EDIT-101 for LCA10 BRILLIANCE trial adult low-dose cohort completed

EDIT-301 for sickle cell disease on track for IND filing by end of 2020

EDIT-201 for solid tumors preclinical data to be presented at SITC and ASH

Cash, cash equivalents, and marketable securities of $541 million as of September 30, 2020

CAMBRIDGE, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2020.

We were pleased to regain full operating control of our ocular programs per our new agreement with AbbVie, which provides us with important flexibility. We have finished dosing the first cohort with EDIT-101 in the landmark BRILLIANCE trial and enrollment remains active, said Cynthia Collins, Chief Executive Officer of Editas Medicine.

Ms. Collins continued, The advancements in our ocular portfolio are complemented by the strategic development of our engineered cell medicines. We are on track to file the IND for EDIT-301 for sickle cell disease in the fourth quarter and are eager to present additional data on this potentially best-in-class medicine and our large-scale manufacturing process at the upcoming American Society of Hematology Annual Meeting. Substantial progress has also been made in the development of EDIT-201, an allogeneic NK cell medicine to treat solid tumors. Preclinical data to be presented at ASH show enhanced tumor killing with EDIT-201 compared to unedited NK cells, and additional data on this program will be featured at the upcoming Society for Immunotherapy of Cancers Annual Meeting.

Recent Achievements and Outlook

In VivoCRISPR Medicines

Ex Vivo CRISPR Cell Medicines

Granted Rare Pediatric Disease Designation for treatment of sickle cell diseaseEDIT-301 was granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA). Under the FDAs Rare Pediatric Disease Designation and Voucher programs, a sponsor who receives approval for a drug or biologic for a rare pediatric disease may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product.

ASH data to highlight efficient editing with clinical scale manufacturing processPreclinical data to be presented at the 62nd American Society of Hematology Annual Meeting & Exposition (ASH) show CRISPR/Cas12a editing efficiency of the HBG1/2 promoter exceeding 90 percent after long term engraftment in mice of healthy donor hematopoietic stem cells manufactured at clinical scale.


Third Quarter 2020 Financial Results

Cash, cash equivalents, and marketable securities as of September 30, 2020, were $541.3 million, compared to $598.7 million as of June 30, 2020.

For the three months September 30, 2020, net income attributable to common stockholders was $7.8 million, or $0.12 per share, diluted, compared to net loss of $32.9 million, or $0.66 per share, for the same period in 2019.

Upcoming Events

Editas Medicine plans to participate in the following scientific and medical conferences:

Editas Medicine plans to participate in the following investor events:

Conference Call

The Editas Medicine management team will host a conference call and webcast today at 5:00 p.m. ET to provide and discuss a corporate update and financial results for the third quarter 2020. To access the call, please dial (844) 348-3801 (domestic) or (213) 358-0955 (international) and provide the passcode 9992536.A live webcast of the call will be available on the Investors section of the Editas Medicine website at http://www.editasmedicine.com and a replay will be available approximately two hours after its completion.

About EDIT-101 EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About BRILLIANCE The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10) is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. Clinical trial sites are enrolling up to five cohorts testing up to three dose levels in this open label, multi-center study. Both adult and pediatric patients (3 17 years old) with a range of baseline visual acuity assessments are eligible for enrollment. Patients receive a single administration of EDIT-101 via subretinal injection in one eye. Additional details are available on http://www.clinicaltrials.gov (NCT#03872479).

About EDIT-201EDIT-201 is an experimental, allogeneic natural killer (NK) cell medicine under investigation for the treatment of solid tumor cancers. EDIT-201 is comprised of NK cells derived from pooled healthy donor blood and genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the CISH and TGFR2 genes. Editing of the CISH and TGFR2 genes is designed to overcome resistance to therapeutic antibodies and improve NK cell persistence.

About EDIT-301EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans with respect to EDIT-101 and filing an IND for EDIT-301 by the end of 2020. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. Those risks and uncertainties include, among other things, that the Company's expectations regarding the effects of COVID-19 may be incorrect, that data from the Company's development programs may not support registration or further development of its potential medicines due to safety, efficacy or other reasons, and other risks listed under Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent Companys views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

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Editas Medicine Announces Third Quarter 2020 Results and Update - GlobeNewswire

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