Skip To Content

Gene Therapy Trials and Tribulations

1.25 credits


Full course description

The Georgia Clinical & Translational Science Alliance- Georgia CTSA and Southern California Clinical and Translational Science Institute -SC-CTSI have collaborated to provide free, high quality educational programs for clinical research professionals at novice to expert levels of experience. At the completion of each course or program, participants earn contact hours recognized by a certificate and/or badge.


A detailed overview of gene therapy focused on cell therapy, including its definition, clinical trial procedures and examples, and potential serious adverse events will be discussed.


  1. Definition of Gene Therapy
  2. Overview of Gene Therapy Procedure
  3. Gene Therapy to Correct Genetic Deficiency
  4. Gene Therapy to Activate the Immune System to Attack Cancer
  5. Coordination of Teams Required for Administration of Gene Modified Cells
  6. Adverse Events Monitoring Following Cell Administration
  7. Potential Serious Adverse Events Lessons from History
  8. Risk Assessment for Insertional Mutagenesis Now Required
  9. Additional Testing Required by FDA
  10. First Approval of Chimeric Antigen Receptor T (CAR-T) Cell Therapy
  11. New Directions in Gene Therapy


Paula Kaplan-Lefko, PhD is a Project Scientist in the Department of Medicine, Division of Hematology/Oncology at the David Geffen UCLA School of Medicine.She is a project manager/regulatory writer/regulatory coordinator managing all aspects of investigator-initiated adoptive cell transfer gene therapy clinical trials in oncology for Dr. Antoni Ribas.Her role includes regulatory submissions to federal agencies as well as local committees, writing clinical protocols, informed consent forms, case report forms, investigational new drug (IND) applications and recombinant DNA advisory committee (RAC) applications as well as summarizing and reporting serious adverse events (SAE) and adverse events (AE). Dr. Kaplan-Lefko received her Ph.D. in cancer biology focusing on hormonal carcinogenesis at Tufts University in Medford, MA.Her postdoctoral work at Baylor College of Medicine in Houston, TX focused on the role of the insulin-like growth factor (IGF) axis in the development of prostate cancer using transgenic mouse models.As a Principal Scientist at Amgen, Inc., Dr. Kaplan-Lefko led the preclinical pharmacology effort on multiple teams developing small molecules and protein therapeutics for oncology

Learner Level:Advanced


This symposium is designed for Clinical Research Coordinators, Project Managers, Monitors, Regulatory, Medical and Clinical Affairs Professionals or junior faculty with 3+ years of experience seeking to further develop clinical research conduct skills and principles.Clinical research professionals should have responsibilities that include clinical trial start up planning, mentoring and complex issues involving site management, study management, and sponsor/ CRO challenges.

Contact Hours:

Emory Nursing Professional Development Center (ENPDC) is accredited as a provider of nursing continuing professional development by the American Credentialing Center’s Commission on Accreditation. Attendees to this CNE activity will be awarded 1.25 contact hours by ENPDC. No conflict of interest has been found with the speaker for this CNE activity nor with the members of the planning committee.

Need Help with Registration? Please contact us at or 404-727-9208.

Program Information: This is the second course in a six (6) session program. To complete the entire program and earn a badge CLICK HERE.

Sign up for this course today!