Events

February 2020

May 2020

July 2020

February 2020

Rare Disease Day at NIH

Feb. 28, 2020
8:30 a.m. to 4:30 p.m. ET

National Institutes of Health (Main Campus)
Natcher Conference Center (Bldg. 45)
45 Center Drive
Bethesda, Maryland 20892

Sponsored by the National Center for Advancing Translational Sciences (NCATS) and Clinical Center (CC) at the National Institutes of Health (NIH), Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH research collaborations under way to address scientific challenges and to advance new treatments.

Admission is free, and the event is open to the public, including patients, patient advocates, health care providers, researchers, industry representatives and government employees. In association with Global Genes®, participants are encouraged to wear their favorite pair of jeans.

RegistrationVideocast

May 2020

NCATS Advisory Council and CAN Review Board Meeting

May 14, 2020

National Institutes of Health (Main Campus)
Bethesda, Maryland 20892

The NCATS Advisory Council and Cures Acceleration Network Review Board will convene a joint meeting on May 14, 2020, on the NIH campus in Bethesda, Maryland. The meeting will feature presentations by NCATS leadership and invited guests. For more information about the Advisory Council and CAN Review Board, visit the NCATS Advisory Council page and the Cures Acceleration Network Review Board page of this site.

July 2020

Workshop on Systemic Immunogenicity Considerations for AAV-Mediated Gene Therapy

Save the Date!
July 7–8, 2020
Day 1: 8:30 a.m. to 5 p.m. ET
Day 2: 8:30 a.m. to 4:30 p.m. ET

National Institutes of Health (Main Campus)
Natcher Conference Center (Bldg. 45), Main Auditorium
45 Center Drive
Bethesda, Maryland 20892

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA) are co-hosting a Workshop on Systemic Immunogenicity Considerations for Adeno-Associated Virus (AAV)-Mediated Gene Therapy. This meeting will bring together scientists, therapeutic developers and patient representatives to focus on immunogenicity issues related to the systemic administration of AAV gene therapy products and identify solutions for those issues.

Goals for this meeting are to—

  • Exchange lessons learned from AAV gene therapy clinical trials to date, including clinical manifestations of immunogenicity, methods for evaluating the risk of immune responses, effective immunosuppressive protocols and other clinical topics.
  • Explore emerging methods and technologies for modulating immune responses to AAV gene therapies with respect to both reducing the immunogenicity of AAV vector molecules and tolerizing strategies.
  • Review basic, pre-clinical and clinical knowledge gaps, including the adequacy of current in-silico algorithms, pre-clinical models, assays and knowledge-sharing mechanisms to address those gaps.
  • Identify and explore potential solutions.

This listing will be updated as more information becomes available.