Sign in
Welcome! Log into your account
Forgot your password? Get helpPrivacy PolicyPassword recovery
Recover your password
A password will be e-mailed to you.
GEN – Genetic Engineering and Biotechnology NewsHomeTopicsBioprocessingOligonucleotide Therapeutic Industry Readies Itself to Move to the Next Phase
Credit: Seksan Mongkhonkhamsao
Synthetic oligonucleotide therapeutics are now validated for treating a wider range of diseases and, as companies move to larger-scale development, they should become familiar with the Chemistry, Manufacturing, and Control (CMC) aspects of their workflow.
That’s the view of Bao Zhang Cai, PhD, vice president of oligonucleotide CMC at GondolaBio. Cai will be giving a talk about CMC strategies for oligonucleotide therapies at the upcoming Bioprocessing Summit in Boston.
“The trend is very clear. Oligonucleotide therapies are becoming increasingly popular, as they tackle the root causes of diseases, not just the symptoms, and I can confidently say they’re now a validated therapeutic modality,” he says.
“But to achieve their potential, you need the right development strategy at the right time—you don’t want to apply late commercial staging to early development.”
According to Cai, synthetic oligonucleotide therapeutics have seen significant development over the last decade. They started as therapies for rare or ultra-rare diseases, but their applications have now widened.
With more patients needing treatment, a growing number of drugs are reaching the later phases of clinical trials and, as a result, CMC is increasingly important.
Cai suggests that product purification is important to consider, as it is often [genetic] sequence-dependent.
“You need to think about it upfront or as early as possible,” he says. “This is why, in general, it helps to identify and take advantage of an existing platform.”
An oligo-specific challenge, he explains, is that these products consist of a hydrophobic element conjugated to a water-soluble section.
“These two extremes put together generate unique challenges during purification, and you need to find the best route—do you want to perform the conjugation in solution or on a solid support?” he explains.
Finally, he says, as synthetic oligonucleotides are a relatively new product type, the critical quality attributes and product specifications can typically start quite broad. A focus on CMC can help manufacturers collect the data to narrow these specifications over time.
“Better process understanding can mean that, in the early phase, you have a poorly resolved method, and you get, say, 90% purity. But, later, you have a better method, and the purity will drop, and that’s a tough case to explain to the regulator,” he says.
“But, if you have process knowledge and understanding, you can demonstrate the product quality remains the same, and the purity level only looks lower because your methods have improved.”
InsightsBioprocessingGene therapy (Therapeutics)Synthetic biotechnologyGondolaBioAlso of Interest
Transcenta Therapeutics and WuXi Biologics Team Up to Accelerate Commercialization of HiCB TechnologySmarter Cell Culture Starts with Better MediaCAR T Manufacturing in Japan Gets Boost from Teijin-Shinshu University Research CollaborationHarnessing Real-Time DataGene Therapy Extends Lifespan and Reduces Symptoms in Sandhoff Disease Cat ModelMicroscale Chromatography Moves Toward Manufacturing ReadinessRelated Media
In Conversation with Visionary Geneticist George ChurchBoston's Bioprocessing Buzz:GENReports Live from BPI 2025Smart Vector Design for Improved Cell and Gene TherapiesBiotech and Beignets: The News from Day One of ASGCTPrecision Base Editing Meets Single-Cell Multiomics to Advance Cell and Gene TherapiesBioprocessing in Boston: GEN Reports Live from BPI 2024Top 5ResourcesRecommended For YouPodcast
Touching Base
Touching Base is the dynamic podcast series from the editors ofGEN. Each episode features a rotating case of senior editors—including John Sterling, Kevin Davies, Julianna LeMieux, Alex Phillippidis, Uduak Thomas, Corinna Singleman, and Fay Lin—who delve into emerging stories, exchange ideas, and debate the latest trends in biotech. Additionally, they talk to some of the leading voices in the industry about what's now and next.Start listening today!
Stay up to date with the lasted episodes of Touching Base bysubscribing to theGENPodcast Newsletter
Copyright © 2026 Sage Publications or its affiliates, licensors, or contributors. All rights reserved, including those for text and data mining and training of large language models, artificial intelligence technologies, or similar technologies.
MORE STORIES
