Gene Therapy for Rare Disease Market Size 2025-2033: Trends & Forecast 2025-2032 | Global Growth Insights

Gene Therapy for Rare Disease Market Size reached US$ 3.01 Billion in 2024 and is expected to reach US$ 24.54 Billion by 2033, growing at a CAGR of 26.5% during the forecast period 2025-2033.

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United States - Recent Industry Developments

✅ November 2025: FDA granted priority review to a novel AAV-based gene therapy targeting Duchenne muscular dystrophy.

✅ October 2025: Pfizer launched large-scale manufacturing expansion for clinical-grade viral vectors for rare disease programs.

✅ September 2025: Bluebird Bio entered into multi-center collaborations to accelerate gene therapy adoption in U.S. pediatric hospitals.

Japan - Recent Industry Developments

✅ November 2025: Takeda Pharmaceuticals progressed Phase III gene therapy trials for hemophilia A using next-gen capsid engineering.

✅ October 2025: Japan's PMDA introduced accelerated approval pathways for advanced therapies treating ultra-rare genetic conditions.

✅ September 2025: A new consortium of universities and biotech firms launched research for localized, population-specific gene variants.

GCC - Recent Industry Developments

✅ November 2025: Dubai Health Authority announced new genomic facilities to support gene-therapy-based precision medicine for rare diseases.

✅ October 2025: Saudi Arabia initiated clinical research centers specializing in gene therapy for inherited metabolic disorders.

✅ September 2025: Qatar Foundation invested in rare-disease biobanking and gene therapy R&D to support regional treatment accessibility.

List of Top Key Player:

Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics Group., Krystal Biotech, Inc

Forecast Projection:

The Global Gene Therapy For Rare Disease Market is poised for significant growth between 2025 and 2033. In 2024, the market maintained a steady upward trajectory, and with strategic initiatives by leading players accelerating adoption, the market is expected to soar throughout the forecast period. Companies leveraging these trends are well-positioned to capture emerging opportunities and maximize revenue potential.

Recent M&A Activity

1. Dealmaking accelerates on CRISPR/in vivo editing for ultra-rare indications, with USD 70B+ pharma M&A in 2025 led by rare disease/cell-gene assets.

2. Eli Lilly acquired Adverum Biotechnologies (2025, ~USD 1B upfront + milestones), adding clinical-stage ocular gene therapies for rare retinal diseases like wet AMD (ADVM-022 AAV vector).​

3. Regeneron partnered Tessera Therapeutics (Nov 2025, USD 150M upfront + USD 125M milestones/equity) for alpha-1 antitrypsin deficiency (AATD) gene editing, sharing costs/profits on liver/lung rare disease program.​

4. Vertex/Orna (Jan 2025, USD 65M upfront + USD 635M milestones) for SCD/TDT gene-editing; Nippon Shinyaku/Regenxbio late-stage rare disease expansion (USD 110M upfront).​

5. H1 2025: 2 mega-deals >USD 2B each in gene therapy vectors for rare diseases/hematology.

Market Intelligence Research Process:

The Global Gene Therapy For Rare Disease Market research report by DataM Intelligence combines primary and secondary data to deliver deep, actionable insights. It examines the full spectrum of factors shaping the industry, from government regulations and market conditions to competitive dynamics, historical trends, technological breakthroughs, upcoming innovations, and potential challenges. This comprehensive analysis not only highlights growth prospects but also identifies barriers, equipping businesses to navigate market volatility and capitalize on emerging opportunities.

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Key Segments:

By Vector Type: (Viral Vectors, Non-Viral Vectors)

By Technique: (Gene Addition, Gene Silencing, Gene Editing)

By Approach: (In-Vivo, Ex vivo)

By Application: (Musculoskeletal Conditions, Blood Disorders, Oncology, Ophthalmology, Others)

Global Growth Regional Analysis:

1. North America: Dominates with USD 438M cell culture media market in 2025 (17% CAGR), supported by NIH funding and fast-track pathways for 7,000 rare diseases, with billion-dollar strategic bets from pairs like Lilly-Adverum and Regeneron-Tessera.

2. Europe: Benefits from EMA orphan incentives and tax relief, while EUR 170B+ grid/digital-twin initiatives indirectly reinforce bioproduction, highlighted by the Genmab-Merus USD 8B rare immunology deal and EU consortia addressing funding bottlenecks.

3. Japan: Advances through Sakigake and conditional approval routes for ATMPs, with TEMCELL HS (allogeneic MSC for GVHD) and priority rare disease reviews, backed by deep CGT-focused R&D spending.

4. Taiwan: Gains indirect momentum from biotech hubs and semiconductor-enabled precision medicine, though standalone rare-disease gene therapy funding remains limited.

5. South Korea: Strengthened by government-led R&D programs for serious and rare diseases, with robust policy backing and PDA conference emphasis on manufacturing and clinical scale-up.

Global Snapshot: Four new gene therapy approvals in Q3 2025, with pipelines dominated by oncology and rare diseases (ASGCT Q2 2025) and key players including Lilly, Regeneron, Vertex, and Regenxbio.

Benefits of the Report:

Chapter 1 - Market Overview: Kickstarts the report with a comprehensive snapshot of the Global Gene Therapy For Rare Disease Market, summarizing key segments by region, product type, and application. Highlights include market size, segment growth potential, and short- & long-term industry outlook.

Chapter 2 - Emerging Trends: Uncovers the game-changing trends and high-impact innovations shaping the future of the industry.

Chapter 3 - Competitive Landscape: Offers a deep dive into market competition, detailing revenue shares, strategic initiatives, and recent mergers & acquisitions.

Chapter 4 - Top Player Profiles: Features detailed company profiles, covering revenue, profit margins, product lines, and major milestones for leading market players.

Chapters 5 & 6 - Regional & Country Analysis: Breaks down revenue performance across global regions, providing insights on market sizes, opportunities, and growth prospects worldwide.

Chapter 7 - Segmentation Analysis: Explores market segmentation by type, revealing high-potential categories and guiding businesses towards lucrative areas.

Chapter 8 - Application Insights: Examines downstream markets and identifies promising sectors for expansion, showing how different applications are driving growth.

Chapter 9 - Supply Chain Mapping: Maps the entire industry supply chain, highlighting upstream and downstream activities for a holistic market perspective.

Chapter 10 - Key Takeaways: Concludes with critical insights and actionable strategies, equipping stakeholders to make informed decisions and stay ahead in the market.

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FAQ's

Q1: What is the current size of the Global Gene Therapy For Rare Disease Market and its future potential?

A: The Global Gene Therapy For Rare Disease Market was valued at US$ 3.01 Billion in 2024 and is projected to surge to US$ 24.54 Billion by 2033

Q2: How fast is the Global Gene Therapy For Rare Disease Market expected to grow in the coming years?

A: The market is forecast to expand at a robust CAGR of 26.5% between 2025 and 2033

Q3: Which regions are dominating the Global Gene Therapy For Rare Disease Market and which are fastest-growing?

A: Key markets include North America, Europe, and Asia-Pacific, led by the U.S., Japan, China, and Germany, GCC

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