How Genetic Engineering Will Move Rare Disease Research at Warp Speed

With the potential for single curative treatments, gene therapy offers great potential for rare diseases lacking treatment options. FDA’s recently launched Warp Speed for Rare Diseases seeks to accelerate research and development of such therapies.

In this webinar, industry experts discuss different types of gene delivery and why some conditions may be “easier” to target with gene therapy. They also explain the potential impact on diverse populations in the U.S.

Learning Objectives:

• Describe how the recent growth in gene therapy research applies to rare diseases
• Explain why some diseases are better candidates for gene therapy
• Summarize the potential impact of rare disease gene therapies on diverse patient populations

• Presenters

Daniel Eisenman

PhD, RBP, SM(NRCM), CBSP

Executive Director, Biosafety Services, Advarra

Meagan Vaughn

PhD, RAC

Associate Clinical Director, Clinical Development at Krystal Biotech

Jennifer Abeles

DO

Assistant Clinical Professor, Internal Medicine and Pediatrics
Conventus Site Medical Director for UBMD
Adult Transitional Sickle Cell Program Director Department of Medicine
Jacobs School of Medicine and Biomedical Sciences University of Buffalo