EDIT) President and CEO Cynthia Collins will discuss the Company’s progress on developing in vivo and engineered cell medicines and building the leading genomic medicine company. 

In her remarks, Ms. Collins will discuss several components of the Company’s progress and detail timelines, including plans to:

  • Dose the first patient in the Brilliance clinical trial of EDIT-101 (AGN-151587) for the treatment of Leber congenital amaurosis 10 (LCA10) in the first quarter of 2020 and complete dosing the adult low- and mid-dose cohorts by the end of the year;
  • File an Investigational New Drug (IND) application for EDIT-301 for the treatment of sickle cell disease by the end of 2020;
  • Initiate IND-enabling studies for engineered natural killer (NK) cell medicine for the treatment of solid tumors;
  • Advance alpha-beta T cell medicines in partnership with Bristol-Myers Squibb Company;
  • Establish in vivo preclinical proof-of-concept for an engineered iPSC-derived NK (iNK) cell medicine; and
  • Establish in vivo preclinical proof-of-concept for a neurological indication.

In addition to sharing details on the Company’s progress and timelines, Ms. Collins will also discuss recent achievements and outlook for 2020:

  • EDIT-101 is on track to be the first in vivo CRISPR-based genome editing medicine with first patient dosing expected in the first quarter of 2020.
  • EDIT-102 development candidate declared for the treatment of Usher syndrome 2A (USH2A).
  • Declare a development candidate for Autosomal Dominant Retinitis Pigmentosa Type 4 (adRP4) in 2020.
  • Plans to establish in vivo preclinical proof-of-concept for a neurological indication in 2020 from collaboration with Asklepios BioPharmaceutical, Inc. (AskBio).

  • File an IND for EDIT-301 for the treatment of sickle cell disease by the end of 2020.
  • Progress on the Company’s collaboration with Bristol-Myers Squibb to advance alpha-beta T cell medicines for the treatment of both solid and liquid tumors.
  • Declare a development candidate and initiate IND-enabling activities this year for a gene edited healthy donor NK cell medicine.
  • Progress towards establishing in vivo preclinical proof-of-concept for an engineered iNK cell medicine using technology from BlueRock Therapeutics.

  • Advanced and strengthened pipeline through multiple collaborations and licensing agreements, including Bristol-Myers Squibb, BlueRock Therapeutics LP, AskBio, MaxCyte, Inc., and Sandhill Therapeutics, Inc.
  • Strengthened the Company’s executive team with hiring of a permanent Chief Executive Officer, Chief Financial Officer, Chief Medical Officer, and Senior Vice President, Operations (Manufacturing).

    • www.editasmedicine.com.

    Categorized in : Natural Medicine