NCT06622668WITHDRAWNanonymous

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

Sponsor

Source record

Intellia Therapeutics

Phase

Source record

Phase 1/2

Modality

AI-normalized

protein therapy

Target

AI-normalized

Alpha-1 Antitrypsin Deficiency (AATD) - associated lung disease, utilizing AAV and CRISPR/Cas9 gene editing technology.

Indication / condition

AI-normalized

Lung Disease

Intervention

Source record

Biological NTLA-3001

Source & freshness

Source record

NCT ID

NCT06622668

Original source

ClinicalTrials.gov

Source last updated

Jan 17, 2025

Ingested at

Jun 13, 2026

Internal sync

Jun 13, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

Open original registry record

View original source fields

NCT ID

NCT06622668

Title

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

Sponsor

Intellia Therapeutics

Status

WITHDRAWN

Phase

Phase 1/2

Condition raw

Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease

Condition normalized

Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease

Modality raw

protein therapy

Modality normalized

protein therapy

Target raw

Alpha-1 Antitrypsin Deficiency (AATD) - associated lung disease, utilizing AAV and CRISPR/Cas9 gene editing technology.

Target normalized

Alpha-1 Antitrypsin Deficiency (AATD) - associated lung disease, utilizing AAV and CRISPR/Cas9 gene editing technology.

Interventions

Biological NTLA-3001

Public preview

Source record

NTLA-3001, developed by Intellia Therapeutics, targets a niche yet significant patient population suffering from AATD-associated lung disease. The market for AATD therapies is currently underserved, with limited treatment options available. The withdrawal of this trial indicates a strategic shift by Intellia towards prioritizing other programs, which may reflect resource allocation challenges or a reassessment of the commercial viability of NTLA-3001. Competitively, the gene editing landscape is rapidly evolving, and Intellia's focus on AAV and CRISPR technologies positions it well, but the withdrawal may raise concerns regarding the asset's future development and market entry.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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TrialSignal

Clinical trial intelligence report

Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease

Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.

Generated

Jun 16, 2026

NCT ID

NCT06622668

Status

WITHDRAWN

Phase

Phase 1/2

Sponsor

Intellia Therapeutics

Executive brief

Investment-Ready Snapshot

Source & freshness

Provenance

SourceClinicalTrials.gov

Source updatedJan 17, 2025

Internal syncJun 13, 2026

Model versiontrialsignal-ai-v1

https://clinicaltrials.gov/study/NCT06622668

Indication

Lung Disease

Modality

protein therapy

Target

Alpha-1 Antitrypsin Deficiency (AATD) - associated lung disease, utilizing AAV and CRISPR/Cas9 gene editing technology.

Intervention

Biological NTLA-3001

Source record