Rare Disease Genome Editing Market on Track for Strong Growth, Estimated to Grow at 17.6% CAGR Through 2029

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What Is the Expected CAGR for the Rare Disease Genome Editing Market Through 2025?
In recent times, there has been a swift expansion in the market size of rare disease genome editing. The market, which is projected to inflate from $1.09 billion in 2024 to $1.29 billion in 2025, is growing at a Compound Annual Growth Rate (CAGR) of 17.9%. Factors such as enhanced government support for genetic studies, heightened consciousness about rare disease diagnosis, an uptick in partnerships between biotech firms, and burgeoning investment in personalized medicine have all contributed to the growth observed in the historical period.

What's the Projected Size of the Global Rare Disease Genome Editing Market by 2029?
The market for genome editing for rare diseases is predicted to experience swift expansion in the foroming years. It is forecasted to reach a value of $2.46 billion in 2029, with a compound annual growth rate (CAGR) of 17.6%. The anticipated surge during this period can be credited to the escalating variance of artificial intelligence in genome editing, increased need for precision treatments, advancing technological developments in gene delivery systems, growing presence of multiple gene disorders, and heightened focus on rare diseases in children. Noteworthy trends during this prognosis period encompass progress in multi-gene editing methods, an amalgamation of AI-powered genomic examination, advancement of custom-made gene therapies using technology, progress in in vivo gene delivery systems, and leaps in high-throughput genome screening.

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Top Growth Drivers in the Rare Disease Genome Editing Industry: What's Accelerating the Market?
The rare disease genome editing market's growth is anticipated to surge due to the increasing emphasis on precision medicine. Precision medicine is an approach that is tailored to an individual's genetic makeup, environment, and lifestyle. It aims to customise prevention, diagnosis, and treatments. This increased emphasis on precision medicine can be attributed to advancements in genomic sequencing and the identification of biomarkers, which pave the way for precise detection and targeted therapies. The rare disease genome editing market facilitates precision medicine by allowing for targeted amendments of genetic mutations, resulting in more bespoke and effective treatments. It revolutionizes healare by tackling the fundamental cause of rare disorders, enhancing patient outcomes and decreasing dependence on symptomatic management. For instance, Novotech, a biotechnology company based in Australia, incorporated DNA or NGS-detectable biomarkers into 78 of 217 FDA-approved oncology therapies, accounting for 43% of them in 2023. Thus, the escalating emphasis on precision medicine is fuelling the growth of the rare disease genome editing market.

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What Trends Will Shape the Rare Disease Genome Editing Market Through 2029 and Beyond?
In the rare disease genome editing market, leading companies are concentrating their efforts on developing sophisticated treatment platforms, such as CRISPR-based gene editing systems. These systems aim to increase the accuracy of treatment, enhance therapeutic results, and tackle genetic diseases that were once deemed untreatable. CRISPR-based gene editing systems refer to cutting-edge technologies that make precise modifications to the genome, facilitating the rectification of genetic mistakes or the incorporation of therapeutic genes into cells. For example, in December 2023, Vertex Pharmaceuticals Inc., an American biopharmaceutical firm, joined forces with CRISPR Therapeutics Inc., an American biotechnology research firm. They received conditional marketing approval from the UK's Medicines and Healare products Regulatory Agency (MHRA) for CASGEVY (exagamglogene autotemcel, exa-cel). This treatment is recommended for patients aged 12 and above suffering from sickle cell disease (SCD) and recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia (TDT). Casgevy functions by editing a patient's hematopoietic stem cells to revive fetal hemoglobin production, thereby reducing or eliminating disease symptoms and potentially providing a functional cure for these conditions.

What Are the Main Segments in 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

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Which Top Companies are Driving Growth in the Rare Disease Genome Editing Market?
Major companies operating 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.

Which Regions Will Dominate the Rare Disease Genome Editing Market Through 2029?
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.

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