Antisense oligonucleotides are single-stranded oligodeoxynucleotides that can alter the RNA and modify protein expression. These oligonucleotides were discovered to influence RNA processing.
CNS Specific Antisense Oligonucleotides: Introduction
- Antisense oligonucleotides are single-stranded oligodeoxynucleotides that can alter the RNA and modify protein expression. These oligonucleotides were discovered to influence RNA processing.
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- Subsequent chemical modifications of antisense oligonucleotides were used to address issues such as off-target toxic effects and insufficient biological activity. These modifications led to an improved mechanism of antisense oligonucleotides and clinical trial design. This paved the way toward translating their use into therapies for various genetic and neurological conditions.
- Antisense oligonucleotides mediated therapies target the source of pathogenesis and have higher chance of success as compared to therapies that target downstream pathways. These are drugs for the treatment of central nervous system (CNS) disorders, as they have specific molecular targeting and extended pharmacological properties.
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Key Drivers and Restraints of Global CNS Specific Antisense Oligonucleotides Market
- The global CNS specific antisense oligonucleotides market is primarily driven by advances in the understanding of pharmacology of antisense oligonucleotides, rapid development of next generation antisense oligonucleotide therapies, and an increase in the prevalence of neurological and genetic disorders
- According to an article published in Orphanet Journal of Rare Diseases, estimated incidence of spinal muscular atrophy is 1 in 6,000 to 1 in 10,000 live births
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Spinal muscular atrophy segment to account for major share of global CNS specific antisense oligonucleotides market
- In terms of indication, the global CNS specific antisense oligonucleotides market can be classified into hereditary transthyretin amyloidosis (hATTR)/ polyneuropathy, spinal muscular atrophy, and Huntington’s disease
- The spinal muscular atrophy segment is anticipated to dominate the global market during the forecast period, due to availability of Food and Drug Administration (FDA) approved antisense oligonucleotides therapy for spinal muscular atrophy and a rise in focus on the development of treatment of rare genetic conditions
- In December 2016, the FDA approved Spinraza, the first drug indicated for the treatment of spinal muscular atrophy in adult and pediatric patients. It is an antisense oligonucleotide utilized to treat spinal muscular atrophy caused by mutation of in chromosome 5q.
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Hospital pharmacies institutes segment to account for major share of global market
- Based on distribution channel, the global CNS specific antisense oligonucleotides market can be categorized into hospital pharmacies, retail pharmacies, and online pharmacies
- The hospital pharmacies segment is expected to account for a major share of the global CNS specific antisense oligonucleotides market by 2030, owing to an increase in number of patients with genetic disorders and a rise in awareness about the need for ensuring availability of approved therapies for these patients.
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North America to dominate global CNS specific antisense oligonucleotides market
- In terms of region, the global CNS specific antisense oligonucleotides market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is projected to dominate the global CNS specific antisense oligonucleotides market during the forecast period.
- The market in North America is anticipated to be driven by an exponential increase in the number of pharmaceutical companies conducting clinical trials for the development of promising drugs, recent FDA approvals & product launch, and a rise in awareness about treatment of neurological conditions. GeneTx Biotherapeutics LLC received orphan-drug designation for GTX-101, indicated for the treatment of Angelman syndrome.
Key players operating in global CNS specific antisense oligonucleotides market
The global CNS specific antisense oligonucleotides market is highly fragmented, with a large number of domestic players accounting for a major share of the market.
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