EDIT-101 Phase 1/2 BRILLIANCE trial initial clinical data demonstrated favorable safety profile and preliminary evidence of clinical benefit; enrollment ongoing in adult high-dose and pediatric mid-dose cohorts
EDIT-301 Phase 1/2 RUBY trial for the treatment of sickle cell disease currently enrolling study participants
Presented preclinical data on novel SLEEK gene editing technology enabling high efficiency, multi-transgene knock-in in multiple clinically relevant cell types
CAMBRIDGE, Mass., , Nov. 08, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2021.
“Editas recently marked an important milestone with the presentation of initial EDIT-101 results demonstrating a favorable safely profile and encouraging signals of clinical activity and evidence of ocular gene editing. Encouraged by these data, we look forward to obtaining additional clinical results, including from our ongoing adult high-dose and pediatric mid-dose cohorts,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “We have also continued to make excellent progress advancing our broader clinical and preclinical programs, including the expansion of our gene editing capabilities, as exemplified by our proprietary SLEEK knock-in technology, which we are already applying in our iNK program for solid tumors.”
Recent Achievements and Outlook
In Vivo Gene Edited Medicines
Ex Vivo Gene Edited Medicines
Cellular Therapy
Gene Editing Platform
Editas Medicine presented preclinical data at the Cold Spring Harbor Laboratory’s Genome Engineering: CRISPR Frontiers meeting on its SLEEK gene editing technology. The data demonstrated that SLEEK, which utilizes the Editas-engineered AsCas12a nuclease, resulted in the knock-in of multiple clinically relevant transgenes through a proprietary process that selects for cells containing the knock-in cargo. More than 90 percent knock-in efficiencies were observed in various clinically relevant target cells, including iPSCs, T cells, and NK cells. Additionally, SLEEK may be used to fine-tune the expression levels of transgene cargos, an important attribute of next-generation cell therapy medicines.
The Company also presented preclinical data at the TIDES Oligonucleotide and Peptide Therapeutics Conference demonstrating the editing and manufacturing advantages of the AsCas12a nuclease. Specifically, the data showed that AsCas12a achieves high editing rates across multiple cell types, highly efficient multiplexed editing, single and dual site-specific knock-ins, and has higher intrinsic specificity and sequence fidelity due to its shorter chemically synthesized guide RNA.
Corporate
Third Quarter 2021 Financial Results
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 8:00 a.m. ET to provide and discuss a corporate update and financial results for the second quarter 2021. To access the call, please dial 877-407-0989 (domestic) or 201-389-0921 (international) and ask for the Editas Medicine earnings call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at www.editasmedicine.com, and a replay will be available approximately two hours after its completion.
About Editas Medicine
As 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 www.editasmedicine.com.
About EDIT-101
EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. 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. Patients are monitored every three months for a year after dosing and less frequently for an additional two years thereafter. Additional details are available on www.clinicaltrials.gov (NCT#03872479).
About EDIT-301
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 is comprised of sickle patient (for SCD) or normal donor (for TDT) CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) that targets the HBG1 and HBG2 promoters in the beta-globin locus where naturally occurring fetal hemoglobin (HbF) inducing mutations reside. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in HbF production, which has the potential to provide a one-time, durable treatment benefit for people living with sickle cell disease and TDT.
About RUBY
The RUBY Trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576).
Forward-Looking Statements
This 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 initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, including completing dosing of both the adult high-dose and pediatric mid-dose cohorts in the first half of 2022 and beginning patient dosing in the RUBY trial in the first half of 2022, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, the timing or likelihood of regulatory filings and approvals, including filing an IND for EDIT-301 for the treatment of transfusion-dependent beta thalassemia by the end of 2021, and the Company’s expectations regarding cash runway. 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 pre-clinical studies and clinical trials, including the BRILLIANCE and RUBY trials, and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical 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 Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings 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 Company’s 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.
EDITAS MEDICINE, INC. Condensed Consolidated Statements of Operations (unaudited) (amounts in thousands, except per share and share data) | |||||||
Three Months Ended | |||||||
September 30, | |||||||
2021 | 2020 | ||||||
Collaboration and other research and development revenues | $ | 6,197 | $ | 62,841 | |||
Operating expenses: | |||||||
Research and development | 29,265 | 33,916 | |||||
General and administrative | 16,185 | 19,936 | |||||
Total operating expenses | 45,450 | 53,852 | |||||
Operating (loss) income: | (39,253 | ) | 8,989 | ||||
Other income (expense), net: | |||||||
Other income (expense), net | 19 | (1,396 | ) | ||||
Interest income, net | 152 | 226 | |||||
Total other income (expense), net | 171 | (1,170 | ) | ||||
Net (loss) income | $ | (39,082 | ) | $ | 7,819 | ||
Net (loss) income per share, basic | $ | (0.57 | ) | $ | 0.13 | ||
Net (loss) income per share, diluted | $ | (0.57 | ) | $ | 0.12 | ||
Weighted-average common shares outstanding, basic | 68,219,742 | 62,144,118 | |||||
Weighted-average common shares outstanding, diluted | 68,219,742 | 62,697,173 |
EDITAS MEDICINE, INC. Selected Condensed Consolidated Balance Sheet Items (unaudited) (amounts in thousands) | ||||||
September 30, | December 31, | |||||
2021 | 2020 | |||||
Cash, cash equivalents, and marketable securities | $ | 657,039 | $ | 511,774 | ||
Working capital | 516,701 | 360,879 | ||||
Total assets | 717,019 | 572,602 | ||||
Deferred revenue, net of current portion | 60,888 | 73,984 | ||||
Total stockholders’ equity | 586,794 | 393,586 |
Contacts:
Media
Cristi Barnett
(617) 401-0113
[email protected]
Investors
Ron Moldaver
(617) 401-9052
[email protected]
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