In Vivo
CRISPR is the therapy
GENETIC DISEASES
Dosed first ATTR patient in November 2020 with single-dose, CRISPR/Cas9-based therapy, NTLA-2001.
Learn more about our in vivo programs.
Program
Research
Candidate Selection
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
NTLA-2001:
Transthyretin (ATTR) Amyloidosis
Learn more
Early-Stage
Clinical
Clinical
NTLA-2002:
Hereditary Angioedema
Learn more
Early-Stage
Clinical
Clinical
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 6, 2021
Ex Vivo
CRISPR creates the therapy
IMMUNO-ONCOLOGY / AUTOIMMUNE DISEASE
We plan to initiate patient screening for a first-in-human Phase 1 study of NTLA-5001, a WT1-directed TCR T cell therapy for the treatment of AML, by the end of 2021.
Learn more about our ex vivo programs.
Program
Research
Candidate Selection
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
OTQ923 / HIX763:
Sickle Cell Disease
Learn more
Early-Stage
Clinical
Clinical
NTLA-5001:
Acute Myeloid Leukemia
Learn more
Early-Stage
Clinical
Clinical
Solid Tumors
Learn more
Research
Allo Undisclosed
Learn more
Research
Other Novartis Programs
Undisclosed
*** Milestones & royalties
Last Updated: October 6, 2021
