ANTISENSE OLIGONUCLEOTIDES MARKET: OVERVIEW
As per Roots Analysis, the global antisense oligonucleotides market valued at USD 2.5 billion in the current year is anticipated to grow at a lucrative CAGR of 15% during the forecast period.
The market sizing and opportunity analysis has been segmented across the following parameters:
Type of Antisense Molecule
- DNA Molecules
- RNA Molecules
Type of ASO Generation
- First-Generation Products
- Second-Generation Products
- Third-Generation Products
Target Disease Indication
- Amyotrophic Lateral Sclerosis
- Duchenne Muscular Dystrophy
- Familial Chylomicronemia Syndrome
- Familial Partial Lipodystrophy
- Hereditary Transthyretin-Mediated (hATTR) Amyloidosis
- Huntington's Disease
- Leber's Congenital Amaurosis
- Spinal Muscular Atrophy
Route of Administration
- Intrathecal Therapies
- Intravenous Therapies
- Intravitreal Therapies
- Subcutaneous Therapies
- Intraorifice Therapies
Type of Therapy
- Combination Therapies
- Monotherapies
Geography
- North America
- Europe
- Asia-Pacific
- Rest of the World
ANTISENSE OLIGONUCLEOTIDES MARKET: GROWTH AND TRENDS
Oligonucleotides are short single stranded DNA or RNA molecules, that comprise 15-20 nucleotide residues. In modern biopharmaceuticals, the applications of these oligonucleotides are vast, including (but not limited to) genetic testing, fundamental biomolecular research, and forensic analysis. Antisense oligonucleotides, a diverse class of oligonucleotides are short, single-stranded RNA / DNA molecules specifically binding to the target mRNA and have the ability to modify protein expression through a variety of mechanisms.

Antisense therapeutics are considered to be one of the most promising agents for impairing protein production and blocking the function of the specific target gene of interest in the human genome. Presently, this mechanism forms the basis for many therapeutics being investigated in different stages of clinical trials for treatment of a variety of disorders, including oncological disorders, genetic diseases, hepatic diseases, respiratory disorders and infectious diseases. In fact, in the recent past, the oligonucleotide drug developers had also investigated the relevance of these interventions against the Coronavirus (COVID-19). Given the pace of innovation and developments in the antisense oligonucleotides market, we can expect antisense oligonucleotides to become a major therapeutic modality in the foreseen future.
ANTISENSE OLIGONUCLEOTIDES MARKET: KEY INSIGHTS
The report delves into the current state of the antisense oligonucleotides market and identifies potential growth opportunities within industry. Some key findings from the report include:
- Around 30 players from are presently engaged in evaluating the potential therapeutic benefits of antisense oligonucleotides for the treatment of a wide range of disease indications, worldwide.
- The pipeline features 170+ candidate therapies in different stages of development, being evaluated either as monotherapies or in combination with other interventions; most of these products are administered parenterally.
- Majority of the approved therapies and late-stage candidates are intended for the treatment of genetic disorders, neurological disorders and oncological disorders.
- Given the advantages of antisense oligonucleotides, these interventions are primarily evaluated as monotherapy. Late-stage drugs being investigated as monotherapy include Tofersen and Pelacarsen.
- Over 18,000 patients have been enrolled in clinical trial sites evaluating close to 70 antisense oligonucleotide-based therapy candidates.

- Majority of the antisense oligonucleotide therapeutics are designed for subcutaneous administration; these can be self-administered by the patients using different drug delivery systems.
- Several organizations have extended financial support to aid research efforts in this domain; currently, the focus, in terms of funds disbursed, is primarily in support of investigations of drugs for treating neurological conditions.
- The number of grants awarded to stakeholders in this domain (in the US) has continuously increased in the past few years; more than 70% of the total amount was awarded for research projects.
- The field has witnessed the involvement of various administering institutes of the NIH; of all the institutes, participation of the NINDS, NHLBI, and NCI has been relatively more prominent.
- The rising interest in this field is reflected in the number of partnerships inked by the various stakeholders across different application areas.
- Given that nearly 20 molecules are in the late stages of development, companies have primarily collaborated for product development and commercialization purposes.
- Both established players and the new entrants have forged strategic partnerships in the recent past; these deals have primarily been inked for genetic and neurological disorders.
- The future opportunity, in terms of revenues from the sales of marketed and late-stage therapies, is anticipated to be well distributed across different disease areas, types of molecules and key geographical regions.

- The market is likely to witness steady growth over the coming decade; the opportunity will be dispersed across different generations, routes of administration and various types of therapies.
Example Players in the Antisense Oligonucleotides Market
- Antisense Therapeutics
- Biogen
- Bio-Path Holdings
- Ionis Pharmaceuticals
- ProQR Therapeutics
- Sarepta Therapeutics
- Sterna Biologicals
- Wave Life Sciences
ANTISENSE OLIGONUCLEOTIDES MARKET: RESEARCH COVERAGE
- Market Sizing and Opportunity Analysis: The report features an in-depth analysis of the global antisense oligonucleotides market, focusing on key market segments, including [A] type of antisense molecule, [B] type of ASO generation, [C] target disease indication, [D] route of administration, [E] type of therapy and [F] geography.
- Market Landscape: A comprehensive evaluation of antisense oligonucleotide therapeutics, based on several relevant parameters, such as [A] type of antisense molecule, [B] ASO generation, [C] phase of development of lead candidates, [D] target genes, [E] target disease indications, [F] target therapeutic areas, [G] route of administration and [H] type of therapy. Additionally, a comprehensive evaluation of drug developers, based on several relevant parameters, such as [A] year of establishment, [B] company size and [C] location of headquarters.
- Company Profiles: In-depth profiles of antisense oligonucleotide therapeutic developers, focusing on [A] overview of the company, [B] financial information (if available), [C] product portfolio and [D] recent developments and an informed future outlook.
- Clinical Trial Analysis: An insightful analysis of clinical trials related to antisense oligonucleotide therapeutics, based on several parameters, such as [A] trial registration year, [B] trial phase, [C] trial recruitment status, [D] enrolled patient population, [E] study design, leading industry sponsors / collaborators (in terms of number of trials conducted), [F] trial focus, [G] target therapeutic area and [H] target genes.
- Grants Analysis: A comprehensive assessment of grants that have been awarded to research institutes for antisense oligonucleotide therapeutic projects, based on various relevant parameters, such as [A] year of grant award, [B] amount awarded, [C] administering institute center, [D] support period, [E] type of grant application, [F] purpose of grant award, [G] activity code, [H] study section involved, [I] type of recipient organizations and [J] focus area. Additionally, a comprehensive assessment of grants focusing on, [A] geographical distribution of recipient organizations, [B] popular therapeutic areas, [C] popular funding institute centers, [D] prominent program officers and [E] popular recipient organizations.
- Partnerships and Collaborations: An insightful analysis of the deals inked by stakeholders in the antisense oligonucleotide market, based on several parameters, such as [A] year of partnership, [B] type of partnership, [C] most active players (in terms of number of partnerships signed) and [D] regional analysis.
- Case Study: A detailed discussion on the oligonucleotide CMOs and purification service providers, highlighting information on the [A] year of establishment, [B] company size, [C] scale of operation, [D] location of headquarters and [E] type of purification method used.
KEY QUESTIONS ANSWERED IN THIS REPORT
- How many companies are currently engaged in this market?
- Which are the leading companies in this market?
- What factors are likely to influence the evolution of this market?
- What is the current and future market size?
- What is the CAGR of this market?
- How is the current and future market opportunity likely to be distributed across key market segments?
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- The report provides a comprehensive market analysis, offering detailed revenue projections of the overall market and its specific sub-segments. This information is valuable to both established market leaders and emerging entrants.
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