Gene therapy has emerged as a promising approach for treating rare diseases by repairing or replacing a mutated gene responsible for a genetic disorder. Gene therapy involves modifying genes or introducing foreign genes into cells and tissues to treat disease. It aims to compensate for abnormal genes that cause genetic disorders by delivering normal genes to replace or supplement missing or mutated genes. The gene therapy for rare disease market has witnessed significant growth in the recent past owing to growing prevalence of genetic disorders and rising R&D investments by biopharmaceutical companies for development of novel gene therapies.

The global gene therapy for rare disease market is estimated to be valued at US$ 8.9 BN in 2024 and is expected to exhibit a CAGR of 9.0% over the forecast period 2024 to 2030.

Key Takeaways
Key players 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 are focusing on new product launches and collaborations to expand their portfolios and geographic presence.

The growing incidence of rare genetic disorders globally is fueling the demand for effective treatment options. Gene Therapy For Rare Disease Market Demand According to some estimates, over 300 million people are affected by rare diseases worldwide. Gene therapy provides a targeted treatment approach with potential to cure genetic diseases at the molecular level.

North America currently dominates the global market due to strong presence of key biopharma companies and availability of favorable regulations. However, Asia Pacific is expected to witness highest growth owing to rising healthcare expenditures, large patient pool, and increasing awareness. Collaborations between global drug developers and Asian governments are also facilitating expansion of clinical trials in the region.

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