On-Demand Webinar

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

Presented By

Dan Eisenman.png

Daniel Eisenman


Executive Director, Biosafety Services


Meagan Vaughn


Associate Clinical Director, Clinical Development
Krystal Biotech


Jennifer Abeles


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

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Who should watch?

Representatives from sponsors, CROs, and research sites involved in rare disease research, genetic engineering techniques, and DE&I in clinical research. Rare disease patients and patient advocates may also find this presentation informative.

CE Credits

The Society of Clinical Research Associates (SOCRA - www.SOCRA.org) accepts documentation of candidate participation in continuing education programs for recertification if the program is applicable to clinical research regulations, operations or management, or to the candidate’s clinical research therapeutic area. This program offers 1 hour of CE credit.

This event may qualify for continuing education (CE) credits. Please check with your professional organization(s) to determine whether the event meets their CE eligibility requirements.

Certificate of Attendance

After watching this webinar in its entirety and completing the webinar survey, you will receive a certificate of attendance. This certificate will be emailed to you within one business day of watching the webinar. Please note that if your organization has other staff members who wish to get a certificate of attendance, each person must register, watch the webinar in its entirety, and complete the webinar survey. Visit our Webinar Help page for more information on certificates of attendance.

DATE: June 17, 2021
Time: 1 pm ET | 10 am PT
Duration: 1 hour

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