Achieving the Promise of Gene Therapy: New Pathways to Overcome Patient Enrollment and Safety Challenges

Life Sciences, Clinical Trials, Patient Recruitment & Retention, Drug Safety,
  • Monday, September 28, 2020

Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine.

This webinar will examine the evolving science of gene therapy, covering current approaches such as gene transfer and gene silencing, safety considerations, and delayed adverse events. It will describe the current (and ever-changing) regulatory landscape, identify opportunities for sponsors to more closely engage with regulators, and explore operational challenges such as patient recruitment and retention and use of sham medical procedures.

Other topics will include:

  • Understanding the disease burden and patients’ treatment goals
  • Designing studies that identify relevant endpoints and meet regulatory and safety requirements
  • Discussing unique case studies that address operational challenges in gene therapy trials



Allan Valmonte, MBA, Executive Director, Program Strategy - Rare Disease & Pediatrics, Premier Research

Allan Valmonte, MBA, is an Executive Director, Program Strategy – Rare Disease & Pediatrics at Premier Research. Valmonte has over 23 years of Clinical Operations and Program Management experience in a variety of therapeutic areas, most recently within the gene therapy space utilizing Lentiviral and AAV technology platforms on the sponsor side. Valmonte has shown a depth of experience when strategizing, operationalizing and conducting rare, pediatric, gene therapy clinical programs. Prior to joining Premier Research, Valmonte held positions of increasing responsibility in both clinical operations and program management at various sponsor companies.

Valmonte has an MBA with honors from Saint Joseph’s University in Philadelphia, PA and a BA in Psychology from California State University – Hayward, in Hayward, CA. Valmonte is currently a member of both the Beta Gamma Sigma (BGS) International Business Honor Society as well as the BGS Atlanta Alumni Chapter.  Valmonte is also an adjunct faculty at Saint Joseph’s University within the Haub School of Business.

Message Presenter

Mesaki Kenneth “Ken” Ndugga-Kabuye, MD, FACMG, Medical Director, Rare Disease/Pediatrics, Premier Research

Dr. Mesaki Kenneth “Ken” Ndugga-Kabuye, MD, FACMG is a Medical Director within the Rare Disease and Pediatric therapeutic area at Premier Research. Dr. Ndugga-Kabuye provides medical affairs and clinical development support to multiple functional areas at Premier Research. Dr. Ndugga-Kabuye’s training and expertise is inclusive of inborn errors of metabolism, adult-onset genetic conditions, general prenatal & pediatric genetics. He is a clinical geneticist with residency and fellowship training in medical genetics & genomics from the University of Washington in Seattle, WA, USA. Dr. Ndugga-Kabuye is board certified in Clinical Genetics and Genomics by the American Board of Medical Genetics and Genomics (ABMGG) and he is a Fellow of the American College of Medical Genetics and Genomics (ACMG). He holds a medical license in Washington State (MD.60883443).

Message Presenter

Naomi Kautz, Director, Regulatory Affairs, Regulatory Professionals

Naomi Kautz brings 20 years of multidisciplinary experience in biotechnology/pharmaceutical industry in Regulatory Affairs, Research and Development, and Policy after receiving her academic training in biochemistry. Her tenure in Regulatory Affairs has spanned early stage through late stage development and NDAs. Since 2009, her area of focus in Regulatory Affairs has been in the development of cell and gene therapy. In 2018, Kautz joined Regulatory Professionals, a regulatory consultancy firm that provides regulatory expertise to small and mid-sized biotechnology and pharmaceutical companies for both biologics and small molecule drug development programs. RPI is a division of Premier Research.

Message Presenter

Who Should Attend?

Managers and above at biotech and pharma companies with job functions including, but not limited to:

  • Clinical Operations
  • Medical Affairs
  • Project Management
  • Regulatory Affairs

What You Will Learn

Join this webinar to learn more about:

  • Understanding the disease burden and patients’ treatment goals
  • Designing studies that identify relevant endpoints and meet regulatory and safety requirements
  • Discussing unique case studies that address operational challenges in gene therapy trials

Xtalks Partner

Premier Research

Premier Research, a clinical research company, is dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments.

As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors.

Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.


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