R&D pipeline*: Looking ahead
By selecting and focusing on sentinel in vivo and ex vivo programs, we are positioned to build a pipeline across a range of diseases and to potentially enable additional related therapeutic applications using the CRISPR/Cas9 technology.
|Programs||Commercial Rights||Type of Edit||Delivery||Status||Comments|
|In Vivo||Transthyretin Amyloidosis (ATTR)||Knockout||LNP||NHP Studies||
|Hepatitis B Virus (HBV)||Knockout||LNP||In vitro Guide Evaluation||
|Alpha-1 Antitrypsin Deficiency (AATD)||Knockout Repair||LNP||Guide Design & Evaluation|
|Inborn Errors of Metabolism – Primary Hyperoxaluria (PH1)||Knockout, Repair, Insertion||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 October 10, 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 (ATTR), which we are co-developing with Regeneron Pharmaceuticals, Inc., alpha-1 antitrypsin deficiency (AATD), hepatitis B virus (HBV), and inborn errors of metabolism (IEMs).
Our sentinel ex vivo programs include both proprietary and partnered programs. Our proprietary programs are lead by our eXtellia division, and information can be found at eXtellia
Our partnered ex vivo programs are focused on chimeric antigen receptor T cells, (“CAR T cells”) and hematopoietic stem cells (“HSCs”), the stem cells from which all of the various types of blood cells originate, which we are researching in collaboration with Novartis Institutes for Biomedical Research, Inc.