The global gene therapy for rare disease market By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), Strimvelis), Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others), By Therapeutic Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), and Region (North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa) was valued at US$ 16.3 million in 2017, and is projected to exhibit a CAGR of 35.0% over the forecast period (2018–2026), as highlighted in a new report published by Coherent Market Insights. Robust pipeline, recent success of newly launched gene therapies such as Kymriah, and Yescarta, and absence of robust therapies for several rare diseases is expected to boost growth of the global gene therapy for rare disease market.
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
Browse 26 Market Data Tables and 37 Figures spread through 240 Pages and in-depth TOC on Global Gene Therapy for Rare Disease Market By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), Strimvelis), Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others), By Therapeutic Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), and Region (North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa) - Global Forecast to 2026
To know the latest trends and insights prevalent in the global gene therapy for rare disease market, click the link below:
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.
Key Takeaways of the Global Gene Therapy for Rare Disease Market:
About Coherent Market Insights:
Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.
Coherent Market Insights
1001 4th Ave,
Seattle, WA 98154
Email: [email protected]
Finding it difficult to find the research that would cater to your business demands? Give us a chance to help you. One of our Research Consultants will connect to provide a customized solution.
Coherent Market Insights
1001 4th Ave, #3200 Seattle, WA 98154
Talk to our research consultant to design an exclusive report as per your research needs.
We aim to fulfil client's research demands with tailored research solutions.
We aim to provide research studies in quickest turnaround time and in a much cost effective manner.
We cover each industry from supply and demand side with an aim to provide a most holistic research study.
We strive to provide most accurate and reliable research findings in our research reports.