Gene therapy is a developing approach utilized to treat different hereditary issue by introducing DNA into patients’ cells while treatment is in progress. Gene therapy holds the potential to treat a wide spectrum of rare diseases by correcting genetic mutations that underlie life-threatening and debilitating medical conditions.
The global gene therapy for rare disease market is estimated to be valued at US$ 8.9 billion in 2024 and is expected to exhibit a CAGR of 9.0% over the forecast period of 2024 to 2030.
Key Takeaways
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. There is growing demand for gene therapies to treat rare genetic disorders owing to the scarcity of alternative treatment options. Technological advancements have enabled more efficient and targeted delivery of gene therapies.
Market Trends
Rare disease patients have poorer quality of life outcomes compared to other chronic conditions due to lack of effective therapeutic options. Gene Therapy For Rare Disease Market Demand offer a promising solution by specifically targeting the underlying genetic defects. Another key trend is the adoption of new technologies such as gene editing to develop more tailored gene therapies with improved safety and efficacy profiles.
Market Opportunities
Increasing investments from biotech companies as well as government funding create potential opportunities for market players. Also, the large bank of genetic data available today facilitates gene therapy development by expediting target identification and validation.
Impact of COVID-19 on Gene Therapy for Rare Disease Market Growth
The COVID-19 pandemic has significantly impacted the Gene Therapy for Rare Disease market. The lockdowns imposed globally led to delays in ongoing clinical trials for gene therapies in rare diseases. Various companies had to pause patient enrollment in clinical trials to adhere to social distancing guidelines. This posed challenges to the timelines and development of new gene therapies entering late-stage clinical trials and market approvals. Telehealth emerged as a important tool for companies to continue monitoring patients already enrolled in ongoing trials and collect required clinical data without in-person visits. However, remote patient monitoring could not replace in-person procedures required for certain gene therapy trials.
As the pandemic continues, companies are adapting strategies to minimize disruption to ongoing clinical programs. Efforts are being made to utilize decentralized or hybrid trial models which allow certain trial procedures to be conducted virtually or at local doctor’s facilities rather than centralized trial sites. New screening and safety protocols have also been established at clinical trial sites to resume enrollment of new patients. The pandemic has highlighted the need for gene therapies in rare diseases with limited treatment options. Government efforts to promote research in cell and gene therapy development are expected to boost the market in the long run despite short term delays caused by the ongoing healthcare crisis.
Gene Therapy Concentration in North America
The North American region, especially the United States, currently holds the largest share of the global Gene Therapy for Rare Disease market in terms of value. This is due to strong presence of leading biotechnology and pharmaceutical companies developing gene therapies for rare diseases. North America also has streamlined regulatory policies through FDA to fast track the approval of newly developed gene therapies intended for life threatening or seriously debilitating rare conditions. Large patient population afflicted with various rare genetic disorders also makes North America an attractive market for clinical trials and commercialization of gene therapies. Rising healthcare investments and increased research funding further supports the growth of the Gene Therapy for Rare Disease market in this region.
Fastest Growing Region – Europe
The European region is expected to witness the fastest growth in the Gene Therapy for Rare Disease market over the forecast period. This can be attributed to growing research collaborations between academic institutes and biopharmaceutical companies supported through funding initiatives by European Commission. Countries like the UK, Germany, France are emerging hubs for conducting clinical trials of novel therapies including gene therapies. Regulatory guidelines under EMA also provide advantages for approval of gene therapies targeting rare disease with no existing treatments. Increasing patient awareness and demand for effective treatments will drive the Gene Therapy for Rare Disease market growth across major European markets.
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