Genetic Therapies Market Size, Share, Trends & Competitive Analysis By Type (Gene Replacement Therapy, Gene Editing Therapy, Oligonucleotide-Based Therapy, Others) By Application: By End-User: By Delivery Method: By Technology: By Regions, and Industry Forecast, Global Report 2023-2030

  • Report ID: FDS562
  • Forecast Period: 2023-2030
  • No. of Pages: 150+
  • Industry: Biotechnology

The global Genetic Therapies market size was valued at USD 14.2 billion in 2023 and is projected to expand at a compound annual growth rate (CAGR) of 27.9% during the forecast period, reaching a value of USD 111.8 billion by 2030.

Genetic Therapies market research report by Future Data Stats, offers a comprehensive view of the market's historical data from 2018 to 2021, capturing trends, growth patterns, and key drivers. It establishes 2022 as the base year, analyzing the market landscape, consumer behavior, competition, and regulations. Additionally, the report presents a well-researched forecast period from 2023 to 2030, leveraging data analysis techniques to project the market's growth trajectory, emerging opportunities, and anticipated challenges.


Genetic Therapies, often referred to as gene therapy, is a cutting-edge medical approach aimed at treating or preventing genetic disorders by modifying a person's genes. It involves introducing genetic material into a patient's cells to correct or replace faulty genes responsible for various inherited conditions. This innovative field of medicine holds the promise of potentially curing genetic diseases at their root cause, offering hope to individuals affected by conditions that were previously considered incurable. While genetic therapies have made significant strides in recent years, ongoing research and clinical trials continue to refine and expand our understanding of their efficacy and safety in addressing a wide range of genetic disorders.


The Genetic Therapies market is influenced by a dynamic interplay of drivers, restraints, and opportunities that shape its growth trajectory. One of the key drivers propelling this market is the growing prevalence of genetic disorders worldwide. As awareness and understanding of genetic diseases increase, there is a heightened demand for innovative treatments like gene therapy. Additionally, advancements in gene-editing technologies such as CRISPR-Cas9 have accelerated the development of novel therapeutic approaches, further driving the market forward. Moreover, regulatory bodies are increasingly streamlining approval processes for genetic therapies, fostering a favorable environment for market growth.

However, the Genetic Therapies market is not without its challenges. High development costs and pricing pressures pose significant restraints. The complexity of genetic therapies, along with concerns about long-term safety and efficacy, can slow down market expansion. Additionally, limited infrastructure and expertise in certain regions may hinder market accessibility. Nonetheless, there are substantial opportunities on the horizon. As research continues, genetic therapies are likely to diversify into new applications, potentially expanding beyond rare diseases to address more common conditions.



Gene Replacement Therapy, a prominent segment, holds significant sway in the market due to its potential to replace defective genes with healthy ones. This approach is particularly promising in treating rare genetic diseases where a single gene mutation is the root cause. Gene Editing Therapy, another major player, has gained prominence with the rise of CRISPR-Cas9 technology. Its precision in modifying specific genes has opened up new avenues for treating a wide range of genetic disorders. These two therapies, among others, are driving innovation and growth in the Genetic Therapies market.

Oligonucleotide-Based Therapy, while not as widely recognized as gene replacement or editing, is a crucial component of the Genetic Therapies market. It involves the use of short DNA or RNA sequences to modulate gene expression, offering a unique approach to treat various genetic conditions. This therapy type is gaining traction in applications such as antisense oligonucleotide therapy, which targets specific genes associated with diseases like spinal muscular atrophy. Additionally, there are other emerging therapies in the "Others" category, which encompass a range of innovative genetic treatment approaches.


Rare Genetic Diseases represent a significant driving force, as these therapies offer a ray of hope to individuals suffering from often devastating and previously untreatable inherited disorders. The potential to correct the genetic anomalies underlying these conditions has garnered substantial attention, both from the medical community and regulatory bodies, contributing to the expansion of this segment within the Genetic Therapies market.

Cancer, another major application area, has witnessed a surge in genetic therapy research and development. Targeted gene therapies hold promise in precision medicine approaches to combat various forms of cancer. These therapies can modify or suppress genes that promote tumor growth, offering personalized treatment options. Cardiovascular Diseases and Neurological Disorders also play pivotal roles in shaping the Genetic Therapies market. The genetic basis of many heart and brain-related conditions has spurred innovative therapeutic approaches, further solidifying these applications' significance.


Hospitals, as pivotal healthcare institutions, play a substantial role in driving market growth. They serve as crucial centers for patient diagnosis, treatment, and follow-up care, making them essential hubs for genetic therapy adoption. Hospitals provide the necessary infrastructure and expertise to administer genetic therapies, particularly for patients with rare genetic diseases and other complex conditions.

Specialty Clinics represent another key end-user in the Genetic Therapies market. These clinics are often specialized in specific genetic disorders or therapeutic areas, offering focused care and expertise. Patients seeking genetic therapies may choose specialty clinics for their specialized knowledge and tailored treatment approaches. Research Institutes, on the other hand, contribute significantly to the market's growth by advancing the understanding of genetic therapies through cutting-edge research. They provide the foundation for the development of new therapies, ensuring continuous innovation in the field.


In Vivo delivery method is a primary driver in this market, where therapeutic genetic material is introduced directly into the patient's body. This method offers the advantage of targeting specific cells or tissues, making it suitable for a wide range of genetic disorders. In Vivo delivery is particularly effective in treating diseases where the affected genes are present within the body, enabling precise and localized intervention.

Ex Vivo delivery method, another crucial factor, involves the removal of cells or tissues from the patient's body, genetic modification outside the body, and then reinfusion of the modified cells. This approach is commonly used in gene therapy for disorders like certain types of cancer and inherited blood disorders. Ex Vivo techniques enable thorough genetic correction or manipulation before reintroducing the treated cells into the patient, ensuring greater control over the therapeutic process.


CRISPR-Cas9, a groundbreaking gene-editing technology, is a major driver in this market. Its precision in modifying specific genes has revolutionized genetic therapies, making it a preferred choice for researchers and clinicians. CRISPR-Cas9's versatility in correcting or replacing faulty genes holds immense potential for treating a wide range of genetic disorders, driving innovation and market growth.

Antisense Oligonucleotides (ASOs) also play a pivotal role in shaping the Genetic Therapies market. ASOs are short DNA or RNA sequences that can modulate gene expression by binding to specific RNA molecules. They are particularly effective in treating conditions where gene expression needs to be regulated or modified. ASOs offer a targeted approach to genetic therapies and are used in applications such as spinal muscular atrophy and certain types of muscular dystrophy.

Viral Vectors, another critical technology, are widely employed in delivering therapeutic genes to the patient's cells. These vectors, derived from viruses, serve as vehicles to transport genetic material into the body. Their ability to efficiently introduce genetic material into target cells has made them indispensable in many gene therapy approaches. Beyond these technologies, the "Others" category encompasses a variety of cutting-edge techniques and tools that continue to expand the horizons of genetic therapies.


North America is a prominent player in this market, characterized by robust research and development efforts and a favorable regulatory environment. The region's advanced healthcare infrastructure and substantial investment in genetic therapy research contribute to its dominance. Europe follows closely, with a strong presence of biotechnology companies and research institutions driving innovation. The European market benefits from collaborative efforts across countries, fostering a conducive environment for genetic therapy advancements.

Asia Pacific presents substantial growth potential in the Genetic Therapies market due to its burgeoning population and increasing awareness of genetic disorders. Countries like China and Japan have witnessed significant research activities and investments in gene therapy. Latin America is also emerging as a promising market, with improving healthcare access and a growing patient pool seeking genetic therapies. Lastly, the Middle East and Africa region are progressively exploring genetic therapies, with a focus on addressing genetic disorders prevalent in the population.


The Genetic Therapies market, like many sectors within healthcare, experienced both challenges and opportunities in the wake of the COVID-19 pandemic. While the pandemic disrupted clinical trials and delayed some research initiatives, it also underscored the importance of innovative therapies like genetic treatments. The crisis emphasized the need for effective solutions to combat genetic disorders and other diseases, driving increased interest and investments in genetic therapies. Additionally, the pandemic accelerated the adoption of telehealth services, facilitating patient access to genetic therapies and clinical consultations.


Mergers & Acquisitions

  • Vertex Pharmaceuticals acquires Exonics Therapeutics (US)
  • Pfizer acquires Biohaven Pharmaceutical Holding Company (US)
  • Roche acquires Spark Therapeutics (US)
  • Novartis acquires AveXis (US)
  • Bayer acquires Asklepios BioPharma (Germany)

Product New Launches

  • Vertex Pharmaceuticals launches Trikafta (gene therapy for cystic fibrosis)
  • Pfizer launches Vyondys 53 (gene therapy for Duchenne muscular dystrophy)
  • Roche launches Luxturna (gene therapy for Leber congenital amaurosis)
  • Novartis launches Zolgensma (gene therapy for spinal muscular atrophy)
  • Bayer launches Luxturna (gene therapy for Leber congenital amaurosis)


  • Novartis AG
  • Roche Holding AG
  • Pfizer Inc.
  • Biogen Inc.
  • Spark Therapeutics (a Roche company)
  • Regeneron Pharmaceuticals, Inc.
  • bluebird bio, Inc.
  • CRISPR Therapeutics AG
  • Editas Medicine, Inc.
  • Vertex Pharmaceuticals Incorporated
  • Sangamo Therapeutics, Inc.
  • Amgen Inc.
  • Intellia Therapeutics, Inc.
  • Orchard Therapeutics plc
  • Abeona Therapeutics Inc.
  • uniQure N.V.
  • Sarepta Therapeutics, Inc.
  • Audentes Therapeutics (a Astellas company)
  • Ultragenyx Pharmaceutical Inc.
  • Astellas Pharma Inc.
  • Homology Medicines, Inc.
  • MeiraGTx Holdings plc
  • LogicBio Therapeutics, Inc.
  • Beam Therapeutics Inc.
  • Precision BioSciences, Inc.
  • others

Table of Contents

  1. Introduction

    • Market Definition
    • Research Methodology
    • Executive Summary
  2. Market Overview

    • Market Trends
    • Market Drivers
    • Market Challenges
    • Market Opportunities
  3. Segmentation by Type

    • Gene Replacement Therapy
    • Gene Editing Therapy
    • Oligonucleotide-Based Therapy
    • Others
  4. Segmentation by Application

    • Rare Genetic Diseases
    • Cancer
    • Cardiovascular Diseases
    • Neurological Disorders
    • Infectious Diseases
    • Others
  5. Segmentation by End-User

    • Hospitals
    • Specialty Clinics
    • Research Institutes
    • Biotechnology Companies
    • Others
  6. Regional Analysis

    • North America
    • Europe
    • Asia-Pacific
    • Latin America
    • Middle East & Africa
  7. Development Stage Analysis

    • Clinical Stage
    • Preclinical Stage
    • Research Stage
  8. Delivery Method Analysis

    • In Vivo
    • Ex Vivo
  9. Technology Analysis

    • CRISPR-Cas9
    • Antisense Oligonucleotides
    • Viral Vectors
    • Others
  10. Regulatory Approval Analysis

    • Approved Therapies
    • Investigational Therapies
  11. Competitive Landscape

    • Key Players
    • Company Profiles
    • Market Share Analysis
  12. Future Outlook

  13. Conclusion

Genetic Therapies Market Segmentation

By Type:

  • Gene Replacement Therapy
  • Gene Editing Therapy
  • Oligonucleotide-Based Therapy
  • Others

By Application:

  • Rare Genetic Diseases
  • Cancer
  • Cardiovascular Diseases
  • Neurological Disorders
  • Infectious Diseases
  • Others

By End-User:

  • Hospitals
  • Specialty Clinics
  • Research Institutes
  • Biotechnology Companies
  • Others

By Delivery Method:

  • In Vivo
  • Ex Vivo

By Technology:

  • CRISPR-Cas9
  • Antisense Oligonucleotides
  • Viral Vectors
  • Others


By Geography:

  • North America (USA, Canada, Mexico)
  • Europe (Germany, UK, France, Russia, Italy, Rest of Europe)
  • Asia-Pacific (China, Japan, South Korea, India, Southeast Asia, Rest of Asia-Pacific)
  • South America (Brazil, Argentina, Columbia, Rest of South America)
  • Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, South Africa, Rest of MEA)

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Primary Research


Desk Research


Company Analysis


•       Identify key opinion leaders

•       Questionnaire design

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•       Company Website

•       Company Annual Reports

•       Paid Databases

•       Financial Reports


•       Market Participants

•       Key Strengths

•       Product Portfolio

•       Mapping as per Value Chain

•       Key focus segment


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Secondary Research:

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•       Top executives of end-use industries

•       C-level executives of the leading Parenteral Nutrition companies

•       Sales manager and regional sales manager of the Parenteral Nutrition companies

•       Industry Consultants

•       Distributors/Suppliers


•       Annual Reports

•       Presentations

•       Company Websites

•       Press Releases

•       News Articles

•       Government Agencies’ Publications

•       Industry Publications

•       Paid Databases


Analyst Tools and Models:



·         Arriving at
Global Market Size

·         Arriving at
Market Size

·         Market Share
of Key Players

·         Key Market Players

·         Key Market Players

·         Market Share
of Key Players

·         Arriving at
Market Size

·         Arriving at
Global Market Size


Genetic Therapies Market Dynamic Factors


  • Growing prevalence of genetic disorders.
  • Advancements in gene-editing technologies like CRISPR-Cas9.
  • Streamlined regulatory approval processes.
  • Increasing investment in genetic therapy research.
  • Rising awareness and acceptance of genetic therapies.


  • High development and treatment costs.
  • Concerns regarding long-term safety and efficacy.
  • Limited accessibility and expertise in certain regions.
  • Ethical and regulatory complexities.
  • Challenges related to scalability and manufacturing.


  • Diversification into new applications.
  • Collaborations between pharmaceutical companies and research institutions.
  • Expansion into more common diseases.
  • Tailored and personalized treatment options.
  • Growing global demand for innovative genetic therapies.


  • Ethical and regulatory considerations.
  • Ensuring affordability and accessibility.
  • Addressing manufacturing and scalability challenges.
  • Long-term monitoring of patients.
  • Competition and market saturation in some areas.

Frequently Asked Questions

The global Genetic Therapies market size was valued at USD 14.2 billion in 2023 and is projected to expand at a compound annual growth rate (CAGR) of 27.9% during the forecast period, reaching a value of USD 111.8 billion by 2030.

Key factors driving the growth of the Genetic Therapies market include the increasing prevalence of genetic disorders, advancements in gene-editing technologies like CRISPR-Cas9, streamlined regulatory approvals, and growing awareness and acceptance of genetic therapies.

Current trends and advancements in the Genetic Therapies market include personalized and targeted treatments, the exploration of new therapy types, such as oligonucleotide-based therapies, and the emergence of innovative gene-editing techniques.

North America and Europe are expected to dominate the Genetic Therapies market, driven by their advanced healthcare infrastructure, substantial research investments, and favorable regulatory environments.

Major challenges in the Genetic Therapies market include high development costs, concerns about long-term safety and efficacy, limited accessibility in certain regions, and ethical and regulatory complexities. Opportunities lie in diversification into new applications, collaborations between pharmaceutical companies and research institutions, and expanding into more common diseases.
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