
In Vivo
CRISPR is the therapy
GENETIC DISEASES
Advancing first-in-human studies for NTLA-2001 and NTLA-2002.
Learn more about our in vivo programs.
Program
Research
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
NTLA-2001:
Transthyretin (ATTR) Amyloidosis
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Early-Stage
Clinical
Clinical

NTLA-2002:
Hereditary Angioedema
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Early-Stage
Clinical
Clinical

NTLA-2003:
AATD-Liver Disease
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IND-Enabling

NTLA-3001:
AATD-Lung Disease
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IND-Enabling

Hemophilia B
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IND-Enabling

Hemophilia A
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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 5, 2022

Ex Vivo
CRISPR creates the therapy
IMMUNO-ONCOLOGY / AUTOIMMUNE DISEASE
Learn more about our ex vivo programs.
Program
Research
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
OTQ923 / HIX763:
Sickle Cell Disease
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Early-Stage
Clinical
Clinical

NTLA-6001:
CD30+ Lymphomas
IND-Enabling

Acute Myeloid Leukemia / Solid Tumors
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Research

Research Programs
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Research

Research Programs
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Research

Other Novartis Programs
Undisclosed

*** Milestones & royalties
Last Updated: January 5, 2022