Goldilocks and the Three Bears

June 5, 2024 by Jeff Briganti

CRISPR gene editing, CDMOs and the art of “just right”

I know. I know. You must be questioning how the story of Goldilocks is related to gene editing and CDMOs. But the connection between the two isn’t far-fetched at all.

The story, if you remember, has Goldilocks wandering through the forest alone. She enters a house and after sitting in three different chairs and trying three different bowls of porridge, she makes her way upstairs where she encounters three beds.

The first bed is too big (how can a bed be too big?); the second too small; the third is “just right.” The same thing can happen when shopping for a CDMO, particularly when your goal is a custom protein for therapeutic gene editing programs.

I’ve been working more closely with our CRISPR gene editing portfolio this year. And, while speaking with clients or potential clients, I’ve been asking more about how they make their decisions regarding products and services such as nucleases, guide RNA providers, and RNP complexing. I believe there is a strong desire for clients to use catalog or standardized products, such as Spy Fi™ Cas9 Nuclease.

However, for a variety of reasons, clients also have the need for custom developed and custom manufacture products. While shopping for custom manufacturing services, clients can run into the Goldilocks problem of trying to figure out what’s “just right.”

Some CDMOs are too big. They focus on manufacturing at huge scale, appeasing clients that need batches done in 15k L fermenters. They may not find small- or mid-scale work important enough to give you the focus you need. Big CDMOs may be more experienced in manufacturing for diagnostics, based primarily on the maturity of the market.

They also might not have developed the expertise to really understand the requirements of CRISPR nucleases for therapeutic programs. They probably can’t help you with guide-RNA (gRNA) or RNP complexing.

On the other hand, some CDMOs are too small or inexperienced, particularly when the goal is therapeutics. They may not have the ability to manufacture both research-use only and cGMP grade material, meaning a potential tech transfer to another CDMO as your program progresses. They might not have the ability to scale as your program matures, and they may not have the right regulatory experience to help progress efficiently.

Finally, there is the CDMO that is “just right” for custom CRISPR gene editing products. Aldevron, being a CDMO dedicated to cell and gene therapy, has the necessary focus to be a leading supplier of catalog and custom nucleases for therapeutics. You can learn more about our expertise in the area with the materials linked below.

• View our Custom Protein Brochure
• Download our CRISPR Gene Editing Brochure
• Read our Enabling Novel CRISPR-Cas9 Delivery Case Study
• Want to learn more? Contact Jeff Briganti
• Have an idea for a topic? Let us know!

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