Market Dynamics:

The increasing research and development investments by pharmaceutical giants are expected to drive the growth of gene therapy for rare disease market over the forecast period. For instance, in 2021, The French Biotech Valneva SE announced an investment of €24 million in the expansion of its gene therapy facilities in Scotland. Furthermore, favorable regulatory policies are also expected to fuel the market growth. The Orphan Drug Act passed in 1983 in the US provides incentives for sponsors to develop drugs for rare diseases. The incentives include research grants, tax credits, and 7 years of market exclusivity upon marketing authorization. Moreover, the increasing prevalence of rare diseases is also propelling the demand for effective treatment options. However, the high costs associated with gene therapy development and manufacturing pose a challenge to market growth.

SWOT Analysis

Strength: Gene therapy for rare diseases offers long-term treatment compared to other conventional treatment options. It has the potential to cure certain genetic diseases permanently by correcting the underlying genetic problem. Researchers are continuously working on refining gene therapy treatment to improve safety and effectiveness.
Weakness: Gene therapy is still an evolving field and not all rare genetic diseases may have effective gene therapy treatment available yet. Developing safe and effective gene therapy is technically challenging and requires extensive research and clinical trials. The high development costs also make the final treatment expensive.
Opportunity: With continued advancements in gene therapy research, more rare genetic diseases could potentially be treated or cured with this approach. Successful gene therapies will provide major clinical benefits to patients and drive the market growth. Growing awareness among patients may further boost the demand.
Threats: Safety issues and long-term effectiveness concerns continue to be evaluated for newer gene therapies. Stringent regulations can delay the approval and commercialization of new treatments. Stiff competition from other emerging drug candidates is also a challenge for growth of the gene therapy market.

Key Takeaways

The Global Gene Therapy For Rare Disease Market Size is expected to witness high growth over the forecast period. Rising prevalence of several genetic disorders and limited treatment options available have boosted the demand for gene therapies. According to estimates, the market valued at US$ 193.56 billion in 2023 and is projected to reach over US$ 306 billion by 2030, exhibiting a CAGR of around 5.9%.

Regional analysis
North America currently dominates the global market and is expected to maintain its lead position during the forecast period as well. Higher adoption of advanced treatments, strong presence of leading biotech companies and growing research activities in the region are some of the key growth drivers. Europe is anticipated to witness the highest growth over the coming years owing to rising government support for gene therapy research and increasing focus on rare disease treatment. Asia Pacific offers immense growth potential with expanding healthcare infrastructure and growing patient population.

Key players
Key players operating in the gene therapy for rare disease market 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. These companies have strong portfolio of gene therapies in clinical development and commercialization for various rare genetic disorders. Strategic collaborations and acquisitions remain important strategies to strengthen their market presence.

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