The gene therapy for rare disease market has seen significant advancements in recent years, as products that deliver genetic material into patient cells aim to treat conditions caused by single gene mutations or abnormalities. Gene therapy involves modifying or manipulating genes for therapeutic benefit and has emerged as a promising approach for treating rare genetic diseases by correcting defective genes or compensating for their absence or effects.

The Global Gene Therapy For Rare Disease Market is estimated to be valued at Us$ 222.99 Mn In 2024 and is expected to exhibit A CAGR Of 5.9% over the forecast period 2024 To 2030.

Key Takeaways

Key players operating in the Gene Therapy for Rare Disease are Topcon Corporation, Agfa-Gevaert N.V., Sonomed Escalon, Carl Zeiss Meditec AG, EyePACS, VersaSuite, Heidelberg Engineering GmbH, ScImage, Inc., IBM Corporation, and Visbion Limited. Key players are focusing on forming strategic partnerships and conducting clinical trials to develop advanced therapies for treating rare genetic disorders.

The growing demand for precision medicine and rise in prevalence of rare genetic diseases are driving the gene therapy for rare disease market. Gene therapies offer the potential for more targeted treatments with lower toxicity than traditional pharmaceuticals. Moreover, increasing funding initiatives for rare disease drug development by governments and non-profit organizations are further fueling market growth.

The global gene therapy for rare disease market is projected to witness steady expansion assisted by increasing healthcare expenditure in emerging nations. Strategic collaborations between industry players, academic research institutes and hospitals are helping in globalization of clinical trials. Moreover, initiatives to spread awareness about treatment options for rare diseases worldwide will aid the market expansion during the forecast period.

Market Key Trends

Vector development for effective and safe gene delivery is one of the major trends being observed in the gene therapy for rare disease market. Viral and non-viral vectors play a pivotal role in transferring therapeutic genes into target cells and developing vectors that maximize therapeutic gene expression while minimizing immune response is crucial. Companies are investing heavily in research focusing on self-complementary vectors, transposon/transposase systems, and designer nucleases to enhance the efficacy and safety of gene therapies. This innovation is expected to boost the number of gene therapy candidates entering clinical trials.

Porter’s Analysis

Threat of new entrants: Gene therapy for rare disease is a niche and highly specialized market. New entrants face significant challenges in terms of technology, expertise, regulatory requirement and capital requirement.

Bargaining power of buyers: Patients have few alternatives for rare diseases. Hence their bargaining power is low as the cost of treatment is generally very high.

Bargaining power of suppliers: There are few biotech/pharma companies developing gene therapies. High R&D costs act as barriers and suppliers have significant power over buyers.

Threat of new substitutes: No significant substitutes exist for gene therapy which offers potentially curative treatment. Alternative treatment options are generally symptoms management only.

Competitive rivalry: Competition is increasing with advancing technologies. Large players are collaborating or acquiring smaller ones to build pipeline of therapies. Funding and faster clinical development differentiates competitors.

Geographical Regions

Currently North America accounts for the largest share in the global gene therapy market for rare diseases, value-wise. This is attributed to factors such as rise in funding for research, presence of advanced healthcare infrastructure, and availability of reimbursement.

Asia Pacific region is poised to witness the fastest growth over the forecast period. Countries such as China, India, Japan, Australia and South Korea are offering lucrative opportunities for market players. Increasing healthcare expenditure, large patient pools, and improving regulatory guidelines are boosting market growth in the region.

Note:
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it