Subjects: Genetic Therapy and Clinical Trials as Topic - Digital Collections - National Library of Medicine Search Results

Start Over Subjects Genetic Therapy ✖ Subjects Clinical Trials as Topic ✖

1. Studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial: guidance for industry

Publication:: Silver Spring, MD : Center for Biologics Evaluation and Research, November 2022
Subject(s):: Cell- and Tissue-Based Therapy
Clinical Trials as Topic
Genetic Therapy
Government Regulation
United States
United States. Food and Drug Administration

2. Human gene therapy for retinal disorders

Publication:: Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020
Subject(s):: Clinical Trials as Topic
Genetic Therapy
Drug Evaluation, Preclinical
Retinal Diseases -- therapy
Investigational New Drug Application
United States
United States. Department of Health and Human Services.
United States. Food and Drug Administration.

3. Human gene therapy for rare diseases

Publication:: Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020
Subject(s):: Drug Development
Genetic Therapy
Rare Diseases -- therapy
Clinical Trials as Topic
Drug Evaluation, Preclinical
United States
United States. Department of Health and Human Services.
United States. Food and Drug Administration.

4. Human gene therapy for hemophilia

Publication:: Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020
Subject(s):: Genetic Therapy
Hemophilia A -- drug therapy
Hemophilia B -- drug therapy
Clinical Trials as Topic
Drug Evaluation, Preclinical
Factor VIII -- therapeutic use
Factor IX -- therapeutic use
United States
United States. Department of Health and Human Services.
United States. Food and Drug Administration.