The Current State of Gene Therapy Trials

Date: Wednesday, April 10, 2019
Time: 10:00 AM Eastern Daylight Time
Duration: 1 hour

While the promise of gene therapy has the attention of everyone in healthcare, significant hurdles remain. The journey from patient diagnosis to randomization and long-term retention requires a deep and ongoing knowledge of regulatory frameworks, location of patients and sites skilled to diagnose them, and management of lab samples. Innovative and creative biotechs and CROs have been working together to identify solutions. In this webinar, our experts will address:

  • Creating an optimal patient pathway, addressing challenges of diagnosis and site training, and long-term retention factors
  • Navigating an evolving global regulatory landscape
  • Laboratory logistics and novel bioanalytical strategies supporting primary endpoints including pharmacokinetics, immunogenicity and safety and efficacy driven biomarkers

Speakers

Timothy M. Miller, M.D. Timothy M. Miller, M.D.
Vice President and Therapeutic Area Head, Pediatrics & Rare Diseases
PPD

Dr. Miller leads PPD’s pediatrics and rare disease functions. He joined PPD in 2018 with more than 20 years of healthcare experience in academic medical centers and the pharmaceutical and biotech industries. His research interests encompass neurologic and orphan diseases, genetic and neuromuscular disorders, and inflammatory/immune-mediated diseases of the nervous system. As a fellowship-trained neuromuscular neurologist, Dr. Miller’s work weaves together deep clinical knowledge, novel science, and an understanding of required field actions to advance the discovery of and access to therapies, addressing the needs of patients living with rare and debilitating diseases.


Patricia Hurley, Ph.D. Patricia Hurley, Ph.D.
Senior Director
PPD Consulting

As a senior director in PPD’s consulting group, Patricia Hurley, Ph.D., provides product development and global strategic regulatory advice to external and internal clients, determining the most appropriate strategy for their projects. She acts as a point of contact to provide regulatory and product development consulting advice and expertise to advanced therapy developers in a partnership setting and is a founder and leader of PPD’s advanced therapy cross-functional expert working group.

Since joining PPD in 2010 as a manager in global regulatory development, Dr. Hurley has successfully supported many clients with global clinical trial authorization applications, marketing authorization applications and strategic consulting discussions in several disease areas, including rare diseases. She has taken part in multiple regulatory agency interactions and scientific advice meetings with clients to discuss their cell and gene therapy product development.

She is based in Cambridge, UK, and belongs to The Organization for Professionals in Regulatory Affairs (TOPRA) and sits on the editorial board of Regulatory Rapporteur. Prior to joining PPD, Dr. Hurley was head of project management and biomarkers division at a London-based regulatory and product development consulting group where she undertook the full range of global regulatory activities.

Dr. Hurley has a doctorate in molecular pharmacology and a joint honors bachelor’s degree in pharmacology and molecular genetics from University College Dublin, Ireland.


Heather Myler, Ph.D. Heather Myler, Ph.D.
Director
PPD Laboratories

Heather Myler, Ph.D., serves as the director of R&D, immunochemistry at PPD Laboratories, leading a group of scientists responsible for regulated PK, ADA, neutralizing antibody and biomarker method development, validation and automation supporting gene and cell therapies, biosimilars and novel biologics. Heather has a strong external scientific presence, currently serving as co-leader of the Bioanalytical Community within AAPS and actively participating on multiple bioanalytical and cross-functional initiatives. Heather is currently leading a team focused on the harmonization of ADA validation testing and reporting, comprised of over 30 KOL from across industry and from various health authority agencies with a strong FDA presence.

Heather received her Ph.D. in biochemistry and cell biology at Rice University and completed her post-doctoral training at Pfizer in drug safety evaluation, later holding positions of increasing responsibility at Amgen, Ambrx, Merck and BMS where she has contributed to the growth of the bioanalytical discipline, supporting PK, immunogenicity and biomarker bioanalysis with an expertise in biologics.


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