Oral presentation of first achievement of in vivo retinal gene editing in non-human primates
Oral presentation of results demonstrating the potential of CRISPR/Cas9 gene editing to treat sickle cell
disease and beta-thalassemia
CAMBRIDGE, Mass., April 24, 2017 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome
editing company, today announced that six scientific abstracts have been accepted for presentation at the 20th Annual
Meeting of the American Society of Gene & Cell Therapy (ASGCT). The meeting will take place May 10-13 in Washington, D.C. The
Company is presenting data on its pipeline and platform technologies to support ongoing development programs.
Key Editas Medicine presentations at ASGCT will include data demonstrating:
- First report of results from in vivo gene editing of inherited retinal disease genes in non-human primates;
- CRISPR/Cas9-mediated editing and engraftment of hematopoietic stem cells preclinically for the treatment of
β-hemoglobinopathies;
- Self-inactivating Cas9: A proprietary approach for controlling exposure while maintaining efficacy in virally-delivered Cas9
applications; and
- UDiTaS™: A proprietary genome editing analytical method to comprehensively characterize multiple forms of editing, including
on- and off-target edits, large deletions, and translocations.
In addition, Editas scientists will be chairing sessions on cardiovascular and pulmonary gene and cell therapies, on AAV vector
systems, and a scientific symposium on getting cures to patients.
“Editas Medicine is at the forefront of the rapidly-advancing science that is unlocking the potential of CRISPR for patients,”
said Katrine Bosley, President and Chief Executive Officer, Editas Medicine. “These data at ASGCT showcase advances towards our
goal of making medicines for patients suffering from genetically-defined diseases and, potentially, for more common
genetically-treatable diseases as well.”
The complete list of Editas Medicine presentations is below. Abstracts can be accessed on the ASGCT website at www.abstractsonline.com/pp8/#!/4399.
Oral Presentations:
Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice
Date/Time: May 10, 11:00-11:15 a.m.
Location: Lincoln 5,6
Session: Genome Editing and Integration Analysis in Metabolic and Endocrine Disorders
CRISPR/Cas9-mediated Editing of Hematopoietic Stem Cells for the Treatment of
β-Hemoglobinopathies
Date/Time: May 11, 9:10-9:45 a.m.
Location: Lincoln 2,3,4
Session: Therapeutic Editing of the Human Genome and Epigenome
Efficient In Vivo Gene Editing of Inherited Retinal Disease Genes in Mice and Non-Human Primates
Date/Time: May 13, 11:00-11:15 a.m.
Location: Thurgood Marshall North
Session: Preclinical Progress Towards Therapies for Neurosensory Disorders
Poster Presentations:
Characterization of Targeted Integration with Viral and Non-Viral DNA Donors
Date/Time: May 11, 5:15-7:15 p.m.
Location: Exhibit Hall A & B South
Session: Gene Targeting and Gene Correction II
Self-inactivating Cas9: A method for Reducing Exposure While Maintaining Efficacy in
Virally-delivered Cas9 Applications
Date/Time: May 11, 5:15-7:15 p.m.
Location: Exhibit Hall A & B South
Session: Gene Targeting and Gene Correction II
UDiTaS™: A Streamlined Genome Editing Detection Method for On- and Off-target Edits, Large
Deletions, and Translocations
Date/Time: May 12, 5:45-7:45 p.m.
Location: Exhibit Hall A & B South
Session: Gene Targeting and Gene Correction III
About Editas Medicine
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting
their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the
promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of
patients.
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,” similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain these identifying words. 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 Annual Report on Form 10-K, 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 speak only as of the date hereof, and the Company expressly disclaims
any obligation to update any forward-looking statements, whether as a result of new information, future events or
otherwise.
Contacts Media: Cristi Barnett (617) 401-0113 cristi.barnett@editasmed.com Investors: Mark Mullikin (617) 401-9083 mark.mullikin@editasmed.com
