DNA String


We believe our approach to selecting our sentinel in vivo and ex vivo programs positions us to build a pipeline across a range of indications and to generate a wealth of data that opens the potential therapeutic applications of the CRISPR/Cas9 technology across a broad range of diseases.
Programs Partnerships Type of Edit Delivery Upcoming Milestones
In Vivo
Transthyretin Amyloidosis (ATTR) Co-developing with Regeneron Knockout LNP to Liver Select one to two development candidates and advance to IND enabling studies in 2H2017/1H2018
Alpha-1 Antitrypsin Deficiency (AATD) Proprietary Knockout
LNP to Liver
Hepatitis B Virus (HBV) Proprietary Knockout LNP to Liver
Inborn Errors of Metabolism (IEMs) Proprietary Knockout
LNP to Liver
Ex Vivo
Hematopoietic Stem Cells (HSCs) Selectively partnered with Novartis; proprietary Knockout
Electroporation First Novartis IND expected to be submitted in 2018
CAR-T Cells Partnered with Novartis Knockout Insertion Electroporation Advance preclinical development

Our sentinel in vivo programs focus on the use of Lipid Nanoparticle (LNPs) for delivery of the CRISPR/Cas9 complex to the liver. Our in vivo pipeline includes proprietary programs targeting transthyretin amyloidosis, or ATTR, which we are co-developing with Regeneron Pharmaceuticals, Inc., alpha-1 antitrypsin deficiency, or AATD, hepatitis B virus, or HBV, and inborn errors of metabolism, or IEMs.

Regeneron PharmaceuticalsIntellia Therapeutics

Initial Focus
  • Liver Diseases
    (LNP Delivery)
Intellia Therapeutics
Additional Exploration
  • Eye
  • Muscle
  • CNS

Our sentinel ex vivo programs include both proprietary and partnered programs focused on chimeric antigen receptor T cells, or CAR T cells, and hematopoietic stem cells, or HSCs, the stem cells from which all of the various types of blood cells originate, which we are developing in collaboration with Novartis Institutes for Biomedical Research, Inc.


  • CAR T oncology
  • HSC

eXtellia Therapeutics

  • Non-CAR T oncology
  • Autoimmune and inflammatory