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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