In Vivo
CRISPR is the therapy
GENETIC DISEASES
Dosed first ATTR patient in November 2020 with single-dose, CRISPR/Cas9-based therapy, NTLA-2001.
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Program
Research
Candidate Selection
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
NTLA-2001:
Transthyretin Amyloidosis
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Early-Stage
Clinical
Clinical
NTLA-2002:
Hereditary Angioedema
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IND-Enabling
Hemophilia A and B
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Research
Research Programs
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Research
Research Programs
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Research
* Lead development and commercial party
** Rights to certain in vivo targets
Last Updated: January 7, 2021
Ex Vivo
CRISPR creates the therapy
IMMUNO-ONCOLOGY / AUTOIMMUNE DISEASE
We expect to submit an IND or IND-equivalent in mid-2021 for WT1-directed TCR T cell therapy, NTLA-5001 for the treatment of AML.
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
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Early-Stage
Clinical
Clinical
NTLA-5001:
Acute Myeloid Leukemia
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IND-Enabling
Solid Tumors
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Research
Undisclosed Programs
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Research
Other Novartis Programs
Undisclosed
*** Milestones & royalties
Last Updated: January 7, 2021
