Gene Therapy for Rare Disease Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2018-2026

 

Gene therapy entails inserting genetic material into patients' cells to provide new functions or restore missing or improper cell function caused by cancer or gene mutations. According to the National Center for Advancing Translational Sciences, while approximately 7,000 rare diseases have been identified, only a few hundred have approved treatments. Furthermore, developing gene therapy for rare diseases is gaining traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause. Small molecule drugs frequently treat symptoms rather than the underlying cause of the disease. To manage the condition, the drugs must be taken on a regular basis. Gene therapy has the potential to correct underlying genetic defects.

The success of preclinical studies has given hope for the future of Gene Therapy for Rare Disease Market, which affects a large proportion of the rare disease population. The ability to correct or replace mutated genes could lead to novel therapy options, and even cures, for rare conditions that previously had few to no treatment options.

In the case of rare diseases, investigators involved in Gene Therapy for Rare Disease Market research are particularly optimistic about the future benefits of gene therapies. A recent cross-sectional global survey assessed the perspectives of authors who had published peer-reviewed articles on rare genetic diseases.

Gene therapy is relevant to rare disease patients and has improved the well-being and personal satisfaction of older children and young adults with X-SCID. These children are expected to have complex clinical issues as a result of receiving life-saving bone marrow transplants to treat their condition.

The global market for Rare Disease Gene Therapy is segmented by type, indication, administration, distribution channel, and region. The market is divided into two types: non-viral vector gene therapy and viral vector gene therapy. The market is divided into indications such as inflammatory diseases, neurological diseases, infectious diseases, hematology diseases, oncology diseases, genetic diseases, and others. In terms of distribution channel segmentation, the market is divided into online pharmacies, drugstores, retail pharmacies, and hospital pharmacies.

The report provides insights into the market, focusing on factors that will boost, repel, challenge, and create opportunities for the market during the forecast period.

Key Players

Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. are among the major players in the global gene therapy for rare diseases market.