Antisense oligonucleotides are synthetic strings of nucleic acid, which reduce expression of messenger RNA (m-RNA). The function performed by interference with pre m- RNA splicing are restoration of protein and modification of protein. Antisense oligonucleotide is a new approach to treat several neurodegenerative disorders to prevent disease onset or stop disease development.
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Government support for development and launch of antisense drugs in North America is expected to bolster the market growth
North America holds the dominant position in the global CNS specific antisense oligonucleotide market, owing to strong government support for antisense drugs. For instance, in March 2018, GeneTx Biotherapeutics LLC’s, GTX-101 received the U.S. Food and Drug Administration’s (FDA) orphan-drug designation for GTX-101 for the treatment of Angelman syndrome. It is a rare neurogenetic disorder that affects around one in 15,000 people, according to the FDA statement.
Increasing prevalence of Alzheimer disease in the region is also expected to boost the market growth. For instance, in August 2012, National Center for Biotechnology Information (NCBI) stated Alzheimer disease as the most frequent cause of dementia in Western societies and estimated around 5.5 million cases in the U.S. and the prevalence worldwide is estimated to be as high as 24 million.
Moreover, rich product pipeline for treatment of neurodegenerative disorders is expected to drive the market growth. For instance, in October 2017, Ionis Pharmaceuticals, Inc., initiated a Phase 1/2 clinical study of IONIS-MAPTRx in patients with mild Alzheimer’s disease. Biogen funded US$ 10 million milestone payment to Ionis Pharmaceuticals, Inc.
Moreover, key players in the players are focused on receiving the U.S. Food and Drug Administration (FDA) approval for novel products to reduce the burden of CNS associated disorders. For instance, in September 2016, Sarepta Therapeutics received the U.S. Food and Drug Administration (FDA) approval for Exondys 51(eteplirsen) used for treatment of rare genetic disorder Duchenne muscular dystrophy.
Major players operating in the global CNS specific antisense oligonucleotide market include Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., and Q-STATE BIOSCIENCES, INC.