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10,906 results for “NCF1 gene”

Trials

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Enrolling By Invitation

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

12 participants · First posted Aug 19, 2024

Open-label Gene Therapy Study in p47-CGD

5 participants · First posted Sep 17, 2026

A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease

31 participants · First posted Jun 10, 2025

Drugs

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TTR gene editing therapy (Trucode Gene)

CYBB gene editing therapy (Trucode Gene)

PAH gene editing therapy (Trucode Gene)

Organizations

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JIUYUAN GENE

Triple-Gene LLC

United States

MOMOTARO-GENE INC.

Japan

News

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World's First Gene Therapy for P47 Chronic Granulomatous Disease Shows Success in Teenage Patient

Prime Medicine Advances Gene Editing Pipeline with Key Programs for CGD, Wilson's Disease, and AATD

Prime Medicine Reports Breakthrough Clinical Data for First Prime Editing Therapy in Chronic Granulomatous Disease

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