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Clinical trials
catalyst calendar across sponsors →Studies where NTLA is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
7 interventional · 2 observational · 1 with posted results
Held by 12 tracked-famous managers (D. E. Shaw & Co., Inc., TWO SIGMA INVESTMENTS, LP, RENAISSANCE TECHNOLOGIES LLC, SUSQUEHANNA INTERNATIONAL GROUP, LLP, AQR CAPITAL MANAGEMENT LLC, +7 more) · FINRA short interest 12.0 days to cover (settled 2026-07-31) · government filings 180d: none disclosed
| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002NCT06262399observationalIncidence of treatment-related Adverse Events (AEs); incidence of treatment-related Serious Adverse Events (SAEs); incidence of treatment-related Adverse Events of Special Interest (AESIs) defined per protocol | — | Enrolling by invitation | — | Hereditary Angioedema | 100 | Apr 2040 | ≤ 5,003 d | 2025-11-28 |
| Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001NCT05697861observationalIncidence of Treatment-related SAEs and Protocol-specified AESIs | — | Enrolling by invitation | — | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis | 72 | Mar 2038 | ≤ 4,242 d | 2026-08-03 |
| MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)NCT06128629Composite outcome of cardiovascular (CV) mortality and CV eventsRandomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Biological: NTLA-2001 · Drug: Placebo | Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | 1,200 | Dec 2027 | ≤ 499 d | 2026-03-27 |
| A Phase 3 Study of NTLA-2001 in ATTRv-PNNCT06672237Modified Neuropathy Impairment Score +7 (mNIS+7)Randomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Biological: nexiguran ziclumeran · Drug: Normal Saline as Placebo | Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Neuromuscular Diseases, Nerve Disorders, Nervous System Disease, Nervous System Diseases, Genetic Disease, Inborn, Amyloidosis, Familial, Amyloidosis, Hereditary, Amyloidosis, Polyneuropathies, Amyloid Neuropathies, Amyloid Neuropathies, Familial, Peripheral Nervous System Disease, Peripheral Nervous System Diseases, Metabolism, Inborn Errors, Metabolic Diseases | 60 | Jul 2027 | ≤ 346 d | 2026-04-16 |
| HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)NCT06634420Time-normalized number of Investigator-confirmed HAE attacksRandomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Biological: NTLA-2002, Normal Saline IV Administration | Hereditary Angioedema | 80 | 2026-02-10actual | — | 2026-07-29 |
| NTLA-2002 in Adults With Hereditary Angioedema (HAE)NCT05120830Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)Randomized · Double-masked · Treatment | Phase 1/2 | Active, not recruiting | Biological: Biological NTLA-2002 · Other: Normal Saline IV Administration | Hereditary Angioedema | 37 | 2024-04-04actual | — | 2026-03-24 |
| 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)NCT04601051Number of Participants with Treatment-Emergent Adverse EventsNon-randomized · Open-label · Treatment | Phase 1 | Completed | Biological: NTLA-2001 | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis | 72 | 2025-09-12actual | — | 2026-01-30 |
| NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung DiseaseNCT06622668Safety and TolerabilityOpen-label · Treatment | Phase 1/2 | Withdrawnsponsor's stated reason: Clinical trial ITL-3001-CL-101 for the investigational product NTLA-3001 was withdrawn due to prioritization of other Sponsor programs. | Biological: Biological NTLA-3001 | Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease | 0 | 2025-01-09 | — | 2025-01-17 |
| Study Investigating NTLA-5001 in Subjects With Acute Myeloid LeukemiaNCT05066165results2023-12-28Participants That Experienced Dose-limiting Toxicities (DLTs)Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Terminatedsponsor's stated reason: Pivoting to an allogeneic version of this program currently in preclinical development. | Genetic: Arm 1: NTLA-5001, Arm 2: NTLA-5001 | Acute Myeloid Leukemia | 6 | 2022-07-21actual | — | 2023-12-28 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19