Gene Therapy for Rare Diseases Market Size Growth Forecast 2025-33 | AAV Vectors, CRISPR & Biotech Innovations

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, according to DataM Intelligence. This growth is propelled by advances in manufacturing technologies, including plug-and-play adeno-associated virus (AAV) serotype production and standardization of analytical and bioanalytical methods, which enhance the efficiency and scalability of gene therapies.

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Emerging opportunities stem from the broadening rare disease pipeline, ongoing innovation in delivery vectors, and growing biotech collaborations aimed at expanding gene therapy indications. Regulatory facilitation and governmental incentives supporting orphan drug development also foster market confidence and accelerate therapy commercialization. Leading players in this market include Novartis (Zolgensma), Roche/Genentech, Biomarin, Sarepta Therapeutics, Bluebird Bio, CSL Behring, and Vertex Pharmaceuticals.

Notable Collaborations and Acquisitions Redefining the Gene Therapy for Rare Diseases Market Outlook in 2025

1. AstraZeneca (Alexion) acquisition of Pfizer's gene therapy portfolio in May 2025: AstraZeneca's rare disease group Alexion completed a purchase and license agreement for a portfolio of preclinical rare disease gene therapy programs and enabling technologies from Pfizer. The deal cost up to $1 billion, including tiered royalties, and strengthened AstraZeneca's genomic medicine efforts by adding several novel adeno-associated virus (AAV) capsids proven effective for gene delivery and gene editing.

2. Castle Creek Biosciences financing and expansion in April 2025: Castle Creek, a rare disease-focused gene therapy company, achieved critical clinical milestones in 2025 (such as D-Fi for recessive dystrophic epidermolysis bullosa). Supported by a $75 million royalty financing from Ligand Pharmaceuticals, Castle Creek is accelerating its pipeline and expanding R&D capabilities for both ex vivo and in vivo gene therapies targeting rare metabolic and genetic disorders.

3. Bristol Myers Squibb acquisition of 2seventy bio in March 2025: BMS acquired 2seventy bio, a bluebird bio spin-off focused on gene therapies for rare diseases, for approximately $286 million. This deal expanded BMS's portfolio with innovative gene therapy candidates and solidified its position in the rare genetic disorder treatment landscape.

Innovative Technological Advancements Revolutionizing the Gene Therapy for Rare Diseases Sector in 2025

• Viral Vector Innovations: Improvements in adeno-associated virus (AAV) serotypes with higher tissue tropism and reduced immunogenicity are expanding the range of treatable rare diseases and improving safety profiles. Plug-and-play manufacturing processes and cell suspension technologies increase scalability and reduce production costs.

• Gene Editing Technologies: CRISPR-Cas9 and base editing approaches are being applied to correct mutations precisely, with therapies like exagamglogene autotemcel (exa-cel) approved for sickle cell disease and transfusion-dependent beta-thalassemia. Advances in delivery methods and off-target effect mitigation are critical focus areas.

• Single Dose and Durable Efficacy: Many approved gene therapies (e.g., Luxturna for inherited retinal disease, Zolgensma for spinal muscular atrophy, Hemgenix for hemophilia B) demonstrate long-term benefit from a one-time administration, distinguishing gene therapy from chronic treatments and driving market adoption.

• Regulatory and Manufacturing Advances: Simplified regulatory pathways, incentives for rare disease therapies, and increasing partnerships between biotech firms and governments accelerate development and approval. Standardization and automation of manufacturing processes boost reliability and accessibility.

• Expanding Disease Targets: Beyond classical rare monogenic diseases, gene therapy development is extending into rare cancers, metabolic disorders, and neuromuscular conditions, broadening market scope.

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Regional Factors Propelling Growth in the Gene Therapy for Rare Diseases Market in 2025

North America dominates the gene therapy market for rare diseases, holding over 56% market share in 2024. This leadership is driven by robust healthcare infrastructure, high investments in research and development, and advanced regulatory frameworks that facilitate faster clinical trial approvals and product commercialization.

Europe holds a strong position supported by a skilled workforce, well-established healthcare systems, and funding initiatives like the European Union's Horizon 2021 program, boosting gene therapy R&D. Countries including Germany, the UK, and France contribute significantly to market development via research collaborations, clinical development, and manufacturing capabilities.

Asia Pacific is the fastest-growing regional market, driven by increasing healthcare investments, expanding infrastructure, and rising prevalence of genetic and rare diseases in populous nations such as China, India, Japan, and South Korea. Government incentives to promote biotechnology and advances in local manufacturing improve market access.

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