Antisense Oligonucleotide Therapeutics Market by Target Indication, Type of Therapy, Type of Molecule Type of Generation and Key Geographies: Industry Trends and Global Forecasts, 2020-2030

Overview

Since the approval of Vitravene™ (for the treatment of cytomegalovirus retinitis) in 1998, antisense oligonucleotide (ASO) therapies have evolved into a prominent class of therapeutics. In addition to the seven drugs, based on such molecules, that are commercially available, around 160 candidates are under development. Examples of recently approved antisense therapeutics include (in reverse chronological order) Viltepso™ (duchenne muscular dystrophy, March 2020), Vyondys 53® (duchenne muscular dystrophy, December 2019) and Waylivra® (hereditary transthyretin-mediated (hATTR) amyloidosis, May 2019). Given their ability to target the root cause of diseases, at the protein expression level, these disease-modifying interventions have potential applications across a wide range of therapeutic areas (including but not limited to oncological disorders, neurodegenerative disorders, respiratory disorders, and even certain rare genetic disorders). Further, recent advances in antisense oligonucleotide chemistry have enabled the development and synthesis of specialized oligonucleotides, having improved safety profiles and better cell targeting capabilities. In fact, these advanced variants of antisense oligonucleotide-based therapeutic candidates are deemed to possess the potential to cater to the unmet need for effective treatment options for diseases that were previously considered difficult to treat.

Presently, several drug developers, along with ASO technology providers, are actively engaged in the development of various antisense therapy candidates. Innovation in this field of research is mostly focused on improving cell targeting ability, target affinity, nuclease resistance, and optimizing toxicity profiles of antisense oligonucleotide candidates. Several big pharma players have also demonstrated interest in antisense oligonucleotide therapeutics and are investing both time and capital in this domain. The market has witnessed substantial partnership activity over the last few years. The activity in this segment of the industry has also attracted the attention of both private and public sector investors/investment funds, which have extended financial support to the initiatives of capable developer companies. Driven by encouraging clinical trial results, the antisense oligonucleotide therapies market is poised to witness healthy growth as multiple late stage drug candidates are approved and marketed over the coming decade.

Scope of the Report

The “Antisense Oligonucleotide Market, 2020-2030” report features an extensive study on the current market landscape, offering an informed opinion on the likely adoption of these therapies over the next ten years. The study underlines an in-depth analysis, highlighting the diverse capabilities of stakeholders engaged in this domain. 

In addition to other elements, the study includes:

• A detailed assessment of the current market landscape of antisense oligonucleotide therapeutics, providing information on type of antisense molecule (RNA molecule and DNA molecule), ASO generation (first-generation, second-generation, third-generation and next-generation), phase of development (commercial, clinical, preclinical, and discovery stage) of lead candidates, target genes, target disease indications, target therapeutic areas, route of administration (subcutaneous, intravenous, intrathecal, intravitreal and others) and type of therapy (monotherapy, combination therapy and both). In addition, it provides details on drug developer(s), including year of establishment, company size, and location of headquarters.
• Tabulated profiles of prominent antisense oligonucleotide therapeutic developers. Each profile features a brief overview of the company, its financial information (if available), product portfolio, recent developments and an informed future outlook.
• An in-depth analysis of completed, ongoing and planned clinical studies of various antisense oligonucleotide therapeutics, based on the various relevant parameters, such as trial registration year, trial phase, trial recruitment status, enrolled patient population, study design, leading industry sponsors/collaborators (in terms of number of trials conducted), trial focus, target therapeutic area and target genes, highlighting popular indications, popular interventions and regional distribution of trials.
• A detailed analysis of grants that have been awarded to research institutes for antisense oligonucleotide therapeutic projects, in the period between 2017 and 2020 (till September), on the basis of important parameters, such as year of grant award, amount awarded, administering institute center, support period, type of grant application, purpose of grant award, activity code, study section involved, type of recipient organizations and focus area. In addition, it highlights geographical distribution of recipient organizations, popular therapeutic areas, popular funding institute centers, prominent program officers, and popular recipient organizations. 
• An analysis of the partnerships that have been established in the recent past (2016-2020), covering acquisitions and mergers, licensing agreements, product development agreements, research agreements, joint venture agreements and other relevant types of deals.
• A case study on the oligonucleotide contract manufacturers and purification service providers, providing information on the year of establishment, company size, scale of operation, location of headquarters and type of purification method used.

One of the key objectives of the report was to estimate the existing market size and the future opportunity for antisense oligonucleotide therapeutics, over the next ten years. Based on multiple parameters, such as target patient population, likely adoption rate and the annual treatment cost, we have provided informed estimates on the evolution of antisense oligonucleotide therapeutics market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] type of antisense molecule (RNA and DNA molecule) [B] different target indications (duchenne muscular dystrophy, spinal muscular atrophy, hereditary transthyretin-mediated (hATTR) amyloidosis, familial chylomicronemia syndrome, familial partial lipodystrophy, pouchitis, leber's congenital amaurosis, huntington’s disease and amyotrophic lateral sclerosis) [C] ASO generations (first-generation, second-generation and third-generation) [D] route of administration (intrathecal, intravenous, intravitreal, subcutaneous, and topical) [E] type of therapy (combination therapy and monotherapy) and [F] key geographical regions (US, UK, EU4, Asia-Pacific and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth. 

All actual figures have been sourced and analyzed from publicly available information forums and inputs from primary research. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Questions Answered

• Who are the leading industry and non-industry players in this market?
• What are the key therapeutic areas for which antisense oligonucleotide therapeutics are being/have been developed?
• What are the prevalent trends within competitive landscape of antisense oligonucleotide therapeutics?
• Which geographies are the most active in conducting clinical trials on antisense oligonucleotide therapeutics?
• What kind of partnership models are commonly adopted by industry stakeholders? 
• How is the current and future market opportunity likely to be distributed across key market segments?