“We plan to nominate a lead product candidate by the end of this year”August 12, 2020
In an exclusive conversation with Future Medicine, Sekar Kathiresan, MD, Co-Founder and Chief Executive, said Verve Therapeutics — as a product-focused company — is agnostic to the technology it uses, and is therefore continuously conducting multiple programmes using base editing or CRISPR-Cas9 against various gene targets in the liver.
Could you elaborate on the lead programme that you are planning for human studies by the year-end?
We have not yet declared a lead programme. We are evaluating multiple programs using base editing or CRISPR-Cas9 against various gene targets in the liver. We plan to nominate a lead product candidate by the end of this year.
We assume that you preferred ABE over CRISPR-CAS9 and other technologies in the animal trials as an obvious choice for better efficiency and reduced off-target activity. However, is it possible to quantify its superior on-target editing efficiency and reduced indel formation rate in comparison with other editing technology?
Verve is a product-focused company, not a platform company. We are largely agnostic to the technology we use. In that spirit, we are simultaneously evaluating both editors – CRISPR-Cas9 as well as ABE – in several pre-clinical models including cells, mice, and non-human primates. Using CRISPR-Cas9 as the editor, we have been able to achieve similar levels of editing in non-human primates as the ABE data I have presented. Beyond efficacy, the two editors differ in a number of ways including the capacity to generate double-strand breaks and the ability to edit RNA (in addition to DNA). We are evaluating these effects carefully and will be looking to identify a lead editor by the end of the year.