Rare Disease Genome Editing Market Set to Reach $2.45 Billion by 2029

The Business Research Company

The Business Research Company's Rare Disease Genome Editing Global Market Report 2025 – Market Size, Trends, And Global Forecast 2025-2034

LONDON, GREATER LONDON, UNITED KINGDOM, October 14, 2025 /EINPresswire.com/ -- What Is The Expected Cagr For The Rare Disease Genome Editing Market Through 2025?
The market size of genome editing for rare diseases has seen significant growth over the past few years. The market will expand from 2024's $1.09 billion to $1.29 billion in 2025, which represents a compound annual growth rate (CAGR) of 17.9%. This growth in the historic period is due to factors like increased government funding towards genetic research, a growing understanding of rare disease diagnosis, further collaboration among biotech firms, and higher investment in personalized medicine.

The market size of genome editing for rare diseases is projected to witness a significant rise in the near future. By 2029, it is anticipated to be valued at $2.46 billion with a compound annual growth rate (CAGR) of 17.6%. This surge during the forecast period can be credited to the escalating adoption of AI in genome editing, increasing need for targeted treatments, ongoing technological progress in gene delivery systems, rising incidents of multi-gene disorders, and an increased emphasis on pediatric rare diseases. Notable trends during this period will include progress in multi-gene editing techniques, implementation of AI-based genomic analysis, the growth of technology-aided personalized gene therapies, advancements in in vivo gene delivery systems, and improvements in high-throughput genome screening.

Download a free sample of the rare disease genome editing market report:
https://www.thebusinessresearchcompany.com/sample.aspx?id=28313&type=smp

What Are The Key Factors Driving Growth In The Rare Disease Genome Editing Market?
The rare disease genome editing market is set to grow due to the rising emphasis on precision medicine. This implies that prevention, diagnosis, and treatment are customized according to an individual's genetic composition, environment, and lifestyle. Precision medicine involves a specialized healthcare approach that employs a patient's genes, environment, and way of life to provide more exact and effective treatments. The expanding focus on this type of medicine stems from advancements in genomic sequencing and biomarker identification, which allow for accurate detection and targeted therapies. The rare disease genome editing market boosts precision medicine by facilitating the targeted correction of genetic anomalies, thereby personalizing and enhancing treatments. It improves healthcare by tackling the underlying causes of rare diseases, enhancing patient results, and cutting down on dependence on symptom management. For example, in March 2024, Novotech, a biotech company based in Australia, announced that in 2023, 43% of the 217 FDA-approved oncology therapies were precision oncology treatments, with 78 incorporating DNA or NGS-detectable biomarkers. Hence, the escalating focus on precision medicine is propelling the growth of the rare disease genome editing market.

What Are The Top Players Operating In The Rare Disease Genome Editing Market?
Major players in the Rare Disease Genome Editing Global Market Report 2025 include:
• 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.

What Are The Upcoming Trends Of Rare Disease Genome Editing Market In The Globe?
Leading firms in the rare disease genome editing market are putting efforts into the creation of superior therapeutic platforms like CRISPR-based gene editing systems. These advancements aim to enhance the precision of treatment, boost therapeutic outcomes and tackle genetically transmitted diseases previously deemed incurable. CRISPR-based gene editing technology is an innovative technique that allows for exact alterations to the genome. This facilitates the correction of genetic faults or the incorporation of therapeutic genes into cells. For example, in December 2023, US-based biopharmaceutical enterprise, Vertex Pharmaceuticals Inc., collaborated with CRISPR Therapeutics Inc., a US-based biotechnology research corporation. They obtained conditional marketing authorization from the United Kingdom's (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) for their product CASGEVY (exagamglogene autotemcel, exa-cel). This product is recommended for patients aged 12 and above suffering from sickle cell disease (SCD) and constant vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT). Casgevy functions by editing a patient's hematopoietic stem cells, reactivating fetal hemoglobin production. This process minimizes or eliminates signs of disease, offering a potential effective cure for the said conditions.

Comprehensive Segment-Wise Insights Into The Rare Disease Genome Editing Market
The rare disease genome editing market covered in this report is segmented as

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

View the full rare disease genome editing market report:
https://www.thebusinessresearchcompany.com/report/rare-disease-genome-editing-global-market-report

Global Rare Disease Genome Editing Market - Regional Insights
For the year 2024, North America led the global market for rare disease genome editing and Asia-Pacific is anticipated to exhibit the highest growth rate in the projected period. The report on the global market for rare disease genome editing encompasses regions such as Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, and Africa.

Browse Through More Reports Similar to the Global Rare Disease Genome Editing Market 2025, By The Business Research Company

Genome Editing Or Genome Engineering Global Market Report 2025
https://www.thebusinessresearchcompany.com/report/genome-editing-or-genome-engineering-global-market-report

Crispr Gene Editing Global Market Report 2025
https://www.thebusinessresearchcompany.com/report/crispr-gene-editing-global-market-report

Gene Editing Global Market Report 2025
https://www.thebusinessresearchcompany.com/report/gene-editing-global-market-report

Speak With Our Expert:
Saumya Sahay
Americas +1 310-496-7795
Asia +44 7882 955267 & +91 8897263534
Europe +44 7882 955267
Email: saumyas@tbrc.info
The Business Research Company - www.thebusinessresearchcompany.com

Follow Us On:
• LinkedIn: https://in.linkedin.com/company/the-business-research-company

Oliver Guirdham
The Business Research Company
+44 7882 955267
info@tbrc.info
Visit us on social media:
LinkedIn
Facebook
X

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.