Cell and Gene Therapy: Transforming Treatment Paradigms for Patient-Centric Care

Session Description

Cell and gene therapies (CGT) have brought a paradigm shift in drug development. CGT offers the promise of treating, and, in some cases, potentially curing diseases that have no therapeutic options or where only symptomatic treatment was traditionally available. Given its potential to address unmet medical needs, CGT area has been rapidly growing and evolving. As of the end of 2023, 76 cell and gene therapies have been launched globally including 19 products have been FDA-approved for treatment of 27 indications. Over the last five years, 3,285 clinical trials have been started to evaluate cell and gene therapies.

However, the complex issues surrounding patient eligibility, clinical uncertainties, and treatment accessibility pose significant barriers to the success in further expansion and sustainability of the field of CGT. It highlights the need for a deeper understanding of the current state and potential evolutionary paths over the coming years, which will be opportunities for clinical pharmacology and translational science leveraging novel quantitative pharmacology and biomarkers. Quantitative methods such as quantitative systems pharmacology (QSP) and exposure‑response relationship analyses, in combination with traditional population pharmacokinetic analyses, can provide guidance on dosing strategies to mitigate adverse events, while maximizing efficacy in CGT.

In this virtual Satellite Session, patient advocates, academia, industry, and regulators will discuss the state of art in CGT, hurdles in patient accessibility and eligibility of CGT, scientific challenges in CGT development and regulation. Case study presentations will demonstrate how translational science and clinical pharmacology can be leveraged in CGT clinical development and decision-making.

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Learning Objectives

1. Explore current challenges and future opportunities in the field of cell and gene therapies from diverse perspectives, including those of patients, healthcare professionals, industry stakeholders, and regulatory agencies.
2. Engage in discussions on how to harness the potential of clinical pharmacology and translational science to streamline and optimize the development of cell and gene therapies.
3. Gain a comprehensive understanding of cell and gene therapies and their unique pharmacological characteristics.

Session Schedule

Opening & Welcome
Matthew Rizk, PhD

Current Status and Future Perspective of Gene Therapy
Fyodor Urnov, PhD

Closing the Gap: Patient Perspectives in Gene Therapy Development and Beyond
Maria Santaella, PhD, RN

Audience Q&A

Clinical Development of AAV Gene Therapies: Learning, Challenges, and Opportunities
Tara Robinson, MD, PhD

Clinical Pharmacology Perspectives of AAV Gene Therapy
Michael Liao, PhD

Model-Informed Drug Development of Gene Therapy
Avery McIntosh, PhD

Panel Discussion and Q&A

Overview of T-cell Immune Therapy & Use of Biomarkers
Chia-Yung Wu, PhD

Clinical Pharmacology Considerations for Cell Therapies: Focus on Allogeneic Therapies for Autoimmune Diseases
Dale Miles, PhD, MS

Model-Informed Drug Development of CAR-T Therapy
John Burke, PhD

Panel Discussion and Q&A

Closing Remarks