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Table Of Content
January 2025
The Genetic Editing for ASCVD Market is expanding with Verve’s PCSK9 base-editing therapy and Lilly’s upcoming Phase 2 trials for cardiovascular gene cures. The market sizing and forecasts are revenue-based (USD Million/Billion), with 2024 as the base year.
Market insights on genetic editing for ASCVD, featuring CRISPR based therapies targeting PCSK9 & ANGPTL3 to transform cardiovascular disease treatment. The genetic editing for ASCVD market is propelled by advanced research infrastructure, strong regulatory support, and growing demand for personalized cardiovascular therapies.
The integration of Artificial Intelligence is transforming the genetic editing for ASCVD by enhancing the efficiency, speed, and safety of gene therapies to a great extent. AI algorithms analyze vast genomic data to pinpoint optimal gene targets like PCSK9 or ANGPTL3, predicting off-target effects and minimizing unintended edits. This enhances the design and delivery of CRISPR treatments. AI also accelerates drug discovery, automates workflow optimization, and enables real-time editing and results monitoring.
The genetic editing for atherosclerotic cardiovascular disease (ASCVD) market refers to the application of next-generation gene editing technologies, such as CRISPR, base editing, and siRNA/ASO gene silencing, to prevent, treat, or reverse cardiovascular disorders caused by genetic dyslipidemia and inflammation, primarily familial hypercholesterolemia (FH), elevated Lp(a), PCSK9/LDLR mutations, and chronic vascular inflammation. The goal is to offer one-time or long-term disease-modifying treatments rather than lifelong lipid-lowering or anti-inflammatory drugs.
The genetic editing for ASCVD market is experiencing substantial growth, driven by advancements in CRISPR and gene therapy technologies. The rising prevalence of ASCVD, coupled with a growing demand for personalized medicine, is accelerating gene-targeted research. Increased funding for genomic research from government and private organizations, along with collaborations between biotech companies and healthcare networks, are further enhancing market growth. Moreover, the identification of genetic mutations, such as PCSK9 and ANGPTL3, linked to ASCVD, has opened up new avenues for therapeutic intervention.
| Report Coverage | Details |
| Dominating Region | North America |
| Fastest Growing Region | Asia Pacific |
| Base Year | 2024 |
| Forecast Period | 2025 to 2034 |
| Segments Covered | Editing Modality, Therapeutic Target, Delivery System, Stage of Development, End User, and Region |
| Regions Covered | North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa |
Focus on Personalized Medicine
The growing emphasis on personalized medicine is one of the major factrs driving the growth of the genetic editing for ASCVD market. With technologies such as CRISPR-Cas9, clinicians can directly edit the genes involved in lipid metabolism and cardiovascular risk, including PCSK9 and ANGPTL3. This individualized method enhances the success of therapy because it targets the underlying cause of the disease, rather than just the symptoms. Additionally, it will reduce the likelihood of negative reactions and improve the effectiveness of treatment. Genomic sequencing and bioinformatics can assist healthcare providers in identifying patients whose health conditions are most likely to be improved with the help of gene editing, reduce resource waste, and ultimately benefit patients.
Ethical and Regulatory Challenges
One of the major inhibitors of the genetic editing market for atherosclerotic cardiovascular disease is the complex ethical and regulatory framework. Methods, such as CRISPR-Cas9, which hold promise for revolutionizing the field of curing, raise significant ethical concerns related to safety, germ-line modification, and their potential unforeseen outcomes. Off-target effects, which result in the alteration of genes other than the intended target gene, are a significant risk to patient safety that regulators can not overlook. As a result, strict regulatory clearances and increased time to clinical trials hamper the commercialization process and their mass adoption.
Innovation of New Treatments
The continual evolution of new gene editing technologies and delivery systems presents a significant opportunity to enhance genetic therapy in the treatment of ASCVD. Most of the newer techniques, such as base editing, prime editing, and epigenome editing, find applications where ordinary CRISPR-Cas9 methods are not feasible. The second-generation tools are more precise, less likely to create off-target effects, and they may enable gene repair without introducing double-stranded breaks in the genome. This change might be safer and more effective. With the progress in research, it is now possible to correct multiple genetic risk factors simultaneously or modulate gene expression in adults using these new techniques.
Why Did the CRISPR/Cas9 Gene Knockout Segment Contribute the Most Revenue in 2024?
The CRISPR/Cas9 gene knockout segment held a 44% share of the genetic editing for ASCVD market in 2024 due to its proven efficacy. CRISPR/Cas9, a powerful gene-editing tool, induces specific DNA breaks, leading to insertions or deletions that disrupt disease-causing genes. This method is used in ASCVD to silence genes like PCSK9 and ANGPTL3, which are involved in lipid metabolism and high cholesterol, key factors in plaque formation. CRISPR/Cas9 gene knockout offers advantages such as a more established research foundation, lower development costs, and easier scalability compared to other methods.
The base editing segment is likely to grow at the fastest rate in the coming years due to its high precision and safety rates, making it a potentially valuable genetic editing and modification tool in managing ASCVD. This specificity reduces off-target effects and genomic instability, and base editing is especially appealing for correcting single-nucleotide polymorphisms (SNPs) associated with cardiovascular risks. Advances such as adenine base editors (ABE) and cytosine base editors (CBE) have been actively investigated as a method of curing pathogenic mutations in several genes involved in lipid metabolism. Due to the constantly increasing efficiency and safety data, base editing promises to transform the field of future ASCVD treatment, presenting extremely personalized and long-lasting treatment options.
How Does the PCSK9 Segment Lead the Genetic Editing for ASCVD Market in 2024?
The PCSK9 segment led the market while holding a 38% share in 2024 due to the clinical validation of PCSK9 as a demonstrable cholesterol-lowering target, specifically low-density lipoprotein cholesterol (LDL-C), a major risk factor for atherosclerotic cardiovascular disease. This leads to increased recycling of the LDL receptor and enhanced clearance of LDL-C from the blood. The long-term advantage of this strategy is that it is likely to reduce LDL-C levels throughout a person's life with a single intervention, thereby preventing the need for lifelong medication such as statins or injectable PCSK9 inhibitors. It has been found that PCSK9 is being focused on by both top biotech companies and research groups, as its biology is well understood and has high therapeutic potential.
The ANGPTL3 segment is expected to grow at a significant CAGR over the forecast period. ANGPTL3 plays a key role in regulating lipid metabolism. It works by blocking lipoprotein lipase and endothelial lipase, which are involved in breaking down triglycerides and LDL-C. This makes ANGPTL3 significant in managing high levels of lipids. Its therapeutic value is especially notable in rare lipid disorders like familial combined hyperlipidemia. Several biotech firms are working on gene editing therapies for ANGPTL3, aiming for a single, long-lasting treatment. Given its promising early results and increasing investment, ANGPTL3 is expected to experience significant growth, becoming a key component in future treatments for ASCVD.
What Made AAV Vectors the Dominant Segment in the Genetic Editing for ASCVD Market in 2024?
The AAV vectors segment dominated the market, accounting for the largest revenue share in 2024, as AAV vectors are a favored method for delivering in vivo gene editing therapies, driven by their efficient gene transfer, lasting gene expression, and low immune response. In treating atherosclerotic cardiovascular disease (ASCVD), AAV vectors show promise for targeting the liver, which is crucial for lipid metabolism. By delivering CRISPR/Cas9 components directly to the liver, AAV's ability to target specific tissues and provide stable gene expression with a single dose makes it an ideal tool for correcting the disease.
The lipid nanoparticles segment is expected to grow at a significant CAGR over the forecast period because they are non-viral, repeatable, and extremely customizable. LNPs offer several advantages over viral vectors due to their higher safety and lower likelihood of causing immune responses, which is not always the case with viral systems. These benefits are especially important in cardiovascular treatments, where long-term safety, patient adherence, and precise targeting are critical. With increasing research and promising early results, LNP-based delivery is rapidly emerging as a leading approach for future genetic treatments for ASCVD.
Why did the Preclinical Research Segment Contribute the Most Revenue in 2024?
The preclinical research contributed the most revenue in 2024 and is expected to sustain its position throughout the projection period. Numerous pharmaceutical companies and research groups are actively working on gene-editing approaches for the PCSK9, ANGPTL3, and Lp(a) genes, which are strongly linked to elevated cholesterol levels and cardiovascular disease risk. To assess their effectiveness, delivery capabilities, and safety, these organizations are conducting in vitro and animal studies to evaluate various editing methods like gene knockouts, base editing, and prime editing.
Significant non-dilutive investments, including grants, government incentives, and early licensing opportunities, are supporting preclinical efforts. Promising results from animal models, indicating sustained control of LDL-C and triglycerides with a single treatment, are further driving interest. Because gene editing aims to provide lasting, single-dose cures for ASCVD, investments in this preclinical phase are expected to continue.
The phase I/II clinical trials segment is expected to grow at a significant rate in the upcoming period, fueled by the development of promising therapies into human trials. Specifically, several companies are advancing in vivo gene-editing programs targeting PCSK9, with some having already initiated patient recruitment and released preliminary safety and efficacy data. These studies evaluate the use of CRISPR/Cas9 therapeutics in humans for the first time, potentially leading to long-term reductions in LDL-C following treatment. The benefits for participating companies include increased investor confidence, enhanced public visibility, and the opportunity to establish leadership in a dynamic, competitive market.
Why Did the Pharmaceutical & Biotechnology Companies Segment Dominate the Genetic Editing for ASCVD Market in 2024?
The pharmaceutical & biotechnology companies segment dominated the genetic editing for ASCVD market, holding the largest revenue share in 2024. These companies heavily utilize reagents and tools, such as CRISPR/Cas9, base editing, and prime editing, to develop groundbreaking therapies that address the genetic origins of atherosclerotic cardiovascular disease. These companies possess significant R&D resources, advanced infrastructure, and collaborative ventures, positioning them as leaders in preclinical research, clinical trials, and commercialization strategies. Furthermore, venture capitalists, government agencies, and strategic partners are prepared to make substantial investments in these companies, generating revenue through licensing, milestone payments, and technology platforms. Many of the leading gene-editing companies have partnered with major pharmaceutical companies to scale delivery systems, optimize clinical performance, and prepare for commercialization.
The specialty cardiology clinics segment is expected to experience rapid growth during the forecast period, fueled by the increasing clinical application of novel genetic treatments. As CRISPR-based interventions transition from research to patient treatment, these clinics will play a crucial role in delivering gene-editing therapies, particularly for conditions with complex cardiovascular risk factors, such as elevated LDL-C and triglycerides. These centers are also better equipped to address the ethical, technical, and logistical challenges associated with one-time gene-editing therapies, including patient eligibility, genetic counseling, and informed consent. Additionally, as patients and physicians become more aware of the potential of personalized medicine, the adoption of genomic-based ASCVD treatments may be more prevalent in specialty clinics compared to general hospitals or primary-care settings.
What Made North America the Dominant Region in the Genetic Editing for ASCVD Market in 2024?
North America led the market, holding a 52% share in 2024. This is mainly due to its well-established genetic therapy ecosystem and robust biomedical innovation infrastructure. Strong financial support from venture capitalists, government organizations like the NIH, and the private sector fuels preclinical and clinical development, ensuring the development of innovative therapies. Coupled with increased awareness of personalized medicine and greater access to genetic testing, North America is poised to remain a key driver of growth in the global market. The U.S. is a major contributor to the market. An aging population, rising rates of obesity and hyperlipidemia, and growing healthcare costs are driving investments in gene editing by healthcare stakeholders. Furthermore, the U.S. regulatory framework, led by the FDA, has shown increasing support for genetic therapies by providing expedited classifications and guidance on genome-editing procedures. The country also hosts the most active Phase I/II trials focused on cardiovascular gene editing, solidifying its leading role in clinical advancements.
Why Is the Asia Pacific Region Expected to Grow at the Fastest CAGR?
Asia Pacific is expected to grow at the fastest CAGR during the forecast period. This is primarily due to increased investment in biotechnology, growing awareness of genetic diseases, and advancements in healthcare infrastructure. Nations such as Japan, India, South Korea, and Singapore are actively establishing regulatory frameworks for gene editing and genomic medicine. Japan, in particular, has introduced accelerated approval pathways for regenerative and genetic treatments, fostering innovation in precision cardiovascular therapies. This progress is further supported by the increasing affordability of genetic testing and the expansion of partnerships between stakeholders.
China is a major player in the market. China's biotech sector is heavily engaged in developing CRISPR-based treatments for cardiovascular and metabolic disorders, with several projects already in early-stage clinical trials. The country benefits from a rapidly expanding genome data pool and a strong talent base in molecular biology and biomedical engineering. Despite ongoing discussions regarding ethical and regulatory challenges, China is working to improve its regulatory system to align with international standards, thereby improving clinical governance.
What Factors Are Influencing Europe’s Genetic Editing for ASCVD Market?
The European genetic editing for ASCVD market is expected to grow at a notable rate, driven by sustained biomedical innovation, robust public research and development funding, and a well-established healthcare infrastructure. The presence of major pharmaceutical and biotech firms, alongside leading academic institutions, fuels continuous research into CRISPR-based treatments targeting ASCVD-related genes, such as PCSK9, ANGPTL3, and Lp(a). Furthermore, Europe faces a substantial ASCVD burden, largely due to an aging population and lifestyle factors, creating a need for innovative, long-term treatment solutions such as gene editing.
The UK plays a central role in the development of the European market for genetic editing for ASCVD. With a strong presence in life sciences and genomic medicine, the UK boasts an ecosystem rich in institutions, including the Francis Crick Institute, the Wellcome Sanger Institute, and the UK Biobank, which contribute extensive genomic data to cardiovascular disease research. UK-based biotech companies and universities are actively developing CRISPR-based solutions for lipid metabolism and inflammatory pathways in ASCVD. Additionally, the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK supports the adoption of innovative therapies in clinical practice through streamlined mechanisms.
By Editing Modality
By Therapeutic Target
By Delivery System
By Stage of Development
By End User
By Region
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