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1. Animal research will make gene therapy happen

Publication:

[Bethesda, Md.] : NIH Animal Research Advisory, [19--]

Subject(s):

Animal Experimentation
Animals, Laboratory
Genetic Therapy

2. Betibeglogene autotemcel for beta thalassemia: effectiveness and value : final evidence report

Author(s):

Beaudoin, Francesca L., author
Richardson, Marina, author
Synnott, Patricia G., author
Lancaster, Victoria, author
Fluetsch, Noemi, author
Herce-Hagiwara, Belén, author
Campbell, Jon D., author
Pearson, Steven D., author
Rind, David M., author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, July 19, 2022

Subject(s):

beta-Thalassemia -- therapy
Biological Products -- therapeutic use
Genetic Therapy
United States

3. Chemistry, manufacturing, and control (CMC) information for human gene therapy investigational new drug applications (INDs)

Publication:

Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020

Subject(s):

Chemistry, Pharmaceutical
Genetic Therapy
Investigational New Drug Application
Drugs, Investigational
Quality Control
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

4. Gene therapies for sickle cell disease: final evidence report

Author(s):

Beaudoin, Francesca, author
Nikitin, Dmitriy, author
Campbell, Jonathan D., author
McKenna, Avery, author
Pearson, Steven D., author
Rind, David M., author
Thokala, P. (Praveen), author
Spackman, David Eldon, author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, August 21, 2023

Subject(s):

Anemia, Sickle Cell -- therapy
Genetic Therapy
United States

5. Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report

Author(s):

Tice, Jeffrey A., author
Chu, Janet N., author
Moradi, Ashton, author
Herce-Hagiwara, Belen, author
Fahim, Shahariar Mohammed, author
Agboola, Foluso, author
Pearson, Steven D., author
Rind, David M., author
Walton, Surrey M., author
Sarker, Jyotirmoy, author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, December 22, 2022

Subject(s):

Antibodies, Bispecific
Antibodies, Monoclonal, Humanized
Cost-Effectiveness Analysis
Genetic Therapy
Hemophilia A -- therapy
Hemophilia B -- therapy
United States

6. Human gene therapy

Publication:

[Bethesda, Md. : Medical Arts and Photography Branch, National Institutes of Health, 1992]

Subject(s):

Genetic Therapy

7. 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

8. 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

9. 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

10. Interpreting sameness of gene therapy products under the orphan drug regulations

Publication:

Silver Spring, MD : Center for Biologics Evaluation and Research, September 2021

Subject(s):

Genetic Therapy
Orphan Drug Production -- legislation & jurisprudence
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration