Our Pipeline

We are building a pipeline of in vivo and ex vivo therapies, as well as continuing to develop innovative modular platform capabilities.

Program
Research
Candidate Selection
IND-Enabling
Phase 1
Phase 2
Phase 3
Partner
NTLA-2001:
Transthyretin Amyloidosis
Learn more
Phase 1
NTLA-2002:
Hereditary Angioedema
Learn more
IND-Enabling
Hemophilia A and B
Learn more
Research
Research Programs
Learn more
Research
Research Programs
Learn more
Research

* Lead development and commercial party
** Rights to certain
in vivo targets

Last Updated: October 19, 2020
Program
Research
Candidate Selection
IND-Enabling
Phase 1
Phase 2
Phase 3
Partner
OTQ923 / HIX763:
Sickle Cell Disease
Learn more
Phase 1/2
Phase 1/2
NTLA-5001:
Acute Myeloid Leukemia
Learn more
IND-Enabling
Solid Tumors
Learn more
Research
Undisclosed Programs
Learn more
Research
Other Novartis Programs
Undisclosed

*** Milestones & royalties

Last Updated: October 19, 2020
Menu