Gene therapy for rare diseases refers to the innovative medical approach that utilizes genes as medicines to treat disease. Gene therapy involves inserting a working copy of the gene into the body’s cells to correct genetic defects. The global rare disease patient population is estimated to be over 300 million. Gene therapy offers advantages over other traditional medicines for rare diseases as it has potential to cure them permanently rather than just manage symptoms. The therapy could help address unmet medical needs of rare disease patients.
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. Advancements and approvals of gene therapies for rare diseases are driving growth of the market.
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. These players are focused on developing novel gene therapy solutions through partnerships and clinical trials.
The key opportunities in the market include large rare disease patient population representing significant commercial potential, increasing research funding and investments for development of gene therapies, and growing demand for personalized medicine. Regulatory bodies support acceleration of development and approval of drugs for rare diseases through various incentives and programs.
Technological advancements like introduction of modified viruses as vectors to deliver new normal copy of gene, development of non-viral vectors, and adoption of advanced gene editing tools like CRISPR-Cas9 are expanding applications of gene therapy and aiding efforts to develop safe and effective treatments.
Growing research in gene therapy approach supported by collaborations between pharmaceutical companies and research institutes is expected to aid new product development. Increasing awareness about potential benefits of gene therapy approach over traditional medicine among patients and healthcare professionals is also likely to boost market growth over coming years.
Challenges in Gene Therapy for Rare Disease Market
Gene therapy for rare diseases market faces many challenges currently due to the rare and unique nature of these diseases. Some of the key challenges are lack of established treatment paradigms, high research and development costs, limited patient pool availability making it difficult to conduct clinical trials, complex manufacturing processes and scaled production, storage and distribution challenges. Regulators also face difficulties in evaluating benefits versus risks for new therapies in small patient populations.
Strength: Gene therapy offers potential for one-time or short-term treatments to provide long lasting benefits for genetic diseases.
Weakness: High costs involved in R&D, clinical trials and manufacturing. Complex scientific knowledge required.
Opportunity: Growing focus on developing treatments for rare diseases presents large market potential. Increasing collaboration between industry, academia and non-profits.
Threats: Safety concerns over viral vectors used and risk of cancer. Reimbursement and regulatory hurdles especially in US.
North America is currently the largest geographical region in the gene therapy for rare disease market in terms of value owing to established research infrastructure, streamlined regulatory system and presence of key market players. Europe is the second largest regional market driven by strong support for rare disease research from European Commission through programs like European Joint Program on Rare Diseases.
Asia Pacific region is witnessing the fastest growth in the gene therapy for rare disease market due to increasing healthcare expenditure, rising awareness, setting up of research hubs and improving regulatory guidelines in countries like China, Japan and India. Government support through funding and initiatives are also supporting growth of gene therapy for rare diseases market in the Asia Pacific region.
1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it