Accelerating Therapy Discovery and Approval With AI

This is the third part of an interview with David Dimmock, MBBS. For the first part, click here. For the second part, click here.

David Dimmock, MBBS

At the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD, Creyon Bio presented a case study on its use of an antisense oligonucleotide (ASO) therapeutic created using its AI discovery platform to treat a patient with an ultrarare disease indication. At the conference, CGTLive® spoke with David Dimmock, MBBS, chief medical officer at Creyon, to learn more about the company's future plans and goals.

Dimmock noted that the company will continue to seek to provide treatments for patients with very rare disease, but also seek to apply its platform to more common indications, as well. He emphasized the significance of having a platform and the importance of obtaining FDA approval for therapeutic products for very rare diseases.

CGTLive: Can you discuss other potential applications for Creyon's AI discovery platform?

David Dimmock, MBBS: The whole point of the platform is that it enables us to make drugs safely, whether that's a drug for 1 child, as in this case, or it's a drug for thousands. The platform, in and of itself, enables us to make drugs very efficiently for common diseases and for rare disease indications. We're at a point in our company where we announced last week that we're partnering with another company to treat neurodegenerative diseases—much more common indications. Similarly, we have several internal assets that we are taking through to investigational new drug application–enabling studies at this point in time.

Would you do more very rare, single patient indications in the future?

My day job 1 week a month is that I'm a rare disease doctor. I spend a week a month watching families go through things that no family should go through. As a company, we want to tackle this long tail of rare diseases where there are very few patients. The FDA has approved a drug based on 4 patients... So, we believe it is possible, even with very small patient populations to get drugs approved. But to get drugs approved, you need to do all the appropriate studies, which means you need to walk into the studies with a drug with a very high prior probability of success. Having the platform in place enables us to walk in with very high expectations that our drug is going to be safe. We could tell in a dish whether or not the drug works, we could see if it engages with RNA. We know how it's distributed in the liver or in the brain. But our dream, our hope, is that we will be at a point where we can get drugs that are approved. Why does it matter to have a drug approved? Well, if a drug is approved, it can then be paid for. Why does that matter? Well, most patients with rare diseases are seen in hospitals where they don't have people like Dr. Nicole Coufal who can go through the rigmarole of an FDA investigator-sponsored trial. If we have the drug approved, then any doctor with a patient with this disease can prescribe it. That actually makes these rare disease drugs more generally available.

Is there anything more the company is looking forward to?

As a company, we're very excited to be at the point where we can take more drugs into the clinic for common disease indications. Obviously, as a company we can't share too many details about them without breaking confidentiality rules. But we're definitely very excited over the next 18 months to start to see more common disease drugs come into the clinic. I'm also excited we have programs working looking at upregulating a gene for more rare disease—so using a different mechanism. I think the beauty of a platform is we don't need to pick a particular gene and run after that; any gene, any target that comes up—we can make a drug for that.

Do you have any additional thoughts on this year's ASGCT conference?

I think it's always an exciting meeting to be at to see cutting edge science. I come to meetings like this sometimes just to provide hope that we will have long-term treatments for many of the patients that I see. I think obviously with another gene therapy death announced a couple of days ago, it reminds everybody why we need to do this carefully and safely. I think there is an appropriate focus on making sure that we be careful and safe.

This transcript has been edited for clarity.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

REFERENCE
Dimmock DP, Bird L, Bouck A, et al. AI Enabled ASO Design Can Lead to Rapid Initiation of Treatment for an Ultra-Rare Disorder Leading to Allele Selective Knockdown of a Toxic Protein and Consequent Clinical Improvement. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #309