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

Program
Research
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
NTLA-2001:
Transthyretin (ATTR) Amyloidosis
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Early-Stage
Clinical
NTLA-2002:
Hereditary Angioedema
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Early-Stage
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: February 24, 2022
Program
Research
IND-Enabling
Early-Stage Clinical
Late-Stage Clinical
Partner
OTQ923 / HIX763:
Sickle Cell Disease
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Early-Stage
Clinical
NTLA-5001:
Acute Myeloid Leukemia
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Early-Stage
Clinical
NTLA-6001:
CD30+ Lymphomas
IND-Enabling
Solid Tumors
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Research
Allo Undisclosed
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Research
Research Programs
Research
Other Novartis Programs
Undisclosed

*** Milestones & royalties

Last Updated: February 24, 2022