Oligo Therapeutics Today - Pharma & CDMO Perspectives on Modality Evolution & Manufacturing Needs

DeciBio conducted 16 interviews with stakeholders at pharmaceutical companies and CDMOs to better understand their perspectives on current trends and key advances in oligonucleotide therapeutic development. Stakeholders gave insights into how oligonucleotide design is progressing, optimization needed for enhanced targeting and performance, and feedback on key considerations when selecting CDMO partners (pharma only). In response to each interview question, DeciBio provides several condensed takeaways and a curated list of quotes indicative of stakeholder feedback.

This report is organized around the responses to 22 key study questions

• How would you describe the evolution of the oligo therapeutic field over the past two years?
• Do you believe there will be a primary therapeutic area or therapeutic areas for oligo-based therapeutics?
• How do you see the therapeutic use of oligos shifting with the rise of broader classes and types of genetic medicine?
• Where do you see the next major breakthroughs coming from in terms of backbone modifications or conjugation strategies?
• Are current chemical modifications sufficient for optimal PK* and biodistribution, or do we need new classes of oligonucleotide chemistries?
• What delivery strategies are showing the most promise for CNS, muscle, or lung-directed oligonucleotide therapies?
• Do you see a future where systemic, non-conjugated oligonucleotides can effectively reach extra-hepatic tissues?
• Do you think ASOs and siRNAs will remain dominant, or will new RNA-based modalities (e.g.,RNA editing, aptamers) displace them?
• What are the biggest gaps in our understanding of the biological mechanisms driving the efficacy of different oligo-based modalities?
• As more oligonucleotide therapies move toward commercialization, what are the biggest bottlenecks in manufacturing?
• What is the role of automation and continuous manufacturing in oligo production — are we on the verge of a step-change in scalability?
• What’s holding back wider adoption of therapeutic nucleotides in common conditions like cardiovascular and metabolic diseases?
• Do you see combination therapies as an untapped opportunity, or do the mechanistic challenges outweigh the potential benefits?
• How would you rate the significance of hurdles to developing oligo-based therapeutics
• What is your familiarity with each of the following therapeutic oligo vendors?
• What are the top 5 most important oligo attributes in regard to selecting external CDMOs?
• What are the most important vendor performance and capability factors in regard to selecting external CDMOs?
• What are the most important vendor attributes when selecting external CDMOs?
• What do you think of new synthesis methods, such as enzymatic-based oligo synthesis(e.g., EnPlusOne, Codexis)?
• What share of your spend was on each development phase in 2024 and what do you expect to spend on each phase in 2025?
• What share of your spend was on GMP-grade oligos in 2024 and what do you expect to spend on GMP-grade oligos in 2025?
• What share of your spend was on each therapy type in 2024 and what do you expect to spend on each therapy type in 2025?

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Companies Discussed

• Agilent
• BOC Sciences
• Brammer
• IDT
• Lonza
• Millipore Sigma
• ThermoFisher
• TriLink
• Wuxi STA
• And more

This advisory board is meant to be a qualitative, limited scope study, and the intent is to highlight the unbiased and unfiltered feedback just from the stakeholders interviewed, not provide a comprehensive market assessment. DeciBio also has a suite of comprehensive market reports if broader industry level insights are of interest. 

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