Item 7.01. Regulation FD Disclosure.
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The information in this Item 7.01, including Exhibit 99.1 attached hereto, is intended to be furnished and shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01. Other Events.
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EDIT-101 was well-tolerated in both the low-dose and mid-dose cohorts, with most adverse events ("AEs") being mild and primarily resulting from the surgical procedure and subretinal injection. There were no dose limiting toxicities, which are defined as vision-threatening toxicities or severe non-ocular AEs that occur before or at the week four visit and assessed by the investigator as being related to EDIT-101 and not the administration procedure. Mild anterior chamber inflammation was observed, and adequately controlled with oral steroids. No Cas9-specific antibody or T-cell response was detected. To date, no treatment-related cataracts, edema, or retinal thinning have been observed.
Efficacy was assessed based on available data from five subjects treated in the low-dose and mid-dose cohorts who had at least three months of post-treatment follow-up, focusing on those measures demonstrated to be consistent and reproducible in subjects with CEP290 retinal degeneration, including best corrected visual acuity ("BCVA"), full-field light sensitivity threshold ("FST") testing and ability to navigate standardized navigation courses, or Visual Function Navigation ("VNC"). Two of three subjects in the mid-dose cohort followed for up to six months showed early efficacy signals suggesting productive editing and potential early clinical benefits, including improvements in BCVA, FST, and/or mobility navigation. Mid-dose cohort Subject 1 showed improvement in BCVA of approximately 0.7 logMAR at month 1.5 which was sustained at month six follow-up. In addition, there was a positive trend toward improved retinal sensitivity by FST in the study eye relative to the untreated eye. The subject also demonstrated a five-level improvement in mobility at month six, as assessed with the VNC. Mid-dose cohort Subject 2 showed improvement by month three with a stable BCVA and a notable improvement in retinal sensitivity in the study eye relative to the untreated eye by FST, detectable at week six that continued to improve through month three.
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Item 9.01. Financial Statements and Exhibits. (d) Exhibits Exhibit No. Description 99.1 Press Release datedSeptember 29, 2021 104 Cover Page Interactive Data File (embedded within the Inline XBRL document)
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