Editas Medicine, Inc. (NASDAQ:EDIT) Q1 2023 Earnings Call Transcript

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Cheng Li : Good morning. This is Chang on the line for Jay. Maybe two from us. So, I’m just wondering if there’s a chance where you have the capacity to dose more than 20 patients for the sickle cell disease program this year. And second question is on your ex-U.S. strategy. What you’re kind of thinking? And if you are planning to partner that program, what is the best timing to do that? Thank you.

Gilmore O’Neill : Thanks very much. So, do we have capacity to dose more than 20 patients? Yes. One of the important points, when we rolled out our strategy was to again sharpen our focus on developing and accelerating 301. And indeed, we have deployed capital to enable, not just the acceleration on the clinical side, but actually also to ensure that we have capacity to edit or other CMC capacity to edit and support those, that clinical acceleration. So indeed, we do have that capacity to dose more than 20 patients. And then, I think your second question was around ex-U.S. and the timing of partnership. I think as I said before, we are interested in partnering. We’re looking to a partner with a global footprint that would actually certainly support ex-U.S., particularly on the development and commercialization. And with regard to timing, we wouldn’t really discuss the timing until we actually have a deal signed and executed.

Operator: Our next question is from Joel Beatty with Baird.

Unidentified Analyst : Hello. This is Benjamin on for Joel. Thanks for taking our questions. Looking across other late-stage products in sickle cell and TDT, it appears that data sets of 30 patients could support approval. So, with Editas being on track to dose 20 patients by year-end, how quickly do you think you’ll be able to secure the necessary data to support regulatory approvals? Thank you.

Gilmore O’Neill: Yes. Thanks very much, Ben. Baisong?

Baisong Mei : As I mentioned earlier, in terms of total number of patients required to support registration and then need to have required alignment with the regulatory agency. And in terms of the progression of the study, we are very positive about the momentum. So, we are very optimistic we will be able to dose patients as we planned.

Gilmore O’Neill: So, I think the key thing, Ben, is that you’ve actually identified sort of a benchmark. But obviously, what we need to do is, as planned, sit with the regulators and come to an agreement on what the data set they would like to see for our programs.

Operator: Our next question is from Joon Lee with Truist Securities.

Joon Lee : So, I had the same question as Steve, but maybe a different way of asking. What percentage of sickle cell patients with hereditary persistence of fetal hemoglobin have mutations along the globin locus versus the BCL11A locus?

Baisong Mei: So, we do not have all the specifics on your question on that, but what we know is the mutation, the promoter region directly impacted the fetal globin expression. But BCLA is a transcription factor, which impacts multiple different cells in it. And the mutation of the BCL11A will have much different impact from the fetal globin expression only, will have other impacts, too. So the gene optimization is a much more complicated issue than the HPFH with the promoter region and mutation for the gamma globin promoters.

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