MANUFACTURING ARTICLES

  • The RNA Ties That Bind: 3 Takeaways From The OPT Congress

    Here, I unpack three takeaways I had from the OPT Congress, each of which I’d argue are indicative of the interconnectedness across the genetic medicines field.

  • Rethinking ATMP Assurance When Sterility Timelines, Reality Misalign

    The compendial 14-day sterility testing window exposes product and patient to numerous risks, but settling on a rapid alternative also presents its own set of challenges.

  • The Potency Chains Of Events For mRNA/LNP Therapeutics

    Potency in mRNA/LNP therapies depends on two chains: manufacturing/cold chain integrity and complex in vivo delivery. Breakdowns at any step — from stability to translation — can limit efficacy and safety.

  • How Informal FDA Feedback Is Quietly Driving RNA Capital Allocation

    Subtle FDA signals — especially in pre-IND talks — are quietly shaping RNA investment. Early regulatory feedback on CMC and comparability now drives how investors assess risk and allocate capital.

  • Inside Alnylam's Playbook For High Volume siRNA Production

    As one of the first companies to establish a chemoenzymatic ligation platform, Alnylam's Maines and Nechev are in the perfect position to espouse wisdom on the practical aspects and remaining challenges of implementing enzymatic ligation at a large scale. Here in this installment, Maines and Nechev outline what they’ve learned thus far about the science of enzymatic ligation, while also paying credence to the unknowns and existing barriers that, as a frontrunner, Alnylam will inevitably (but willingly) be tasked with facing in the future.

  • The "Middle Ground" Advantage: Why Alnylam Is Betting Big On Chemoenzymatic Ligation

    Though conversations are picking up around this hybrid approach, we aren’t exactly known as an industry that embraces risk no-holds barred. While there are a handful of manufacturers and CDMOs who are embracing the hybrid model today, there are many more that are likely to hold out for the next-next gen approach: Fully enzymatic oligo production. However, as Alynlam’s CTO and Chief Quality Officer Tim Maines argued, embarking into the “middle ground” of enzymatic ligation is essential for garnering the step-by-step learnings needed to help us unlock even bigger manufacturing wins in the future. 

MANUFACTURING VIDEOS

Explore critical steps and key regulatory requirements for developing CRISPR therapeutics and gain practical advice on how to avoid common pitfalls.

Looking to enhance performance and manufacturing from your DNA backbone without the regulatory concerns? Gain advantages over legacy plasmid DNA backbones and minicircles.

Primer Support™ 5G solid support is the fifth-generation solid support for oligonucleotide synthesis from Cytiva. By using Cytiva consumables, you can better leverage the whole family knowledge and expertise to efficiently optimize your molecule’s development.

Cell & Gene: The Podcast Host, Erin Harris, welcomes Romesh Subramanian, Ph.D., CEO of Ascidian Therapeutics, a Boston-based biotech focused on rewriting RNA to the pod. During the episode, Subramanian defines RNA exon editing, and they discuss the differences between RNA exon editing and gene editing. With current programs in ophthalmology, neurological and neuromuscular disorders, as well as rare diseases, Subramanian explains the overwhelming potential of RNA exon editing.

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