GENE THERAPY FOR RARE DISEASE MARKET ANALYSIS

Gene Therapy For Rare Disease MarketSize and Trends

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).

Increasing drug approvals and launches of novel gene therapies for treatment of various rare diseases are expected to boost the gene therapy for rare disease market growth over the forecast period

Ongoing research and development in gene therapies for rare diseases is expected to boost growth of the global gene therapy for rare disease market

Presence of multiple novel gene therapies for various rare disease treatment in pipeline, which are expected to gain approval is expected to boost the market growth over the forecast period. For instance, Spark Therapeutics, Inc. is developing gene therapy namely Fidanacogene Elaparvovec, a novel, investigational gene therapy indicated for the treatment of patients suffering from hemophilia B. Currently, the therapy is under Phase 3 clinical trial and is expected to receive approval in near future. In August 2018, uniQure N.V. treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In May 2018, Orchard Therapeutics Plc. received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate OTL-200, indicated for treatment of patients with metachromatic leukodystrophy (MLD).  PTC Therapeutics, In. has GT-AADC in phase 3 clinical trials and is indicated for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.

 Gene Therapy for Rare Disease Market - Insights

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).