Antisense oligonucleotides (ASOs) are 15–25 nt oligonucleotides designed to tie complementary RNA targets for the degradation. They are synthesized so that they can be used to block disease processes by altering the synthesis of a specific protein. ASOs offers promising treatment options for a range of medical disorders. They facilitate the development of therapeutics affecting protein targets which cannot be treated with protein therapeutics. They can target a particular malfunctioning gene, interfere with RNA function at cellular level, and can also be silenced or modulated. It allows modifications in immune system that facilitates treatment of a wide range of autoimmune disorders – that cannot be treated with available drugs. It is easy to scale up the commercial scale GMP production of these oligonucleotides compared to other biologic therapies. Moreover, the side effects of antisense oligonucleotides are minimal and can be controlled with ease as compared to other class of drugs.
Though, the antisense oligonucleotides market has witnessed four FDA approvals [fomivirsen (Vitravene) approved in 1998 – which was discontinued due to low demand; pegaptanib (Macugen) in 2004; mipomersen (Kynamro) in 2013; and eteplirsen (Exondys 51) in 2016). However, this market is yet to achieve recognition as a valuable drug category like biologics, small molecules, and gene and cell therapy. However, in December 2016, this was projected to change with the approval of nusinersen (Spinraza) for treating spinal muscular atrophy (SMA), which is one of the most common genetic cause for infant mortality with no approved treatments. Spinraza has shown unprecedented results for the treatment of SMA in clinical trials.
Antisense Oligonucleotides Market: Drivers and Restraints
Strong pipeline of antisense oligonucleotides and increased focused on large number of product approvals and commercialization are some of the primary factors driving the growth of the global antisense oligonucleotides market. Moreover, significant advances in the field of antisense oligonucleotides at a therapeutic or clinical level is further contributing to the growth of the global antisense oligonucleotides market. However, the global antisense oligonucleotides market is grappling with numerous challenges related to diversity of oligonucleotides, delivery, and regulatory complexity. Despite several advancements at clinical levels, the delivering of active oligonucleotide to the actual site within target cells is still one of the major challenges hindering the growth of the global antisense oligonucleotides market. In addition to this, developing a new set of unique regulatory guidelines is another challenge limiting the growth of the global antisense oligonucleotides market.
Antisense Oligonucleotides Market: Segmentation
Tentatively, the global antisense oligonucleotides market has been segmented on the basis of drug, indication, end user and geography.
Based on drug, the global antisense oligonucleotides market is segmented as below:
- Approved Drugs
- Pipeline Analysis
Based on indication, the global antisense oligonucleotides market is segmented as below:
- Ocular Diseases
- Amyotrophic Lateral Sclerosis (ALS)
- Duchenne Muscular Dystrophy
- Spinal Muscular Atrophy
Based on application, the global antisense oligonucleotides market is segmented as below:
- Basic Research
- Target Validation
- Drug Discovery
Antisense Oligonucleotides Market: Overview
Although the development of oligonucleotide therapeutics commenced in 1970s, FDA has approved only four oligonucleotide drugs (of which fomivirsen was discontinued) nearly a half century later. However, the filed is gaining momentum and is set to offer promising growth opportunities with a strong pipeline of 135 oligonucleotides in various stages of clinical trials. Even when the market performance of antisense oligonucleotides is not predictable as like other segments of the industry, the future looks very promising primarily reflected by the leadings players in the market and the amount of research and development investments.
Antisense Oligonucleotides Market: Regional Outlook
Geographically, the global antisense oligonucleotides market is segmented into regions viz. North America, Latin America, Western Europe, Eastern Europe, Asia-Pacific excluding Japan, Japan, Middle East and Africa. North America clearly dominates the global antisense oligonucleotides market due to a strong product pipeline and increasing number of FDA approvals in the region.
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Antisense Oligonucleotides Market: Key Players
Some of the players identified in the global antisense oligonucleotides market include Ionis Pharmaceuticals, Inc., Sarepta Therapeutics, Biogen, Alnylam Pharmaceuticals, Inc., Antisense Therapeutics Limited, Isarna Therapeutics GmbH, Arrowhead Pharmaceuticals, Inc., Atlantic Pharmaceuticals, Inc., Enzon Pharmaceuticals, Inc., Bio-Path Holdings, Inc., Gene Signal International SA, GlaxoSmithKline plc, Geron Corporation, Gradalis, ICO Therapeutics, Aptose Biosciences, Marina Biotech, miRagen Therapeutics, Inc., Synlogic, Inc., OncoGenex Pharmaceuticals Inc., Pharmaxis Ltd, Regulus Therapeutics Inc., Rexahn Pharmaceuticals, Inc., and RXi Pharmaceuticals, among others.