Gene Therapy Market

Drivers

There is an increase in the prevalence of chronic diseases such as neurological disorders, oncology disorders, cancer, autoimmune diseases, and rare genetic disorders like muscular dystrophy and cystic fibrosis, as well as cardiovascular diseases globally.

• According to the National Organization for Rare Diseases, the predicted incidence is 1 in 100,000 live births per year diagnosed with spinal muscular atrophy.

Expanding therapeutic areas in unmet medical needs along with advancements in gene delivery technologies are helping companies gain a first-mover advantage and increasing competition among leading players for approval and commercialization of gene therapies.

The number of regulatory approvals for gene therapy products is increasing due to the advantages of these products, especially in the USA and Europe. For instance, the FDA estimated that by 2025, 10 to 20 innovative cell and gene therapies will be approved each year. The USFDA has received more than 200 applications for gene therapy.

Restraints

• The high price of gene therapy limits accessibility and can lead to market withdrawals due to a lack of economic sustainability.
• The cost of manufacturing critical raw materials, batch-to-batch variance, and the availability of raw materials such as viral vectors is high.
• The cost includes laboratory research, precision equipment, stringent quality control, preclinical testing, clinical trials, regulatory approval, vector production, and engineering, which increases the overall manufacturing cost.
• Due to its high price, gene therapy remains inaccessible and has limited adoption in many underdeveloped and developing countries.

Opportunities

The patient pool for gene therapies is anticipated to create opportunities in the coming years due to a wide range of treatment possibilities and effective targeting of cells. Rising healthcare expenditure, improved healthcare infrastructure, and the availability of reimbursement policies are expected to create several opportunities for key players. Gene therapy holds tremendous promise for treating a wide range of genetic disorders and rare diseases.

Challenges

• Clinical trials for gene therapies face challenges in recruiting sufficient patients, especially for rare diseases. Strict EU privacy laws require each patient’s consent for data use, slowing trials.
• In both the American and European markets, cell and gene therapies may present limited clinical data. Limited clinical data due to small patient populations creates uncertainty for payers evaluating benefits versus existing treatments.
• Delivering therapeutic genes to the right cells in the body effectively is a significant challenge in gene therapy.
• A limited understanding of genetic diseases poses a major obstacle to advancing treatment options in the gene therapy field.
• The high treatment cost is one of the factors hindering the growth of the gene therapy market.

Based on region, North America dominated the gene therapy market in 2023 in terms of revenue and is anticipated to continue its dominance during the projected period.

Based on the vector segment, AAV dominated the gene therapy market in 2023 and will continue its presence during the projected period.

Based on the delivery system, In Vivo dominated the gene therapy market in 2023 and will also continue to exhibit its dominance during the projected period.

• Viral Vectors
• Non-Viral Vectors

• In Vivo
• Ex Vivo

• Oncological
• Rare Diseases
• Neurological
• Sensory
• Alimentary/Metabolic Disease
• Musculoskeletal
• Other

• In Feb 2024, Voyager Therapeutics entered a Capsid License Agreement and Strategic Collaboration with Novartis to advance novel gene therapies.
• In Jan 2024, Avrobio merged with Tectonic Therapeutic, forming a combined company focused on advancing Tectonic’s novel GPCR-targeted therapeutic protein.
• In Jan 2024, Oxford Biomedica plc completed the acquisition of ABL Europe, strengthening Oxford Biomedica’s position as a global pure-play cell and gene therapy CDMO.
• In Jan 2024, Novartis and Voyager entered a strategic collaboration and licensing agreement to develop gene therapies for Huntington’s disease and spinal muscular atrophy.
• In Nov 2023, AstraZeneca invested $245 million in Cellectis to boost gene therapy prospects.

1. Introduction
   1. 1.1 Different Types of Gene Therapy
   2. 1.2 Classification of Gene Therapy
2. Key Journey in development of Gene Therapy
   1. 2.1 Difference between Traditional Pharma and Gene Therapy
3. Different Processes of Gene Therapy
4. Trends
5. Drivers
6. Restraints
7. Opportunities
8. Challenges
9. Gene Therapy Ecosystem
10. Gene Therapy Value Chain
11. Key elements in Gene Therapy Value Chain
12. Gene Therapy Organizational Structure and its Functions
13. Gene Therapy Forecast
14. Segmentation (Delivery Method, Vector, Therapeutic Area)
15. Spectrum of Diseases Application
16. Key Market Analysis
    1. 16.1 North America
    2. 16.2 Europe
    3. 16.3 Asia Pacific
    4. 16.4 Middle East and Africa
    5. 16.5 Latin America
17. SWOT Analysis of Gene Therapy
18. Porter’s Five Forces Analysis
19. Gene Therapy Stakeholders
    1. 19.1 Stakeholders Involvement in GT Manufacturing and Supply
20. Digital Ecosystem
    1. 20.1 Digitally Driven system enabling gene therapy advancements.
    2. 20.2 Technological Advancements and its Applications in Gene Therapy
21. Regulatory Landscape
    1. 21.1 Regulatory Framework
22. Gene Therapy Approval Pathway in Various Countries
23. Reimbursement model
    1. 23.1 Table of Reimbursement model across Various Countries
24. Challenges and Solutions in Reimbursement Models
25. Mergers and Acquisitions
26. Collaboration / Investment / Agreement
27. FDA Approved Gene Therapy
28. EU and Other Regulatory bodies Gene therapy approval.
    1. 28.1 Gene Therapy Approval Status Across Globe
29. Anticipated Gene therapy Approval 2024
30. Players in Gene Therapy Market
31. Players Market Analysis
    1. 31.1 Astellas Pharma
       1. 31.1.1 Introduction
       2. 31.1.2 Astellas Pipeline Analysis
       3. 31.1.3 Merger and Acquisitions
       4. 31.1.4 Headlines Related to Astellas Pharma
    2. 31.2 Novartis Gene Therapies
       1. 31.2.1 Introduction
       2. 31.2.2 Product Portfolio
       3. 31.2.3 Pipeline Analysis
       4. 31.2.4 Merger and Acquisitions
       5. 31.2.5 Collaboration/ Investment / Agreement
       6. 31.2.6 Headlines Related to Novartis Gene Therapies
    3. 31.3 Bluebird Bio Inc
       1. 31.3.1 Introduction
       2. 31.3.2 Product Portfolio
       3. 31.3.3 Pipeline Analysis
       4. 31.3.4 SWOT Analysis
    4. 31.4 Sarepta Therapeutics
       1. 31.4.1 Introduction
       2. 31.4.2 Product Portfolio
       3. 31.4.3 Pipeline Analysis
       4. 31.4.4 Merger and Acquisition
       5. 31.4.5 Agreement / Collaboration
    5. 31.5 Regenxbio
       1. 31.5.1 Introduction
       2. 31.5.2 Pipeline Analysis
       3. 31.5.3 SWOT Analysis
       4. 31.5.4 Agreements / Collaboration