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www.biopharminternational.com March 2021 eBook BioPharm International 9 control, the company is able to iso- late single isomers rather than the stereorandom, mixture-based oligo- nucleotides being advanced by oth- ers. "For this reason, we have the potential to fully characterize and investigate the structure activity relationship of all RNA therapeu- tics in our pipeline, as has become standard with small molecule and antibody development. This allows us to develop precise and rationally designed medicines that leverage the full potential of RNA therapeutics," Panagoulias says. Successful commercialization can also depend on identifying indica- tions that maximize the strengths of a drug class, Templin remarks. Oligos encompass a diverse range of molecules, from single stranded ASOs to double stranded siRNAs, and can function through varying inter- actions with their target; as such, development programs can be, and are being, focused on diseases such as cancer and chronic inflamma- tion, which continue to be leading causes of morbidity and mortality. However, these indications are also favored targets for small molecules and biologics, and clinical and commercial competition is intense, Templin emphasizes. This intense competition is likely one reason why there has been a determined effort to identify con- ditions that have been difficult to address with other drug classes, Templin continues. "Most notable, diseases in which there is a clear link between a specific genetic vari- ant and a distinct consequence can be optimal for a drug that is based on Watson-Crick base pairing," he says. Templin points to the recent example of Spinraza (nusinersen), an ASO approved by FDA in 2016 (5) for treating spinal muscular atrophy, a rare disease. "While providing a much-needed treatment option for a devastating disease, this also helped to change the view of how drug development could be focused on a specific genetic variant and/or biasing how genetic code is trans- lated," he says. "When thinking from a gene-up perspective, this pro- vides a means to treat individuals at a root cause as well as opening new treatment options for Alzheimer's, Park inson's, Hunting ton's, and many other neurologic diseases," Templin adds. Wave Life Sciences, meanwhile, tackles the challenge of ASO drug development with the help of its pro- prietary discovery and drug devel- opment platform, PRISM, which combines a unique ability to con- struct stereopure oligonucleotides with a deep understanding of how the interplay among oligonucleotide sequence, chemistry, and backbone stereochemistry impacts key phar- Regulatory Sourcebook Quality Connect with BIOPHARMINTERNATIONAL.COM Twitter.com/BioPharmIntl LinkedIn.com/company/ BioPharm-International Sign up for our eNewsletters Get the latest news, research, and trends in the biopharmaceutical industry delivered straight to your inbox by signing up. BP_Social_HouseAds_101420.indd 2 BP_Social_HouseAds_101420.indd 2 10/14/20 2:57 PM 10/14/20 2:57 PM