On April 30, 2018 Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, reported that 10 scientific abstracts, including three from research collaborations, have been accepted for presentation at the 21st Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) (Press release, Editas Medicine, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345605 [SID1234525852]). The meeting will take place May 16-19 in Chicago. The Company is presenting data on its pipeline and platform technologies to support ongoing development programs.
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Key Editas Medicine presentations at ASGCT (Free ASGCT Whitepaper) will include data demonstrating:
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 ASGCT (Free ASGCT Whitepaper) Gene Editing Workshop, a pre-meeting program, on May 15.
"We are making significant scientific advances in our programs to unlock the potential of CRISPR genome editing for making medicines," said Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. "At ASGCT (Free ASGCT Whitepaper), we will showcase our recent scientific advances as we make progress towards the clinic and towards our goal of making medicines for people living with serious diseases."
The complete list of Editas Medicine presentations is below. Abstracts can be accessed on the ASGCT (Free ASGCT Whitepaper) website at View Source
Oral Presentation:
Evaluation of Tolerability and Immunogenicity of EDIT-101 Following Subretinal Injection in Non-human Primate
Date/Time: May 18, 4:00 – 4:15 p.m.
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: May 16, 5:30 – 7:30 p.m.
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: May 16, 5:30 – 7:30 p.m.
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: May 16, 5:30 – 7:30 p.m.
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: May 16, 5:30 – 7:30 p.m.
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: May 17, 5:15 – 7:15 p.m.
Location: Stevens Salon C, D
Session: Hematologic & Immunologic Diseases II
Gene Editing Specificity Assessment for EDIT-101, an LCA10 Therapeutic Candidate
Date/Time: May 18, 5:45 – 7:45 p.m.
Location: Stevens Salon C, D
Session: Pharmacology/Toxicology Studies or Assay Development
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: May 17, 5:15 – 7:15 p.m.
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: May 18, 5:45 – 7:45 p.m.
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: May 18, 5:45 – 7:45 p.m.
Location: Stevens Salon C, D
Session: Neurologic Diseases (Including Ophthalmic and Auditory Diseases)