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세계의 희귀질환 유전체 편집 시장 보고서(2025년)
Rare Disease Genome Editing Global Market Report 2025
상품코드 : 1855967
리서치사 : The Business Research Company
발행일 : On Demand Report
페이지 정보 : 영문 250 Pages
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한글목차

희귀질환 유전체 편집 시장 규모는 최근 급속히 확대되고 있습니다. 2024년 10억 9,000만 달러로 평가되었고, 2025년에는 12억 9,000만 달러에 달하고, CAGR 17.9%로 성장할 전망입니다. 역사적인 기간에 있어서 성장의 원동력은 유전자 연구에 대한 정부 자금 증가, 희귀질환 진단에 대한 의식 증가, 생명공학 기업 간의 협력 관계의 확대, 맞춤형 의료에 대한 투자의 확대입니다.

희귀질환 유전체 편집 시장 규모는 향후 수년간 급성장할 것으로 전망됩니다. 2029년에는 CAGR 17.6%로 24억 6,000만 달러로 성장할 전망입니다. 예측 기간 동안의 성장은 유전자 편집에서 인공지능의 통합 증가, 표적 치료에 대한 수요 증가, 유전자 도입 시스템의 진보, 다유전자 질환의 유병률 증가, 소아 희귀질환에 대한 관심 증가가 기여하고 있습니다. 예측기간의 주요 동향에는 다유전자 편집기술의 개발, AI를 활용한 유전체 해석의 통합, 기술을 활용한 개별화 유전자 치료, in vivo 유전자 도입 시스템의 혁신, 하이 스루풋 유전체 스크리닝의 진보 등이 포함됩니다.

정밀의료가 중시되고 희귀질환 유전체 편집 시장의 확대가 전망되고 있습니다. 정밀의료는 개인의 유전적 체질, 환경, 라이프 스타일에 따라 예방, 진단 및 치료 전략을 조정하는 것입니다. 이 접근법에 대한 관심이 증가하는 배경은 보다 정확한 검출 및 표적 치료를 가능하게 하는 유전체 시퀀싱 및 바이오마커 식별 진보를 포함합니다. 희귀질환 유전체 편집 시장은 유전자 돌연변이의 정확한 수정을 가능하게 하고 치료를 보다 개별화되고 효과적인 것으로 만들어 정밀의료에서 중요한 역할을 하고 있습니다. 이 접근법은 희귀질환의 근본 원인을 다루고, 환자의 예후를 개선하고, 대증 요법에 대한 의존을 줄입니다. 예를 들어, 2024년 3월 호주에 기반을 둔 생명공학기업인 노보텍은 2023년 FDA가 승인한 217종양 치료제 중 43%가 정밀 종양 치료제이며, 그 중 78개가 DNA 또는 NGS로 검출 가능한 바이오마커를 포함한 것으로 보고했습니다. 이러한 정밀의료에 대한 관심 증가는 희귀질환 유전체 편집 시장의 성장에 기여하고 있습니다.

희귀질환 유전체 편집 시장의 기업은 치료의 정확성을 높이고 치료 불가능했던 유전성 질환의 예후를 개선하기 위해 치료 플랫폼, 특히 CRISPR 기반 유전자 편집 시스템을 추진하고 있습니다. CRISPR 기반 유전자 편집 시스템은 유전체의 정확한 변형을 가능하게 하는 최첨단 기술로, 유전적 결함을 수정하고 치료 유전자를 세포에 도입할 수 있습니다. 예를 들어, 2023년 12월 미국의 바이오 의약품 회사인 버텍스 퍼머슈티컬스 잉크는 CRISPR 세라퓨틱스 잉크와 제휴하여 CASSEVY(exagamglogene autotemcel, exa-cel)에 대해 영국 의약품 및 헬스케어 제품 규제청(MHRA)으로부터 조건부 판매를 승인했습니다. CASGEVY는 12세 이상의 겸상 적혈구증(SCD)에서 재발성 혈관 폐색성 클리제 또는 수혈 의존성 β-살라세미아(TDT) 환자에게 적용됩니다. 이 치료는 환자의 조혈 줄기 세포를 편집하여 태아 헤모글로빈의 생산을 재활성화하여 질병의 증상을 완화하거나 없애고 이러한 질병의 기능적 치유를 초래할 수 있습니다.

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 동향과 전략

제4장 시장 : 금리, 인플레이션, 지정학, 무역전쟁과 관세, 그리고 코로나 및 회복이 시장에 미치는 영향을 포함한 거시경제 시나리오

제5장 세계의 성장 분석과 전략 분석 프레임워크

제6장 시장 세분화

제7장 지역별/국가별 분석

제8장 아시아태평양 시장

제9장 중국 시장

제10장 인도 시장

제11장 일본 시장

제12장 호주 시장

제13장 인도네시아 시장

제14장 한국 시장

제15장 서유럽 시장

제16장 영국 시장

제17장 독일 시장

제18장 프랑스 시장

제19장 이탈리아 시장

제20장 스페인 시장

제21장 동유럽 시장

제22장 러시아 시장

제23장 북미 시장

제24장 미국 시장

제25장 캐나다 시장

제26장 남미 시장

제27장 브라질 시장

제28장 중동 시장

제29장 아프리카 시장

제30장 경쟁 구도와 기업 프로파일

제31장 기타 주요 기업 및 혁신 기업

제32장 세계 시장 경쟁 벤치마킹과 대시보드

제33장 주요 인수합병(M&A)

제34장 최근 시장 동향

제35장 시장의 잠재력이 높은 국가, 부문, 전략

제36장 부록

SHW
영문 목차

영문목차

Rare disease genome editing refers to the application of precise genetic modification technologies to correct or alter disease-causing mutations in patients with rare genetic disorders. This approach restores normal gene function, prevents disease progression, and enables the development of personalized therapeutic solutions for conditions with limited or no treatment options. It targets the root cause of the disease at the DNA level rather than managing symptoms.

The primary technologies used in rare disease genome editing include CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats with CRISPR-associated protein 9), transcription activator-like effector nucleases (TALEN), zinc finger nucleases (ZFN), and others. CRISPR-Cas9 employs a guide RNA and the Cas9 enzyme to precisely cut and modify DNA for therapeutic and research purposes. These technologies are applied to various disease types, including monogenic disorders, multifactorial disorders, and chromosomal disorders, and are used in therapeutic development and research by biotechnology and pharmaceutical companies, academic and government research institutes, and hospitals.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the pharmaceutical companies contend with tariffs on APIs, glass vials, and lab equipment inputs with few alternative sources. Generic drug makers, operating on razor-thin margins, are especially vulnerable, with some reducing production of low-profit medicines. Biotech firms face delays in clinical trials due to tariff-related shortages of specialized reagents. In response, the industry is expanding API production in India and Europe, increasing inventory stockpiles, and pushing for trade exemptions for essential medicines.

The rare disease genome editing market research report is one of a series of new reports from The Business Research Company that provides rare disease genome editing market statistics, including rare disease genome editing industry global market size, regional shares, competitors with a rare disease genome editing market share, detailed rare disease genome editing market segments, market trends and opportunities, and any further data you may need to thrive in the rare disease genome editing industry. This rare disease genome editing market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.'

The rare disease genome editing market size has grown rapidly in recent years. It will grow from $1.09 billion in 2024 to $1.29 billion in 2025 at a compound annual growth rate (CAGR) of 17.9%. The growth during the historic period was driven by increasing government funding for genetic research, rising awareness of rare disease diagnosis, growing collaboration between biotech companies, and expanding investment in personalized medicine.

The rare disease genome editing market size is expected to see rapid growth in the next few years. It will grow to $2.46 billion in 2029 at a compound annual growth rate (CAGR) of 17.6%. The growth during the forecast period is fueled by increasing integration of artificial intelligence in genome editing, rising demand for targeted therapies, advancements in gene delivery systems, growing prevalence of multi-gene disorders, and heightened focus on pediatric rare diseases. The primary trends in the forecast period include development of multi-gene editing techniques, integration of AI-driven genomic analysis, technology-enabled personalized gene therapies, innovations in in vivo gene delivery systems, and advancements in high-throughput genome screening.

The growing emphasis on precision medicine is expected to drive the expansion of the rare disease genome editing market. Precision medicine involves tailoring prevention, diagnosis, and treatment strategies based on an individual's genetic makeup, environment, and lifestyle. The increasing focus on this approach is fueled by advances in genomic sequencing and biomarker identification, which enable more accurate detection and targeted therapies. The rare disease genome editing market plays a key role in precision medicine by enabling the precise correction of genetic mutations, making treatments more personalized and effective. This approach addresses the root causes of rare disorders, improving patient outcomes and reducing reliance on symptom-based treatments. For example, in March 2024, Novotech, an Australia-based biotechnology company, reported that 43% of the 217 FDA-approved oncology therapies in 2023 were precision oncology treatments, with 78 of these incorporating DNA or NGS-detectable biomarkers. This growing focus on precision medicine is contributing to the growth of the rare disease genome editing market.

Companies in the rare disease genome editing market are advancing therapeutic platforms, particularly CRISPR-based gene editing systems, to enhance treatment precision and improve outcomes for previously untreatable genetic diseases. CRISPR-based gene editing systems are cutting-edge technologies that allow for precise alterations to the genome, enabling the correction of genetic defects or the introduction of therapeutic genes into cells. For example, in December 2023, Vertex Pharmaceuticals Inc., a US-based biopharmaceutical company, partnered with CRISPR Therapeutics Inc. and received conditional marketing authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for CASGEVY (exagamglogene autotemcel, exa-cel). CASGEVY is indicated for patients aged 12 and older with sickle cell disease (SCD) and recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia (TDT). The treatment works by editing a patient's hematopoietic stem cells to reactivate fetal hemoglobin production, potentially reducing or eliminating disease symptoms and offering a functional cure for these conditions.

In November 2022, Alexion Pharmaceuticals Inc., a subsidiary of AstraZeneca, acquired LogicBio Therapeutics Inc. for an undisclosed amount. This acquisition allows Alexion to expand its presence in the field of rare disease genomics and leverage LogicBio's GeneRide platform to develop innovative therapies. LogicBio Therapeutics is a clinical-stage company specializing in genome-editing therapies for rare genetic disorders.

Major players in the rare disease genome editing market are Regeneron Pharmaceuticals Inc., Sarepta Therapeutics Inc., Ultragenyx Pharmaceutical Inc., Beam Therapeutics Inc., CRISPR Therapeutics AG, Rocket Pharmaceuticals Inc., Sangamo Therapeutics Inc., Arcturus Therapeutics Holdings Inc., Editas Medicine Inc., Krystal Biotech Inc., Caribou Biosciences Inc, Bluebird Bio Inc., SpliceBio S.L., Cellectis SA, Generation Bio Co., iECURE Inc., Affinia Therapeutics Inc., Precision Biosciences Inc., Intellia Therapeutics Inc., and MeiraGTx Holdings plc.

North America was the largest region in the rare disease genome editing market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in rare disease genome editing report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the rare disease genome editing market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The rare disease genome editing market consists of revenues earned by entities by providing services such as gene editing therapy development, genetic testing and diagnostics, preclinical research and validation, clinical trial management, and regulatory consulting for genome-based therapeutics. The market value includes the value of related goods sold by the service provider or included within the service offering. The rare disease genome editing market also includes sales of zinc finger nucleases, delivery vectors, modified cells, and therapeutic genome-editing formulations. Values in this market are 'factory gate' values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values and are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Rare Disease Genome Editing Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses on rare disease genome editing market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

Where is the largest and fastest growing market for rare disease genome editing ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward, including technological disruption, regulatory shifts, and changing consumer preferences? The rare disease genome editing market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

The forecasts are made after considering the major factors currently impacting the market. These include the technological advancements such as AI and automation, Russia-Ukraine war, trade tariffs (government-imposed import/export duties), elevated inflation and interest rates.

Scope

Table of Contents

1. Executive Summary

2. Rare Disease Genome Editing Market Characteristics

3. Rare Disease Genome Editing Market Trends And Strategies

4. Rare Disease Genome Editing Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, And Covid And Recovery On The Market

5. Global Rare Disease Genome Editing Growth Analysis And Strategic Analysis Framework

6. Rare Disease Genome Editing Market Segmentation

7. Rare Disease Genome Editing Market Regional And Country Analysis

8. Asia-Pacific Rare Disease Genome Editing Market

9. China Rare Disease Genome Editing Market

10. India Rare Disease Genome Editing Market

11. Japan Rare Disease Genome Editing Market

12. Australia Rare Disease Genome Editing Market

13. Indonesia Rare Disease Genome Editing Market

14. South Korea Rare Disease Genome Editing Market

15. Western Europe Rare Disease Genome Editing Market

16. UK Rare Disease Genome Editing Market

17. Germany Rare Disease Genome Editing Market

18. France Rare Disease Genome Editing Market

19. Italy Rare Disease Genome Editing Market

20. Spain Rare Disease Genome Editing Market

21. Eastern Europe Rare Disease Genome Editing Market

22. Russia Rare Disease Genome Editing Market

23. North America Rare Disease Genome Editing Market

24. USA Rare Disease Genome Editing Market

25. Canada Rare Disease Genome Editing Market

26. South America Rare Disease Genome Editing Market

27. Brazil Rare Disease Genome Editing Market

28. Middle East Rare Disease Genome Editing Market

29. Africa Rare Disease Genome Editing Market

30. Rare Disease Genome Editing Market Competitive Landscape And Company Profiles

31. Rare Disease Genome Editing Market Other Major And Innovative Companies

32. Global Rare Disease Genome Editing Market Competitive Benchmarking And Dashboard

33. Key Mergers And Acquisitions In The Rare Disease Genome Editing Market

34. Recent Developments In The Rare Disease Genome Editing Market

35. Rare Disease Genome Editing Market High Potential Countries, Segments and Strategies

36. Appendix

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