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|
|Transthyretin Amyloidosis (ATTR)||
|Knockout||LNP||Progressing to NHP 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|
|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.
- Liver Diseases
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
- Non-CAR T oncology
- Autoimmune and inflammatory