Demystify Your Disease’s R&D Readiness

Genetic Therapies

Gene therapy, genome editing, RNA-based therapy, and cell therapy are several of the promising therapies making headlines in the popular press. These advanced biologic products have the potential to treat, and in some cases cure, many types of rare diseases and cancer. However, it can be hard to know if any have the possibility to treat your disease. You may wish to gain a basic understanding of these intriguing advances and then discuss their relevance to your disease with your Medical/Scientific Advisory Board and possibly experts in the respective therapy field.

Resources

Research Tools
How Biomarkers Can Improve the Drug Development Process U. S. Food and Drug Administration (FDA) (link)
Rare Diseases Registry Program (RaDaR) National Center for Advancing Translational Sciences (NCATS) (link)
Genetic Therapies
What is Gene Therapy? U. S. Food and Drug Administration (FDA) (link)
Human Gene Therapy for Rare Diseases U. S. Food and Drug Administration (FDA) (link)
Gene Therapy Yesterday, Today and Tomorrow National Organization for Rare Disorders (NORD) (link)
The Science Behind Gene Therapy National Organization for Rare Disorders (NORD) (link)
The FDA's Role in Gene Therapy National Organization for Rare Disorders (NORD) (link)
Understanding the Gene Therapy Process and Aftercare National Organization for Rare Disorders (NORD) (link)
Life After Gene Therapy National Organization for Rare Disorders (NORD) (link)
What is genome editing? National Human Genome Research Institute (NHGRI) (link)
What is CRISPR-Cas9? Wellcome Genome Campus YourGenome (link)
Somatic Cell Genome Editing National Center for Advancing Translational Sciences (NCATS) (link)
Finding Disease and Technology Info
PubMed National Library of Medicine (NLM) (link)
ClinicalTrials.gov National Library of Medicine (NLM) (link)
Institutional Review Boards Frequently Asked Questions U.S. Food and Drug Administration (FDA) (link)
Research Portfolio Online Reporting Tool (RePORTER) National Institutes of Health (NIH) (link)
List of Registries National Institutes of Health Clinical Center (link)
Keeping Up with Advances
Free NIH Email Updates National Institutes of Health (NIH) (link)
News & Events for Human Drugs U. S. Food and Drug Administration (FDA) (link)
Preclinical Innovation National Center for Advancing Translational Sciences (NCATS) (link)
Core Technologies National Center for Advancing Translational Sciences (NCATS) (link)
Monthly NCATS e-newsletter National Center for Advancing Translational Sciences (NCATS) (link)
Receive NCATS Announcements National Center for Advancing Translational Sciences (NCATS) (link)
Accomplishments in Genomic Medicine National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
2019 Genomic Medicine Year in Review National Human Genome Research Institute (NHGRI) Genomic Medicine Working Group (GMWG) (link)
Genomics and Precision Health Weekly Update Centers for Disease Control and Prevention (CDC) (link)
PhRMA: Scientific Discoveries Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
PhRMA: Research and Development Pharmaceutical Research and Manufacturers of America (PhRMA) (link)
Sign Up for NORD Email Updates National Organization for Rare Disorders (NORD) (link)
Rare Disease Scientific Workshop EveryLife Foundation for Rare Diseases (link)
OrphaNews Orphanet (link)