Rare Disease Gene Therapy Market

Rare Disease Gene Therapy Market Overview

Diseases that impact only small percentage of population are known as rare diseases. Rare disease generally occurs at younger age and are genetic, hence present throughout patient’s life. Rare diseases are also referred to as orphan disease due to lack of resources and patient population for discovering treatment.

Rare disease gene therapy includes replacing or inactivating mutated gene to fight against disease. According to the National Institute of Health (NIH), 25 million people in the U.S. are affected with 7,000 different rare diseases.

There has been shift of focus among biopharmaceutical players to develop coronavirus cure due to Covid-19, this is delaying research and development activities in rare disease gene therapy market. Halt of manufacturing activities and supply chain disruption due to trading across borders will have impact on rare disease gene therapy market growth rate.

Reduction in diagnosis and treatment adoption rate owing to decreased patient visits to healthcare facilities will reduce growth rate of rare disease gene therapy market.Increasing number of rare diseases are expected to drive the growth of rare disease gene therapy market.

Increasing collaborative research and development activities among leading biopharmaceutical innovative players is expected to fuel the growth of rare disease gene therapy market. In addition, clinical-stage development companies are allocating significant funds towards the development of the rare disease gene therapy market.

For instance, in August 2020, the U.S. based Ovid Therapeutics collaborated with Italian firm Angelini Pharma to develop, manufacture and commercialize drug gaboxadol for treatment of Angelman syndrome (rare genetic disease) in European market.

Increasing awareness among general population about available treatment options boost rare disease gene therapy market. Stringent regulatory procedure for drug approval is expected to hinder growth of rare disease gene therapy market. Risk factors associated with gene therapy will challenge rare disease gene therapy market.

Non-viral vector type gene therapy is expected to hold major revenue share for rare disease gene therapy market with increased market penetration. Based on application genetic disorder contributes higher revenue in rare disease gene therapy market. Parenteral drugs are most common type in rare disease gene therapy market.

Intravenous drugs are expected to have significant growth rate in coming decade. Hospital pharmacies is largest distribution channel in rare disease gene therapy market followed by retail pharmacies. Online pharmacies will have exponential growth with increased adoption of e-commerce in rare disease gene therapy market.

North America will continue to dominate the rare disease gene therapy market in the coming decade with a high rate of approvals and commercialization of genomic medicine. Increased awareness and availability of advanced technology propels the demand for rare disease gene therapy market in North America.

Europe holds significant share in rare disease gene therapy market with increased investments towards research and development activities. Asia-pacific is lucrative with a high growth rate in the rare disease gene therapy market during the forecast period with a large patient population in the region.

The Middle East and Africa are expected to observe the least growth in rare disease gene therapy market due to less healthcare expenditure in the region. Poor economic conditions in the Middle East and Africa make them untapped regions with fewer growth opportunities of rare disease gene therapy market.

Some key players contributing in rare disease gene therapy market are

• Pfizer Inc.
• Amgen Inc.
• Bristol-Myers Squibb Company
• AbbVie Inc.
• Novartis AG
• F. Hoffmann-La Roche Ltd.
• Sanofi S.A.
• Merck & Co.
• Teva Pharmaceutical Industries Ltd.
• Johnson & Johnson
• Bayer AG
• Baxter
• Allergen plc.
• Novo Nordisk A/S
• Vertex Pharmaceuticals
• Alexion

The report on rare disease gene therapy market covers exhaustive analysis on

• Market Segments
• Market Dynamics
• Historical Actual Market Size 2015 - 2019
• Market Size & Forecast 2020 to 2030
• Supply & Demand Value Chain
• Market Current Trends/Issues/Challenges
• Competition & Companies involved
• Technology
• Value Chain
• Market Drivers and Restraints

Regional analysis for rare disease gene therapy market includes

• North America
• Latin America
• Europe
• East Asia
• South Asia
• Oceania
• Middle East & Africa

Rare Disease Gene Therapy Market : - Segmentation

The global rare disease gene therapy market is classified based on gene therapy, indication, route of administration, distribution channel and region.

Gene Therapy

• Viral vector gene therapy
• Non-viral vector gene therapy

Indication

• Genetic diseases
• Oncology diseases
• Haematology diseases
• Infectious diseases
• Neurological diseases
• Inflammatory diseases
• Others

Administration

• Parenteral
• Intravenous
• Intraperitoneal
• Others

Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Drugstores
• Online Pharmacies