Editas Medicine to Present New Data Demonstrating Progress Towards Creating Genome Editing Medicines at the American Society of Gene & Cell Therapy Annual Meeting
Key
- EDIT-101, the Company’s experimental medicine for the treatment of Leber Congenital Amaurosis type 10, administered to non-human primates via subretinal injection was well-tolerated;
- Therapeutically relevant editing levels in non-human primates regardless of pre-existing or induced immunity to Staphylococcus aureus Cas9;
- Specificity of EDIT-101 with no verified off-targets in the human genome as assessed by a two-staged approach using orthogonal methods;
- In vitro validation of an exon deletion editing strategy with the potential to treat Usher Syndrome type 2a (USH2A)-associated retinal disease;
- In vivo proof-of-concept in animal models for developing a CRISPR-based medicine for the treatment of ocular herpetic keratitis caused by latent herpes simplex virus-1 (HSV-1); and
- Fetal hemoglobin induction by editing of novel therapeutic sites identified through saturation genomic CRISPR screening of the beta-globin locus as a potential treatment for sickle cell anemia.
In addition, Editas Medicine’s Chief Scientific Officer will speak at the
“We are making significant scientific advances in our programs to unlock the potential of CRISPR genome editing for making medicines,” said
The complete list of
Oral Presentation:
Evaluation of Tolerability and Immunogenicity of EDIT-101 Following Subretinal Injection in Non-human Primate
Date/Time:
Location: Salon A-5
Session: Preclinical Pharmacology and Toxicology Studies and Assessment of Gene Therapy in Large Animal Models
Editas Medicine Poster Presentations:
Treatment of Herpetic Keratitis with CRISPR/Cas9 Gene Editing in a Rabbit Disease Model
Date/Time:
Location: Stevens Salon C, D
Session: Neurologic Diseases (Including Ophthalmic and Auditory Diseases) I
Potent HbF Induction Following ssODN-Mediated Repair of Cas9-Induced DSB at the HBG Promoter in CD34+ HSPC
Date/Time:
Location: Stevens Salon C, D
Session: Hematologic & Immunologic Diseases I
Saturated Mutagenesis Surrounding Beta-globin Locus Identifies Novel Therapeutic Targets for Fetal Globin Induction and Treatment of Sickle Cell Anemia
Date/Time:
Location: Stevens Salon C, D
Session: Hematologic & Immunologic Diseases I
Improving Efficacy of CAR T cells Through CRISPR/Cas9 Mediated Knockout of TGFbR2
Date/Time:
Location: Stevens Salon C, D
Session: Cancer – Targeted Gene & Cell Therapy I
Efficient Targeted Integration in Human T cells with CRISPR-Cas9 for the Treatment of X-Linked Hyper-IgM Syndrome
Date/Time:
Location: Stevens Salon C, D
Session: Hematologic & Immunologic Diseases II
Gene Editing Specificity Assessment for EDIT-101, an LCA10 Therapeutic Candidate
Date/Time:
Location: Stevens Salon C, D
Session: Pharmacology/Toxicology Studies or
Research Collaboration Poster Presentations (Editas Medicine Author):
Development of an Assay to Detect Pre-existing Anti-Cas9 Antibodies and an Estimate of the Prevalence of Anti-Staphylococcus- and Streptococcus-Cas9 Antibodies in the US Population
Date/Time:
Location: Stevens Salon C, D
Session: Gene Targeting & Gene Correction II
Preclinical Modeling Highlights the Therapeutic Potential of the Adoptive Transplant of Gene Corrected T cells in X-Linked Hyper-IgM Syndrome
Date/Time:
Location: Stevens Salon C, D
Session: Hematologic & Immunologic Diseases
Evaluation of Therapeutic Potential of Human USH2A Gene Lacking Exon 13 (USH2A-∆Ex13) for Restoring Ciliogenesis
Date/Time:
Location: Stevens Salon C, D
Session: Neurologic Diseases (Including Ophthalmic and Auditory Diseases) I
About
As a leading genome editing company,
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. 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
Contacts:
Media
(617) 401-0113
cristi.barnett@editasmed.com
Investors
(617) 401-9083
mark.mullikin@editasmed.com
Source: Editas Medicine, Inc.