Hot Topic: Applications of qPCR/dPCR in Cell and Gene Therapies; Case Studies

Wednesday, May 15, 2024

10:00 AM – 11:00 AM PT

Location:Yosemite A

Hot Topic Moderator(s)

Jacqueline Murphy, MS

Principal Scientist
Janssen

With the remarkable growth in Cell therapies (CTx) and Gene therapies (GTx) in the field of pharmaceuticals, application of quantitative polymerase chain reaction (qPCR), digital polymerase chain reaction (dPCR) and reverse transcriptase PCR (RT-qPCR) in the assessment of biodistribution, vector shedding, transgene expression and cellular kinetics are on the rise. Target specificity and high sensitivity of PCR assays make them ideal tools for such evaluations in both preclinical and clinical stages of drug development. Harmonized recommendations for the conduct of nonclinical biodistribution and nonclinical as well as clinical vector shedding studies issued by the FDA, EMA, ICH, and other health authorities highly recommend the use of a PCR-based method in these studies. Similarly, reliable measurement of transgene expression can only be performed using RT-qPCR. This session will include multiple talks on the application PCR methods in drug development; specific biodistribution, vector shedding safety, transgene expression and cellular kinetics case studies will be presented.

Learning Objectives:

Upon completion of, participants will be able to learn about the utility and application of PCR-based method in drug cell and gene therapy drug development.
Upon completion of, participants will be able to understand the diversity of preclinical/clinical safety and quantitative assessments which can be achieved with PCR.
Upon completion of, participants will be able to learn about interpretation of PCR data

Presentations:

10:00 AM – 10:10 AM PT

Introduction to PCR Assay Development and Validation
Hot Topic Speaker: Timothy L. Lochmann, PhD (he/him/his) – PPD, part of Thermo Fisher Scientific
10:10 AM – 10:20 AM PT

A Case Study in AAV Gene Therapy Viral Shedding
Hot Topic Speaker: Mitra Azadeh, PhD (she/her/hers) – Ultragenyx
10:20 AM – 10:30 AM PT

A Case study in Cell Therapy Persistence by qPCR
Hot Topic Speaker: Kate B. Herr, M.S. (she/her/hers) – Johnson & Johnson
10:30 AM – 10:40 AM PT

A Case Study in Cell Therapy Persistence by ddPCR
Hot Topic Speaker: Nanda Balasubramanian, PhD (he/him/his) – Bristol-Myers Squibb Company
10:40 AM – 11:00 AM PT

Panel Discussion
Hot Topic Speaker: Mitra Azadeh, PhD (she/her/hers) – Ultragenyx
Hot Topic Speaker: Timothy L. Lochmann, PhD (he/him/his) – PPD, part of Thermo Fisher Scientific
Hot Topic Speaker: Nanda Balasubramanian, PhD (he/him/his) – Bristol-Myers Squibb Company
Hot Topic Speaker: Kate B. Herr, M.S. (she/her/hers) – Johnson & Johnson
Hot Topic Moderator: Jacqueline Murphy, MS – Janssen