Taking New Steps to Meet the Challenges of Gene Therapy for Rare Disease Market With Major Key Players Kite Pharma, Novartis International AG, Juno Therapeutics

The global gene therapy for rare disease market size was valued at US$ 16.3 million in 2017, and is expected to witness a robust CAGR of 35.0% over the forecast period (2018–2026).

Key players in the market are focused on research and development of gene therapies for rare diseases in order to introduce their pipeline products in the market. For instance, in October 2018, bluebird bio, Inc. received European Medicines Agency (EMA) marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype. In August 2018, Kite Pharma, Inc., a Gilead Company, received European Commission (EC) Marketing approval for its novel Yescarta (axicabtagene ciloleucel), as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. Moreover, in September 2018, Spark Therapeutics Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of LUXTURNA (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss

Increasing research and development by leading players in gene therapy for rare disease is expected to support growth of the gene therapy for rare disease market. Several companies have novel gene therapies in the clinical stages for treatment of rare diseases ranging from rare cancers to rare genetic diseases. For instance, Spark Therapeutics, Inc. has SPK-7001 (Choroideremia/ Phase ½), SPK-8011 (Hemophilia A/Phase ½), and SPK-3006 (Pompe Disease/preclinical) in the pipeline. Moreover, Orchard Therapeutics has multiple therapies in the pipeline such as OTL-101, OTL-103, OTL-102, OTL-200, and OTL-201 for various indications. Some of these therapies are expected to receive the U.S. Food & Drug Administration approvals during the forecast period. These factors are expected to boost the global gene therapy for rare disease market growth over the forecast period.

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The global gene therapy for rare disease market is expected to exhibit a CAGR of 35.0% over the forecast period, owing to increasing research and development by key players in gene therapy.

Price for therapies are highly dependent on prevalence of the disease and therefore, some drugs (which are in development for ultra-rare disease) may be expensive

There could be a significant delay in commercialization of approved therapies in regions such as Latin America, Asia Pacific, Middle East, and Africa, as these therapies have novel technologies (as patient cells may be needed for modification and reintroduction into the patient) and high prices of gene therapy treatment.

Asia Pacific is expected to witness highest growth in gene therapy for rare disease market over the forecast period

Major players operating in the global gene therapy for rare disease market include 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.

Gene therapy involves insertion of genetic material into cells of the patients to provide new function or to restore missing or inappropriate functioning of the cells due to cancer or gene mutations. According to National Center for Advancing Translational Sciences, although around 7,000 rare diseases are identified, only a few hundred have approved treatments. Moreover, developing gene therapy for rare diseases is gaining significant traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause. Small molecule drugs often treat symptoms only rather than curing underlying cause of the disease. This requires frequent administration of the drugs to manage the condition. Gene therapy holds the potential to correct underlying genetic defects. Moreover, a clinically sound and successful gene therapy may only require a single dose to cure a disease rather than requiring a lifetime treatment. Commercially approved gene therapies include Kymriah (Novartis AG), Yescarta (Gilead Sciences, Inc.), Luxturna (Spark Therapeutics), and Strimvelis (Orchard Therapeutics).

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