DNA String

R&D Pipeline*: Looking Ahead

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 Commercial Rights Type of Edit Delivery Status Comments
In Vivo
Transthyretin Amyloidosis (ATTR)
Knockout LNP Progressing to NHP Studies
  • 2017 NHP Studies
  • 2H17-1H18 IND-enabling studies
Hepatitis B Virus (HBV) Knockout LNP In vitro Guide Evaluation 2017 Positioned to commence animal model studies
Alpha-1 Antitrypsin Deficiency (AATD) Knockout Repair LNP Guide Design & Evaluation
Inborn Errors of Metabolism – Primary Hyperoxaluria (PH-1) Knockout
LNP Guide Design & Evaluation
Ex Vivo
Hematopoietic Stem Cells (HSC) Knockout
Electroporation Late Stage Preclinical Development
Chimeric Antigen Receptor T Cell (CAR-T) Knockout Insertion Electroporation Preclinical Development

*The table illustrates our discovery programs and opportunities as of March 31, 2017.

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