Anticipated 2022 milestones include initial clinical data for EDIT-301 in sickle cell disease by year-end, dosing of first TDT patient with EDIT-301, initiation of pediatric high-dose cohort for EDIT-101 in LCA10, and a clinical data update on EDIT-101 in the second half of 2022
Company announces new development candidates EDIT-103 for RHO-adRP and EDIT-202 multiplexed iNK cell therapy for solid tumors
CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that James C. Mullen, Chairman, President, and Chief Executive Officer, will discuss the Company’s recent progress and outlook for its gene editing medicines and platform technology at the 40th Annual J.P. Morgan Healthcare Conference being held virtually on Wednesday, January 12 at 10:30 a.m. EST.
“Editas is developing a high-value pipeline of gene editing therapeutic candidates enabled by best-in-class next generation technologies, such as our efficient, high precision AsCas12a nuclease and SLEEK editing platform,” said Mr. Mullen. “In 2021, we achieved proof-of-concept with EDIT-101 in LCA10, which supports our foundational in vivo technology in additional ocular indications, such as our new development candidate EDIT-103 for RHO-adRP. This year we expect to obtain initial clinical results from our ex vivo EDIT-301 program in sickle cell disease and dose the first patient in the EDIT-301 study of transfusion-dependent beta thalassemia. We’re also excited to advance our alpha-beta T cell oncology program, partnered with Bristol Myers Squibb, and our EDIT-202 iNK cell therapy for solid tumors towards the clinic.”
Recent Progress and Upcoming Milestones
In Vivo Gene Edited Medicines Outlook
Editas Medicine completed dosing of all adult cohorts in its BRILLIANCE study of EDIT-101, an investigational treatment for Leber Congenital Amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. Previously announced preliminary
EDIT-101 clinical results
demonstrated a favorable safety profile and encouraging signals of clinical benefit. The Company remains on track to complete dosing of the pediatric mid-dose cohort in the first half of 2022, and expects to initiate dosing of the pediatric high-dose cohort this year following a safety assessment by an independent data monitoring committee.Additionally, the Company is expanding enrollment in one or more of the previously completed adult cohorts to support anticipated registrational trial design and endpoints. Editas will provide additional details regarding the expansion later this year.Editas also expects to provide a clinical update on the BRILLIANCE trial in the second half of 2022, including safety and efficacy assessments on all patients who have had at least 6 months of follow-up evaluations.The Company also anticipates establishing registrational trial criteria for EDIT-101 by year-end 2022.The Company is advancing additional in vivo ocular editing programs, leveraging its learnings from EDIT-101, and has declared a new development candidate, EDIT-103, for treatment of rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive form of retinal degeneration, which is progressing towards IND-enabling studies. EDIT-103 uses two adeno-associated virus (AAV) vectors to knockout and replace mutations in the rhodopsin gene in order to preserve photoreceptor function. Editas Medicine believes that simultaneously knocking out and replacing a gene in the same cell in vivo is a significant technical breakthrough, potentially leading to findings that could address other autosomal dominant diseases, where gain of negative function needs to corrected.An optimized version of EDIT-102 for treatment of Usher syndrome 2…….