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Intellia Therapeutics, Inc. (NTLA)

Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Sector: In Vitro & In Vivo Diagnostic Substances

Industry: Biotechnology

Intellia Therapeutics, Inc. logo

Intellia Therapeutics, Inc.

NASDAQ:NTLAHealthcare
-5.74%$12.47
AH$12.35-0.96%vs close
Mkt Cap$1.75B
Float115.06M
O/S140.13M
Vol
$ Vol
RVOL
Inst Own-
Earnings
Dilution
Intellia Therapeutics, Inc. logoNASDAQ:NTLA$12.47-5.74%
Phase Distribution
9 lead-sponsored trials·6 active·1 completed
Phase 11
Phase 1/23
Phase 33
Not applicable2
(1 more as collaborator)
Status Overview
Recruiting4
Active, not recruiting2
Completed1
Stopped2

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Recruiting

    MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

    NTLA-2001, Placebo

    Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

    Enrollment
    1,200
    Start
    Dec 2023
    Primary Completion
    Dec 2027est.
    Completion
    Apr 2028est.
    NCT06128629
  • Phase 3
    Active, not recruiting

    HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)

    NTLA-2002, Normal Saline IV Administration

    Hereditary Angioedema

    Enrollment
    80
    Start
    Jan 2025
    Primary Completion
    Feb 2026
    Completion
    Sep 2027est.
    NCT06634420
  • Phase 3
    Recruiting

    A Phase 3 Study of NTLA-2001 in ATTRv-PN

    nexiguran ziclumeran, Normal Saline as Placebo

    Neuromuscular Disease, Neuromuscular Diseases (NMD) +18

    Enrollment
    60
    Start
    Nov 2024
    Primary Completion
    Jul 2027est.
    Completion
    Aug 2028est.
    NCT06672237
  • Phase 1/2
    Terminated

    Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

    Arm 1: NTLA-5001, Arm 2: NTLA-5001

    Acute Myeloid Leukemia

    Enrollment
    6
    Start
    Dec 2021
    Primary Completion
    Jul 2022
    Completion
    Aug 2022
    NCT05066165
  • Phase 1/2
    Active, not recruiting

    NTLA-2002 in Adults With Hereditary Angioedema (HAE)

    Biological NTLA-2002

    Hereditary Angioedema

    Enrollment
    37
    Start
    Dec 2021
    Primary Completion
    Apr 2024
    Completion
    May 2027est.
    NCT05120830
  • Phase 1/2
    Withdrawn

    NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

    Biological NTLA-3001

    Lung Disease, Pulmonary Disease +3

    Enrollment
    0
    Start
    Nov 2024
    Primary Completion
    Jan 2025est.
    Completion
    Jan 2025
    NCT06622668
  • Phase 1
    Completed

    Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

    NTLA-2001

    Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy +1

    Enrollment
    72
    Start
    Nov 2020
    Primary Completion
    Sep 2025
    Completion
    Sep 2025
    NCT04601051
  • Observational
    Enrolling by invitation

    Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

    -

    Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy +1

    Enrollment
    72
    Start
    Jul 2023
    Primary Completion
    Mar 2038est.
    Completion
    Mar 2038est.
    NCT05697861
  • Observational
    Enrolling by invitation

    Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

    -

    Hereditary Angioedema

    Enrollment
    100
    Start
    Apr 2024
    Primary Completion
    Apr 2040est.
    Completion
    Apr 2040est.
    NCT06262399
Search ClinicalTrials.gov for “Intellia Therapeutics”