Gene Therapy for Cardiomyopathies - Market Insights, Epidemiology, and Market Forecast - 2034" report provides a detailed outlook of the current and future gene therapies of cardiomyopathies market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Gene Therapy for Cardiomyopathies Market Size by Countries

The gene therapies of cardiomyopathies market size is assessed separately for various countries, including the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2024, the United States held a significant share of the overall 7MM (Seven Major Markets) gene therapies of cardiomyopathies market, primarily attributed to the country's higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.

Gene Therapy for Cardiomyopathies Drugs Uptake

This section focuses on the sales uptake of potential gene therapies of cardiomyopathies that have recently been launched or are anticipated to be launched in the gene therapies of cardiomyopathies market between 2025 and 2034. It estimates the market penetration of gene therapies of cardiomyopathies for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the gene therapies of cardiomyopathies market.

The emerging gene therapies of cardiomyopathies are analyzed based on various attributes such as efficacy and safety in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the gene therapies of cardiomyopathies market.

Gene Therapy for Cardiomyopathies Market Access and Reimbursement

DelveInsight's "Gene Therapy for Cardiomyopathies - Market Insights, Epidemiology, and Market Forecast - 2034" report provides a descriptive overview of the market access and reimbursement scenario of gene therapies of cardiomyopathies. This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current gene therapies of cardiomyopathies market trends and to fill gaps in secondary findings, we interview KOLs' and SMEs' working in the gene therapies of cardiomyopathies domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or gene therapies of cardiomyopathies market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the gene therapies of cardiomyopathies unmet needs.

Gene Therapies of Cardiomyopathies: KOL Insights

DelveInsight's analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as University of Texas MD Anderson Cancer Center, US; University Medical Center Hamburg-Eppendorf, Germany; PSL Research University, France; University of Campania "Luigi Vanvitelli, Italy; Complutense University, Spain; Liverpool John Moores University, UK; and Keio University School of Medicine, Japan; among others.

As per KOLs from the US, "As gene therapies advance, we are witnessing a paradigm shift in the treatment of cardiomyopathies. Unlike conventional therapies that primarily manage symptoms, gene-based approaches directly target the underlying genetic drivers of hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy. By delivering corrective or silencing constructs, these therapies offer the potential to halt or even reverse disease progression, which could fundamentally alter patient outcomes."

As per KOLs from Germany, "Cardiomyopathies are highly heterogeneous, both genetically and clinically, which has historically made treatment difficult. Gene therapies bring a new level of precision by tailoring interventions to the specific mutations causing disease. This is particularly important for younger patients, where a one-time treatment could provide durable benefits and reduce the lifelong burden of progressive heart failure, arrhythmias, and risk of sudden cardiac death."

As per KOLs from Japan, "The most exciting aspect of gene therapy for cardiomyopathies is its potential to deliver disease-modifying benefits beyond symptomatic control. Current investigational candidates using adeno-associated virus vectors or gene silencing platforms are designed to restore heart muscle integrity and improve contractile function. If successful, these therapies could redefine the standard of care by shifting management from chronic lifelong interventions to curative, mechanism-driven solutions."

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the gene therapies of cardiomyopathies. Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Gene Therapy for Cardiomyopathies Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for gene therapies of cardiomyopathies. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging gene therapies of cardiomyopathies.

Gene Therapy for Cardiomyopathies Report Insights

• Cardiomyopathies Patient Population
• Therapeutic Approaches
• Gene Therapy for Cardiomyopathies Pipeline Analysis
• Gene Therapy for Cardiomyopathies Market Size and Trends
• Gene Therapy for Cardiomyopathies Market Opportunities
• Impact of Upcoming Therapies

Gene Therapy for Cardiomyopathies Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• Cardiomyopathies Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Gene Therapy for Cardiomyopathies Market
• Gene Therapy for Cardiomyopathies Uptake

Gene Therapy for Cardiomyopathies Report Assessment

• Cardiomyopathies Current Treatment Practices
• Unmet Needs
• Gene Therapy for Cardiomyopathies Profiles
• Gene Therapy for Cardiomyopathies Market Attractiveness

Key Questions:

• How common is cardiomyopathies?
• What are the key findings of cardiomyopathies epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020-2034)?
• What are the currently available treatments for cardiomyopathies?
• What are the disease risk, burden, and unmet needs of cardiomyopathies?
• At what CAGR is the gene therapies of cardiomyopathies market and its epidemiology is expected to grow in the 7MM during the forecast period (2025-2034)?
• How would the unmet needs impact the gene therapies of cardiomyopathies market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of cardiomyopathies in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2025-2034)?
• How many companies are currently developing gene therapies for the treatment of cardiomyopathies?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the gene therapies of cardiomyopathies market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of current treatment in the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the attribute analysis section to provide visibility around leading classes.
• Highlights of Market Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Frequently Asked Questions:

1. What are the treatment goals for cardiomyopathies?

The primary treatment goals for cardiomyopathies with gene therapy are to correct or silence the underlying genetic defects, restore normal heart muscle structure and function, and halt or reverse disease progression. Additional objectives include reducing the risk of heart failure, arrhythmias, stroke, and sudden cardiac death, while improving exercise capacity, quality of life, and long-term survival. Gene therapies aim to deliver durable, disease-modifying benefits beyond conventional care, minimizing the lifelong treatment burden and providing patients with sustained cardiac health and daily functioning.

2. What are the challenges in managing gene therapies of cardiomyopathies?

The challenges in managing cardiomyopathies with gene therapy include the diverse genetic mutations and variable disease expression across hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy, which complicate patient selection and therapeutic targeting. Early diagnosis remains difficult, as symptoms may be subtle or overlap with other cardiac conditions. Gene therapy development faces hurdles such as optimizing vector delivery, ensuring durable expression, managing immune responses, and addressing manufacturing complexities. Furthermore, variability in patient response, limited long-term outcome data, and the need for specialized monitoring create additional challenges, while accessibility and cost considerations may also affect widespread adoption.

3. What are the key factors driving the growth of the gene therapies of cardiomyopathies market?

Key factors driving the growth of the cardiomyopathies market with gene therapy include the rising recognition of genetic underpinnings in hypertrophic cardiomyopathy, dilated cardiomyopathy, and restrictive cardiomyopathy, as well as increasing awareness of the need for disease-modifying interventions. Advances in gene replacement, silencing, and editing technologies are creating new opportunities to target root causes and improve long-term outcomes. Growing adoption of precision medicine approaches, combined with expanding investment in rare and inherited cardiovascular diseases, further accelerates development. Additionally, ongoing clinical trials, regulatory designations, and strategic partnerships continue to fuel momentum by addressing previously unmet needs and reshaping the treatment paradigm.

4. How will the Gene Therapy for Cardiomyopathies Market and Epidemiology Forecast Report benefit the clients?

The Gene Therapies for Cardiomyopathies Market and Epidemiology Forecast Report provides clients with comprehensive insights into disease prevalence, genetic subtypes, and evolving treatment trends. It enables identification of unmet needs, evaluation of competitive dynamics, and strategic planning for pipeline development and market positioning. The report supports informed decision-making by analyzing regulatory designations, clinical trial progress, and emerging gene therapy platforms, helping stakeholders optimize investments, partnerships, and access strategies in this rapidly advancing field.