Gene therapy involves the delivery of corrective genes to treat rare diseases caused by missing or defective genes. It has shown great potential in the treatment of inherited disorders like hemophilia, cystic fibrosis, and muscular dystrophy. These therapies aim to either introduce a normal copy of the defective gene or inactivate a mutated gene that is responsible for causing the disease. As genetic treatments advance, gene therapy may provide life-changing benefits for patients with rare diseases that currently have limited or no treatment options.

The global Gene Therapy for Rare Disease Market is estimated to be valued at US$ 193.56 Bn in 2023 and is expected to exhibit a CAGR of 5.9% over the forecast period 2023 to 2030, as highlighted in a new report published by Coherent Market Insights.

Market Dynamics:

One of the key drivers for the growth of the Global gene therapy for rare disease market size is the rising prevalence of rare diseases globally. According to the National Organization for Rare Disorders (NORD), it is estimated that over 300 million people are living with a rare disease worldwide. Rising awareness about genetic diseases and their treatment options is also contributing to the adoption of gene therapies. Gene therapy addresses rare diseases at their genetic root cause and provides long term treatment benefits over conventional medicines. However, high development costs and regulatory challenges continue to restrain the market growth to some extent. The growing success of gene therapy clinical trials and regulatory approvals indicate a positive outlook for the market over the forecast period.

SWOT Analysis

Strength: Gene therapy for rare diseases has a large target patient pool as rare diseases collectively affect a significant number of people worldwide. There are over 7,000 known rare diseases that collectively affect over 300 million people globally. Developing targeted gene therapies can potentially cure these rare genetic diseases. Gene therapy offers the possibility of a one-time treatment to achieve a long-lasting effect, reducing lifelong treatment and management costs compared to other therapeutics.
Weakness: Gene therapy is a complex process and developing an effective therapy requires a clear understanding of the underlying genetic cause of a disease. Due to the complexity, the success rate for gene therapy has been relatively low historically. High research and development costs associated with gene therapy development makes these treatments expensive on launch.
Opportunity: The orphan drug designation and regulatory incentives help accelerate the development and approval of therapies for rare genetic diseases. Growing support from funding agencies and foundations encourages more research in this area. Increasing awareness and diagnosis of rare diseases have accelerated the demand for targeted treatment options like gene therapy.
Threats: Safety issues and side effects remain a concern with gene therapy as permanent changes are made to a patient's DNA. Finding effective viral and non-viral delivery methods to successfully deliver the corrective genetic material inside the target cells poses technical challenges. Fierce competition from other promising emerging modalities like gene editing, mRNA, and antisense oligonucleotides threatens gene therapy's position as the premier approach for monogenic diseases.

Key Takeaways

The global gene therapy for rare disease market is expected to witness high growth over the forecast period of 2023 to 2030. The market size is projected to reach US$ 193.56 Billion in 2024 from US$ 167.12 Billion in 2022, growing at a compound annual growth rate (CAGR) of 5.9% during the forecast period.

Regional analysis: North America currently dominates the global gene therapy for rare disease market and is expected to continue its lead during the forecast period as well. Major factors driving the region's large share include the growing biotech industry, presence of major market players, rising research funding, and favorable regulatory environment in the US. Europe is the second-largest regional market supported by strategic initiatives aimed at boosting innovation and adoption of advanced therapies in rare disease treatment.

Key players: Top 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. Key players are focusing on expanding their application-specific product portfolios through partnerships, acquisitions, and collaborative agreements. They are also investing in R&D to develop innovative gene therapies with improved safety profiles and technical capabilities.

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