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EDIT-101 is an experimental CRISPR genome editing medicine being investigated for the treatment of Leber congenital amaurosis 10 (LCA10). It is set to be the first in vivo, or editing inside the body, CRISPR-based medicine administered to people anywhere in the world. In the Phase 1/2 clinical trial,
“At Editas Medicine, we are pioneering the possible by harnessing the power of genome editing, engineered cell therapy and AAV gene delivery to develop a pipeline of genomic medicines for people living with serious diseases,” said Bosley. “With our recent successes, including the FDA’s acceptance of our IND for EDIT-101, we are entering 2019 with strong momentum towards achieving our EM22 goals. We look forward to entering the clinic later this year, and we hope to transform the lives of people living with LCA10.”
Ms. Bosley will also provide an update on the Company’s progress on EM22. By year-end 2022,
Continued Commitment to Ocular Disorders
- The LCA10 program is on track to be the first in vivo CRISPR-based genome editing medicine with patient dosing expected in the second half of 2019.
- The Company now has ocular programs in early research to treat Usher syndrome 2A (USH2A) and retinitis pigmentosa.
Important progress in engineered cell medicines
- The Company made recent advances toward a durable medicine for sickle cell and beta-thalassemia. Editing at the HBG1/2 site is a differentiated approach for development of a human therapeutic for the treatment of sickle cell disease and beta-thalassemia as compared to other medicines currently under development that edit at the BCL11A erythroid enhancer (BCL11Ae) site. Notably, editing HBG1/2 promoters upregulated fetal hemoglobin with superior repopulation of red blood cell precursors as compared to editing the BCL11Ae site. The red blood cell precursors from bone marrow edited at the BCL11Ae site had lower productive editing rates compared to other lineages and showed increased level of apoptosis, or programmed cell death, in erythroid culture compared to HBG1/2 promoter-edited cells.
- In the Company’s collaboration with
Juno Therapeutics, Inc., a Celgenecompany, CRISPR-edited product candidates are advancing in both solid and liquid tumors.
Advancing Organizational Excellence
- The Company added key talent across hematology, oncology, ophthalmology, manufacturing, and ex vivo (editing outside the human body) research in 2018. This expertise is critical to the continued advancement of Editas Medicine’s pipeline and platform.
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 Leber Congenital Amaurosis
Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.
As a leading genome editing company,
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 clinical trial timeline of EDIT-101 and the Company’s EM22 goals. 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 Company’s 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 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 Quarterly Report on Form 10-Q, which is on file with the
Source: Editas Medicine, Inc.