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Wednesday, June 27 • 2:00pm - 3:15pm
#366: Gene Therapy Clinical Trials: Current Challenges

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Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-663-L04-P; CME 1.25; IACET 1.25; RN 1.25

Gene therapies and genetically modified organisms face special regulatory challenges for the conduct of clinical trials. This is particularly true for global trials that face a myriad of approval requirements in addition to the regulatory agency and ethics committee approvals. While some countries have a well defined regulatory framework addressing these products, other countries do not and the requirements may be less clear. With an increased emphasis on conducting global clinical trials the investigation of the diverse additional requirements becomes paramount for developing a time- and cost-effective regulatory strategy. The session will provide an overview of requirements for some global countries from an industry professional.

Learning Objectives

Recognize that gene therapies require special considerations and approvals in addition to those from the regulatory and ethical perspective; Analyze all required approvals that are critical for devising a global regulatory strategy; Apply a sound regulatory strategy for gene therapy products which is required for cost and time efficient trial approvals.


Kirsten Messmer


Key Regulatory Requirements and Challenges: EU/US/Canada
Janet C. Rae, RAC

Successfully Operationalizing Gene Therapy Clinical Trials
Venkata Jaggumantri


Venkata Jaggumantri

Clinical Scientist, PRA Health Sciences
Venkata Jaggumantri has over 11 years of clinical research experience both in the industry and academia. At PRA, he provides consulting and therapeutic expertise to assist in the design and conduct of rare disease clinical development programs. Mr. Jaggumantri is a proponent of precision... Read More →
avatar for Kirsten Messmer

Kirsten Messmer

Principal Regulatory Affairs Specialist, PPD
Kirsten Messmer, PhD, RAC, is a Principal Regulatory Affairs Specialist in the Regulatory Intelligence Solutions team at PPD which provides regulatory intelligence to clients and departments within PPD to support drug development and clinical research. Prior to joining PPD she completed... Read More →

Janet Rae

Vice President, Global Head, Gene Therapy Regulatory Affairs, Ultragenyx
Janet Rae has over 25 years regulatory affairs experience in biotechnology and pharmaceutical development, focusing on rare diseases including 3 years in the gene therapy space. At Ultragenyx, she leads the development and execution of regulatory strategy including direct interface... Read More →

Wednesday June 27, 2018 2:00pm - 3:15pm EDT
Room 156ABC Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  06: PreClin Dev-EarlyPhaseCR, Session