ANTISENSE OLIGONUCLEOTIDE THERAPEUTICS Knowpia

Given the multifaceted advantages of antisense oligonucleotide therapeutics, the field has witnessed an increase in the number of players involved in the development of such solutions. The domain features the presence of over 30 companies which are involved in the development of over 170 antisense oligonucleotide drugs.

Majority of the developers (55%) of antisense oligonucleotide therapeutics are start-ups or small-sized (less than 50 employees), followed by 29% large (501-5000 employees) and 16% mid-sized companies (51-500 employees). Notable examples of the start-ups in the antisense oligonucleotide domain include (in alphabetical order) Atlantic Healthcare, Dynacure, Isarna Therapeutics, and Sterna Biologicals.

Majority of the antisense oligonucleotide therapeutics (81%) are single stranded DNA, which are complementary to the mRNA target. Examples of antisense DNA molecules include (in alphabetical order) AKCEA-APOCIII-LRx (Ionis Pharmaceuticals), Casimersen (Sarepta Therapeutics) and Tominersen (Hoffman-La Roche). Further, nearly 20% of the antisense oligonucleotides are RNA molecules. Moreover, nearly half of the antisense oligonucleotide therapeutics are second-generation molecules. These therapies have been developed to overcome the challenges of the first-generation therapeutics, such as poor binding affinity and adverse side effects.

Additionally, it is worth highlighting that seven molecules (capturing 4% of the share), namely, Spinraza (by Ionis Pharmaceuticals), Vyondys 53 (Sarepta Therapeutics), Tegsedi (Ionis Pharmaceuticals), Waylivra (Ionis Pharmaceuticals), Viltepso (Nippon Shinyaku) and Exondys 51 (Sarepta Therapeutics) have been commercialized till date and over 60% molecules are currently in advanced stages of development (phase II and above). In addition, majority of the therapies (28) are designed for administration through subcutaneous route. The subcutaneous route of administration facilitates rapid onset of the therapeutic action that can be extended to a longer duration. Examples of late stage molecules that are being administered subcutaneously include (in alphabetical order) ATL1102 (Antisense Therapeutics), IONIS-FB-LRx (Ionis Pharmaceuticals) and Pelacarsen (Novartis). Further, the second most preferred route of administration is intravenous route. This route of delivery gives reproducible effects and the administered dose directly reaches systemic circulation.

For more information, please click on the following:

https://www.rootsanalysis.com/reports/antisense-oligonucleotide-therapeutics-market.html