Gene Therapy for Rare and Ultra-rare Diseases
Understanding Scientific and Ethical Considerations

A WCG | CenterWatch FREE Webinar
Wednesday, Feb. 28, 2018 · 1:30 p.m. - 2:30 p.m. EST

Many diseases categorized as rare or ultra-rare are caused by specific genetic defects.  This makes them solid candidates for the development of gene transfer therapies, including approaches that replace or correct defective or mutant genes. 

But conducting clinical research to determine the effectiveness of a specific therapy is particularly dicey in this space. Do you really understand the scientific and ethical considerations of conducting gene therapy clinical trials?

Not to worry, this webinar will systematically review the science of gene therapy and gene editing and provide examples of therapies currently in development for the treatment of rare diseases. The webinar also will discuss several ethical challenges inherent in rare disease clinical research specific to gene therapy research.

In just 60 minutes, from the comfort of your own office, you will gain a fuller understanding of the scientific and ethical considerations involved in gene therapy for rare diseases. So don’t spend thousands more dollars on attorneys, scientific experts or consultants. In fact, don’t spend any money at all because this webinar is free. Simply save the date Feb. 28, 2018 — which not coincidentally happens to be Rare Disease Day — to hear two of the leading authorities on rare disease safety explain the scientific and ethical issues surrounding gene therap. Here’s what you’ll learn during this interactive session:

  • Basic science of gene replacement therapies
  • Examples of therapies in development for rare diseases
  • Specific challenges in the use of gene therapy to treat rare diseases
  • Ethical issues in research involving patients with rare diseases

Our experts will provide you with a more complete and practical understanding of the science, terminology, potential and risks of gene therapies in the treatment of rare diseases. You’ll come away with an enhanced ability to recognize the upside and pitfalls of gene therapy for rare and ultra-rare diseases and an increased comprehension of ethical challenges in this area.

Take advantage of this free, timely presentation to learn the nuances of gene therapy for rare diseases. Sign up now.

Who Will Benefit

This session is appropriate for professionals who conduct research on gene therapy for clinical trials, including individuals holding these job titles:

  • Patients and patient advocacy representative
  • IRB members
  • IRB administrators
  • Clinical project manager
  • Clinical research associates
  • Clinical research team member
  • Biosafety professional

Sign Up for FREE Below

Meet Your Presenters


Lindsay McNair, MD, MPH, MSBioethics

Chief Medical Officer
WIRB-Copernicus Group (WCG)

Lindsay McNair, MD, MPH, MSBioethics, is the Chief Medical Officer for the WIRB-Copernicus Group (WCG). Prior to joining WCG, she was a consultant to multiple biopharma companies, providing medical guidance on clinical development strategies and study designs for new drug studies, and medical oversight of all phases of clinical trials. Before becoming a consultant, McNair was the medical lead for the telaprevir development program at Vertex Pharmaceuticals, with oversight of the Phase I-III studies. McNair is adjunct faculty at Boston University and teaches graduate courses on the scientific design of clinical research studies. She now oversees the physician team within the WCG IRBs and provides consultation to institutions and pharma/biotech companies on a wide range of issues related to protocol design, regulatory compliance, human subject protection and ethical policy development.


Daniel Kavanagh, PhD

Senior Director of Biosafety and Gene Therapy
WCG Biosafety, a division of the WIRB-Copernicus Group

Daniel Kavanagh, PhD, is Senior Director of Biosafety and Gene Therapy at WCG Biosafety, a division of the WIRB-Copernicus Group. Prior to joining WCG, he was Assistant Professor of Medicine at Harvard Medical School, Assistant Immunologist at the Massachusetts General Hospital and a principal investigator studying infectious diseases at the Ragon Institute of MGH, MIT and Harvard. While at Harvard Medical School, Kavanagh served as Vice Chair of the Partners Institutional Biosafety Committee, which oversees biosafety at two main Harvard teaching hospitals. He was co-chair of a Phase I clinical trial of an autologous mRNA-transfected dendritic cell vaccine in HIV+ subjects.