Editas lijkt vrij zeker van haar zaak getuige het navolgende citaat uit haar laatste update:
"We’ve developed a comprehensive data package supporting EDIT-101. Last month, we presented preclinical safety and pharmacology data at the European Society of Gene and Cell Therapy, or ESGCT meeting. These data demonstrated several important attributes of EDIT-101.
First, it has a rapid onset of editing on the order of a few weeks to few months, both in nonhuman primates and mice. Second, it’s well tolerated in nonhuman primates at AAV doses that are predicted to be therapeutically relevant in people. Third, it’s a highly targeted and specific medicine with editing restricted to photoreceptors and no detectable off-target activity. And fourth, neither preexisting nor induced immunity to either AAV5 or Staph aureus Cas9 impacted productive editing with EDIT-101.
This compelling data set underpins the decision by Allergan, our partner, to exercise their option to develop and commercialize EDIT-101 globally. And our confidence in this program led us to exercise our options to co-develop and share equally in the profits and losses from EDIT-101 in the United States.
We continue to work hard to put the pieces in place to dose patients following the clearance of the IND. We’re pleased with the National Institutes of Health determined that a Recombinant DNA Advisory Committee meeting was not necessary and our Phase 1/2 protocol is now registered with the NIH.
Once the IND is cleared, we will then need to get approval of the protocol from the Institutional Review Boards and the Institutional Biosafety Committees at our clinical trial sites and the sites can then begin to screen patients into the study. The Phase 1/2 trial of EDIT-101 will be an open label, dose escalation study, with primary endpoints designed to assess safety and tolerability and secondary endpoints measuring efficacy.
We expect to enroll 10 to 20 patients, starting with a low, but potentially efficacious dose in adults with severe vision loss. We will then progress to higher dose levels in adults with a broader range of vision loss. And then following a review of safety data by an Independent Data Monitoring Committee, pediatric patients with a broad range of vision loss will be included. Because the dose response curve is expected to be fairly sharp based on our preclinical data, we anticipate testing only a few dose levels.
Patients will be followed for one year in the primary analysis period, though, we do expect to also take to conduct an interim analyses at earlier time points. This is a truly exciting moment for us, and we look forward to embarking on our next chapter as a clinical-stage company"