시장보고서
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1855967

세계의 희귀질환 유전체 편집 시장 보고서(2025년)

Rare Disease Genome Editing Global Market Report 2025

발행일: | 리서치사: The Business Research Company | 페이지 정보: 영문 250 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




■ 보고서에 따라 최신 정보로 업데이트하여 보내드립니다. 배송일정은 문의해 주시기 바랍니다.

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.

자주 묻는 질문

  • 희귀질환 유전체 편집 시장 규모는 어떻게 변화하고 있나요?
  • 희귀질환 유전체 편집 시장의 성장 원동력은 무엇인가요?
  • 정밀의료가 희귀질환 유전체 편집 시장에 미치는 영향은 무엇인가요?
  • CRISPR 기반 유전자 편집 시스템의 역할은 무엇인가요?
  • 희귀질환 유전체 편집 시장의 주요 기업은 어디인가요?

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

  • 세계의 희귀질환 유전체 편집 : PESTEL 분석(정치, 사회, 기술, 환경, 법적 요인, 촉진요인과 억제요인)
  • 최종 이용 산업의 분석
  • 세계의 희귀질환 유전체 편집 시장 : 성장률 분석
  • 세계의 희귀질환 유전체 편집 시장 실적 : 규모와 성장, 2019년-2024년
  • 세계의 희귀질환 유전체 편집 시장 예측 : 규모와 성장, 2024년-2029년, 2034년
  • 세계의 희귀질환 유전체 편집 : 총잠재시장 규모(TAM)

제6장 시장 세분화

  • 세계의 희귀질환 유전체 편집 시장 : 기술별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • CRISPR 또는 CRISPR 관련 단백질 9
  • 전사 액티베이터 유사 이펙터 뉴클레아제(TALEN)
  • 징크 핑거 뉴클레아제(ZFN)
  • 기타 기술
  • 세계의 희귀질환 유전체 편집 시장 : 질환 유형별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 단일유전자 질환
  • 다인자 질환
  • 염색체 질환
  • 세계의 희귀질환 유전체 편집 시장 : 용도별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 치료
  • 연구
  • 세계의 희귀질환 유전체 편집 시장 : 최종 사용자별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 생명공학과 제약회사
  • 학술·정부연구기관
  • 병원
  • 세계의 희귀질환 유전체 편집 시장 : 세분화 CRISPR 또는 CRISPR 관련 단백질 9, 유형별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 클러스터형 규칙적 간격의 짧은 회문 반복서열(CRISPR) 관련 단백질 9
  • 클러스터형 규칙적 간격의 짧은 회문 반복서열(CRISPR) 관련 단백질 12
  • 클러스터형 규칙적 간격의 짧은 회문 반복서열(CRISPR) 관련 단백질 13
  • 기본 편집
  • 프라임 편집
  • 세계의 희귀질환 유전체 편집 시장 : 세분화 전사 액티베이터님 이펙터 뉴클레아제(TALEN), 유형별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 표준 전사 액티베이터 유사 이펙터 뉴클레아제(TALEN)
  • 디자이너 전사 액티베이터 유사 이펙터 뉴클레아제(dTALEN)
  • 전사 액티베이터-유사 이펙터 뉴클레아제(TALEN) 페아뉴클레아제
  • 세계의 희귀질환 유전체 편집 시장 : 세분화 징크 핑거 뉴클레아제(ZFN), 유형별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 표준 징크 핑거 뉴클레아제(ZFN)
  • 인공 징크 핑거 뉴클레아제(ZFN)
  • 모듈형 징크 핑거 뉴클레아제(ZFN)
  • 세계의 희귀질환 유전체 편집 시장 : 세분화 기타 기술, 유형별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 메가뉴클레아제
  • 리보 핵산(RNA) 유도형 뉴클레아제
  • 클러스터형 규칙적 간격의 짧은 회문 반복서열(CRISPR) 연관 트랜스포사아제

제7장 지역별/국가별 분석

  • 세계의 희귀질환 유전체 편집 시장 : 지역별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년
  • 세계의 희귀질환 유전체 편집 시장 : 국가별, 분석과 예측, 2019년-2024년, 2024년-2029년, 2034년

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

  • 희귀질환 유전체 편집 시장 : 경쟁 구도
  • 희귀질환 유전체 편집 시장 : 기업 프로파일
    • Regeneron Pharmaceuticals Inc. : 개요, 제품 및 서비스, 전략 및 재무 분석
    • Sarepta Therapeutics Inc. : 개요, 제품 및 서비스, 전략 및 재무 분석
    • Ultragenyx Pharmaceutical Inc. : 개요, 제품 및 서비스, 전략 및 재무 분석
    • Beam Therapeutics Inc. : 개요, 제품 및 서비스, 전략 및 재무 분석
    • CRISPR Therapeutics AG : 개요, 제품 및 서비스, 전략 및 재무 분석

제31장 기타 주요 기업 및 혁신 기업

  • Rocket Pharmaceuticals Inc.
  • Sangamo Therapeutics Inc.
  • Arcturus Therapeutics Holdings Inc.
  • Editas Medicine Inc.
  • Krystal Biotech Inc.
  • Caribou Biosciences Inc
  • Bluebird Bio Inc.
  • SpliceBio SL
  • Cellectis SA
  • Generation Bio Co.
  • iECURE Inc.
  • Affinia Therapeutics Inc.
  • Precision Biosciences Inc.
  • Intellia Therapeutics Inc.
  • MeiraGTx Holdings plc

제32장 세계 시장 경쟁 벤치마킹과 대시보드

제33장 주요 인수합병(M&A)

제34장 최근 시장 동향

제35장 시장의 잠재력이 높은 국가, 부문, 전략

  • 희귀질환 유전체 편집 시장 2029년 : 새로운 기회를 제공하는 국가
  • 희귀질환 유전체 편집 시장 2029년 : 새로운 기회를 제공하는 부문
  • 희귀질환 유전체 편집 시장 2029년 : 성장 전략
    • 시장 동향에 기초한 전략
    • 경쟁 전략

제36장 부록

SHW

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.

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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 market characteristics section of the report defines and explains the market.
  • The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
  • The forecasts are made after considering the major factors currently impacting the market. These include:

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.

  • Market segmentations break down the market into sub markets.
  • The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth.
  • The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
  • The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.

Scope

  • Markets Covered:1) By Technology: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Or CRISPR-Associated Protein 9; Transcription Activator-Like Effector Nucleases (TALEN); Zinc Finger Nucleases (ZFN); Other Technologies
  • 2) By Disease Type: Monogenic Disorders; Multifactorial Disorders; Chromosomal Disorders
  • 3) By Application: Therapeutics; Research
  • 4) By End User: Biotechnology And Pharmaceutical Companies; Academic And Government Research Institutes; Hospitals
  • Subsegments:
  • 1) By Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Or CRISPR-Associated Protein 9: Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 9; Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 12; Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 13; Base Editing; Prime Editing
  • 2) By Transcription Activator-Like Effector Nucleases (TALEN): Standard Transcription Activator-Like Effector Nucleases (TALEN); Designer Transcription Activator-Like Effector Nucleases (dTALEN); Transcription Activator-Like Effector Nucleases (TALEN) Paired Nucleases
  • 3) By Zinc Finger Nucleases (ZFN): Standard Zinc Finger Nucleases (ZFN); Engineered Zinc Finger Nucleases (ZFN); Modular Zinc Finger Nucleases (ZFN)
  • 4) By Other Technologies: Meganucleases; Ribonucleic Acid (RNA)-Guided Nucleases; Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Transposases
  • Companies Mentioned: 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.; MeiraGTx Holdings plc
  • Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
  • Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
  • Time Series: Five years historic and ten years forecast.
  • Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita,
  • Data Segmentations: country and regional historic and forecast data, market share of competitors, market segments.
  • Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
  • Delivery Format: PDF, Word and Excel Data Dashboard.

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

  • 4.1. Supply Chain Impact from Tariff War & Trade Protectionism

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

  • 5.1. Global Rare Disease Genome Editing PESTEL Analysis (Political, Social, Technological, Environmental and Legal Factors, Drivers and Restraints)
  • 5.2. Analysis Of End Use Industries
  • 5.3. Global Rare Disease Genome Editing Market Growth Rate Analysis
  • 5.4. Global Rare Disease Genome Editing Historic Market Size and Growth, 2019 - 2024, Value ($ Billion)
  • 5.5. Global Rare Disease Genome Editing Forecast Market Size and Growth, 2024 - 2029, 2034F, Value ($ Billion)
  • 5.6. Global Rare Disease Genome Editing Total Addressable Market (TAM)

6. Rare Disease Genome Editing Market Segmentation

  • 6.1. Global Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Or CRISPR-Associated Protein 9
  • Transcription Activator-Like Effector Nucleases (TALEN)
  • Zinc Finger Nucleases (ZFN)
  • Other Technologies
  • 6.2. Global Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Monogenic Disorders
  • Multifactorial Disorders
  • Chromosomal Disorders
  • 6.3. Global Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Therapeutics
  • Research
  • 6.4. Global Rare Disease Genome Editing Market, Segmentation By End User, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Biotechnology And Pharmaceutical Companies
  • Academic And Government Research Institutes
  • Hospitals
  • 6.5. Global Rare Disease Genome Editing Market, Sub-Segmentation Of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Or CRISPR-Associated Protein 9, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 9
  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 12
  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Protein 13
  • Base Editing
  • Prime Editing
  • 6.6. Global Rare Disease Genome Editing Market, Sub-Segmentation Of Transcription Activator-Like Effector Nucleases (TALEN), By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Standard Transcription Activator-Like Effector Nucleases (TALEN)
  • Designer Transcription Activator-Like Effector Nucleases (dTALEN)
  • Transcription Activator-Like Effector Nucleases (TALEN) Paired Nucleases
  • 6.7. Global Rare Disease Genome Editing Market, Sub-Segmentation Of Zinc Finger Nucleases (ZFN), By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Standard Zinc Finger Nucleases (ZFN)
  • Engineered Zinc Finger Nucleases (ZFN)
  • Modular Zinc Finger Nucleases (ZFN)
  • 6.8. Global Rare Disease Genome Editing Market, Sub-Segmentation Of Other Technologies, By Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • Meganucleases
  • Ribonucleic Acid (RNA)-Guided Nucleases
  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Associated Transposases

7. Rare Disease Genome Editing Market Regional And Country Analysis

  • 7.1. Global Rare Disease Genome Editing Market, Split By Region, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 7.2. Global Rare Disease Genome Editing Market, Split By Country, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

8. Asia-Pacific Rare Disease Genome Editing Market

  • 8.1. Asia-Pacific Rare Disease Genome Editing Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 8.2. Asia-Pacific Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 8.3. Asia-Pacific Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 8.4. Asia-Pacific Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

9. China Rare Disease Genome Editing Market

  • 9.1. China Rare Disease Genome Editing Market Overview
  • 9.2. China Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion
  • 9.3. China Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion
  • 9.4. China Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F,$ Billion

10. India Rare Disease Genome Editing Market

  • 10.1. India Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 10.2. India Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 10.3. India Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

11. Japan Rare Disease Genome Editing Market

  • 11.1. Japan Rare Disease Genome Editing Market Overview
  • 11.2. Japan Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 11.3. Japan Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 11.4. Japan Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

12. Australia Rare Disease Genome Editing Market

  • 12.1. Australia Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 12.2. Australia Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 12.3. Australia Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

13. Indonesia Rare Disease Genome Editing Market

  • 13.1. Indonesia Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 13.2. Indonesia Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 13.3. Indonesia Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

14. South Korea Rare Disease Genome Editing Market

  • 14.1. South Korea Rare Disease Genome Editing Market Overview
  • 14.2. South Korea Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 14.3. South Korea Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 14.4. South Korea Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

15. Western Europe Rare Disease Genome Editing Market

  • 15.1. Western Europe Rare Disease Genome Editing Market Overview
  • 15.2. Western Europe Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 15.3. Western Europe Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 15.4. Western Europe Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

16. UK Rare Disease Genome Editing Market

  • 16.1. UK Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 16.2. UK Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 16.3. UK Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

17. Germany Rare Disease Genome Editing Market

  • 17.1. Germany Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 17.2. Germany Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 17.3. Germany Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

18. France Rare Disease Genome Editing Market

  • 18.1. France Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 18.2. France Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 18.3. France Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

19. Italy Rare Disease Genome Editing Market

  • 19.1. Italy Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 19.2. Italy Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 19.3. Italy Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

20. Spain Rare Disease Genome Editing Market

  • 20.1. Spain Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 20.2. Spain Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 20.3. Spain Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

21. Eastern Europe Rare Disease Genome Editing Market

  • 21.1. Eastern Europe Rare Disease Genome Editing Market Overview
  • 21.2. Eastern Europe Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 21.3. Eastern Europe Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 21.4. Eastern Europe Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

22. Russia Rare Disease Genome Editing Market

  • 22.1. Russia Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 22.2. Russia Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 22.3. Russia Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

23. North America Rare Disease Genome Editing Market

  • 23.1. North America Rare Disease Genome Editing Market Overview
  • 23.2. North America Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 23.3. North America Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 23.4. North America Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

24. USA Rare Disease Genome Editing Market

  • 24.1. USA Rare Disease Genome Editing Market Overview
  • 24.2. USA Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 24.3. USA Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 24.4. USA Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

25. Canada Rare Disease Genome Editing Market

  • 25.1. Canada Rare Disease Genome Editing Market Overview
  • 25.2. Canada Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 25.3. Canada Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 25.4. Canada Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

26. South America Rare Disease Genome Editing Market

  • 26.1. South America Rare Disease Genome Editing Market Overview
  • 26.2. South America Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 26.3. South America Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 26.4. South America Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

27. Brazil Rare Disease Genome Editing Market

  • 27.1. Brazil Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 27.2. Brazil Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 27.3. Brazil Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

28. Middle East Rare Disease Genome Editing Market

  • 28.1. Middle East Rare Disease Genome Editing Market Overview
  • 28.2. Middle East Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 28.3. Middle East Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 28.4. Middle East Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

29. Africa Rare Disease Genome Editing Market

  • 29.1. Africa Rare Disease Genome Editing Market Overview
  • 29.2. Africa Rare Disease Genome Editing Market, Segmentation By Technology, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 29.3. Africa Rare Disease Genome Editing Market, Segmentation By Disease Type, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion
  • 29.4. Africa Rare Disease Genome Editing Market, Segmentation By Application, Historic and Forecast, 2019-2024, 2024-2029F, 2034F, $ Billion

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

  • 30.1. Rare Disease Genome Editing Market Competitive Landscape
  • 30.2. Rare Disease Genome Editing Market Company Profiles
    • 30.2.1. Regeneron Pharmaceuticals Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 30.2.2. Sarepta Therapeutics Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 30.2.3. Ultragenyx Pharmaceutical Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 30.2.4. Beam Therapeutics Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 30.2.5. CRISPR Therapeutics AG Overview, Products and Services, Strategy and Financial Analysis

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

  • 31.1. Rocket Pharmaceuticals Inc.
  • 31.2. Sangamo Therapeutics Inc.
  • 31.3. Arcturus Therapeutics Holdings Inc.
  • 31.4. Editas Medicine Inc.
  • 31.5. Krystal Biotech Inc.
  • 31.6. Caribou Biosciences Inc
  • 31.7. Bluebird Bio Inc.
  • 31.8. SpliceBio S.L.
  • 31.9. Cellectis SA
  • 31.10. Generation Bio Co.
  • 31.11. iECURE Inc.
  • 31.12. Affinia Therapeutics Inc.
  • 31.13. Precision Biosciences Inc.
  • 31.14. Intellia Therapeutics Inc.
  • 31.15. MeiraGTx Holdings plc

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

  • 35.1 Rare Disease Genome Editing Market In 2029 - Countries Offering Most New Opportunities
  • 35.2 Rare Disease Genome Editing Market In 2029 - Segments Offering Most New Opportunities
  • 35.3 Rare Disease Genome Editing Market In 2029 - Growth Strategies
    • 35.3.1 Market Trend Based Strategies
    • 35.3.2 Competitor Strategies

36. Appendix

  • 36.1. Abbreviations
  • 36.2. Currencies
  • 36.3. Historic And Forecast Inflation Rates
  • 36.4. Research Inquiries
  • 36.5. The Business Research Company
  • 36.6. Copyright And Disclaimer
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