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|
|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|
|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.
- 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