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1. 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

2. 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

3. 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

4. Testing of retroviral vector-based human gene therapy products for replication competent retrovirus during product manufacture and patient follow-up

Publication:

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

Subject(s):

Drug Contamination
Genetic Therapy
Genetic Vectors
Retroviridae
Virus Replication
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

5. 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

6. 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

7. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value : final report

Author(s):

Rind, David M., author
Walton, Surrey M., author
Agboola, Foluso, author
Herron-Smith, Serina, author
Quach, Danny, author
Chapman, Rick M., author
Pearson, Steven D., author
Bradt, Pamela, author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, November 20, 2020

Subject(s):

Antibodies, Monoclonal
Genetic Therapy
Hemophilia A -- drug therapy
United States

8. Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report

Author(s):

Ellis, Alexandra, author
Mickle, Kristin, author
Herron-Smith, Serina, author
Kumar, Varun M., author
Cianciolo, Laura, author
Seidner, Matt, author
Rind, David, author
Pearson, Steven, author
Thokala, P. (Praveen), author
Stevenson, M. (Matt), author

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, May 24, 2019

Subject(s):

Genetic Therapy
Muscular Atrophy, Spinal -- drug therapy
United States

9. Voretigene neparvovec for biallelic RPE65-mediated retinal disease: effectiveness and value : final evidence report

Publication:

[Boston, Massachusetts] : Institute for Clinical and Economic Review, February 14, 2018

Subject(s):

Genetic Therapy
Leber Congenital Amaurosis -- genetics
Leber Congenital Amaurosis -- therapy
Retinal Diseases -- genetics
Retinal Diseases -- therapy
Retinal Pigment Epithelium -- abnormalities
United States

10. S12 nonclinical biodistribution considerations for gene therapy products: guidance for industry

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, May 2023

Subject(s):

Genetic Therapy
Government Regulation
Research Design
United States

11. 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

12. 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

13. 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

14. 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

15. Letter from Bernard D. Davis to Maxine Singer

Publication:

Produced: 10 March 1992

Subject(s):

Scientific Misconduct
Genetic Therapy

Archival Collection:

The Maxine Singer Papers (Profiles in Science)

16. The National Institutes of Health presents a conference on human gene therapy: December 4-6, 1991, Masur Auditorium, National Institutes of Health, Bethesda, Maryland

Publication:

Bethesda, Md. : National Heart, Lung, and Blood Institute, 1991

Subject(s):

Genetic Therapy

17. Human gene therapy

Publication:

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

Subject(s):

Genetic Therapy

18. Animal research will make gene therapy happen

Publication:

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

Subject(s):

Animal Experimentation
Animals, Laboratory
Genetic Therapy