AGENDA - Specialized Session F2
June 26, 2020, 11am to 4pm ET: Part 1
June 29, 2020, 11am to 4pm ET: Part 2

"Cell & Gene Therapy Bioanalysis: Recent Advancements and New Frontiers for Bioanalytical Labs on Flow Cytometry, qPCR, ELISPOT, Regulatory Requirements, CAR-T and CRISPR Assays Case Studies"

Catching up with “explosion” of recent developments Cell & Gene Therapy Bioanalysis with specific focus on Flow Cytometry, qPCR, ELISPOT and Regulatory Requirements. Ongoing research in CAR-T and CRISPR Bioanalytical, Biodistribution Biomarkers and Immunogenicity Case Studies. Strategies to be prepared to handle the increasing demand of drug discovery/development bioanalytical data in support of ongoing and future research

Cell & Gene Therapy Bioanalysis Part 1: June 26, 2020 (EDT time listed)

11:00am-11:10am: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
"Introduction of Cell & Gene Therapy Bioanalysis Part 1"

Recent Advancements in Gene & Cell Therapy and Building on 2019 WRIB Recommendations

Chair: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
11:10am-11:30am: Dr. Mark Milton, Executive Director Pharmacokinetic Sciences Ophthalmology and Department Lead Gene Therapies, Novartis
"Gene & Cell Therapy Bioanalysis: Building on 2019 WRIB Recommendations and Updates on Gene & Cell Therapy Advancements"
11:30am-11:50am: Ms. Anna Ma, Associate Director Translational Pharmacology, CRISPR Therapeutics
Dr. Heather Hirsch, Sr. Director Translational Pharmacology and Head Clinical Biomarkers, CRISPR
"Next Generation Approaches for Tracking and Evaluating Gene/Cell Therapy: Looking beyond qPCR and Flow Cytometry"

Flow Cytometry for CAR-T & Lesson Learnt from Vaccine

Chair: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
11:50am-12:10pm: Dr. Sylvie Bertholet, Director Preclinical Evidence Generation,Vaccines GlaxoSmithKline
"High Parametric Flow Cytometry Applied to Clinical Trial T-cell Exploratory Endpoints"
12:10pm-12:30pm: Dr. Naveen Dakappagari, Head Protein Biomarker Solutions, Navigate/Novartis
Dr. Shyam Sarikonda, Assoc. Director Flow Cytometry Immuno-Oncology Biomarker Development, Navigate/Novartis
"CAR-T Monitoring by Flow Cytometry: Assuring Quality While Acknowledging Limitations"
12:30pm-01:00pm: Social Networking - Time to "Visit" Exhibitors and Posters
01:00pm-01:30pm: Dr. Mark Milton, Executive Director Pharmacokinetic Sciences Ophthalmology and Department Lead Gene Therapies, Novartis
Ms. Anna Ma, Associate Director Translational Pharmacology, CRISPR Therapeutics
Dr. Heather Hirsch, Sr. Director Translational Pharmacology and Head Clinical Biomarkers, CRISPR
Dr. Sylvie Bertholet, Director Preclinical Evidence Generation,Vaccines GlaxoSmithKline
Dr. Naveen Dakappagari, Head Protein Biomarker Solutions, Navigate/Novartis
Dr. Shyam Sarikonda, Assoc. Director Flow Cytometry Immuno-Oncology Biomarker Development, Navigate/Novartis
Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
"Panel Discussion on Immunogenicity Gene Therapy, Cell Therapy and Vaccines"

qPCR, ddPCR and NGS

Chair: Dr. Nirjal Bhattarai, Principal Investigator and Product Reviewer Division of Cellular and Gene Therapies, CBER, US FDA
01:30pm-01:50pm: Dr. Jean-Claude Marshall, Sr. Director Clinical Biomarker Group and Precision Medicine, Pfizer
"Viral Vector Shedding for AAV Gene Therapy"
01:50pm-02:10pm: Dr. Lynne Jesaitis, Director BioAnalytical Sciences, BioMarin
"Best Practices in Digital PCR to Measure Vector/Transgene Quantities and Structural Kinetics"
02:10pm-02:30pm: Dr. Yanmei Lu, Sr. Director Biomarker and BioAnalytical Sciences, Sangamo Therapeutics
"NGS and qPCR-based Methods to Quantify Genome Editing and Gene Integration for Patient Monitoring"
02:30pm-03:00pm: Dr. Jean-Claude, Marshall, Sr. Director Clinical Biomarker Group and Precision Medicine, Pfizer
Dr. Lynne Jesaitis, Director BioAnalytical Sciences, BioMarin
Dr. Yanmei Lu, Sr. Director Biomarker and BioAnalytical Sciences, Sangamo Therapeutics
Dr. Nirjal Bhattarai, Principal Investigator and Product Reviewer Division of Cellular and Gene Therapies, CBER, US FDA
"Panel Discussion on qPCR, ddPCR and NGS"
03:00pm-03:30pm: Social Networking - Time to "Visit" Exhibitors and Posters

ASK THE REGULATORS!
Interactive Panel Discussion with All the Regulators

03:30pm-04:10pm: Address any questions you have on Cell & Gene Therapy Bioanalysis directly to the Regulator Experts
 Regulatory Panelists:
  • Dr. Sara Gagneten (US FDA)
  • Dr. Heba Degheidy (US FDA)
  • Dr. Nirjal Bhattarai (US FDA)
  • Dr. Xiaofei Wang (US FDA)
  • Dr. Andrew Exley (UK MHRA)
  • Dr. Therese Solstad Saunders (Norway NoMA)
  • Dr. Richard Siggers (Health Canada)


Cell & Gene Therapy Bioanalysis - Part 2: June 29, 2020 (EDT time listed)

11:00am-11:10am: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
"Introduction of Cell & Gene Therapy Bioanalysis Part 2"

ELISPOT for Gene Therapy/Vaccine & Single Cell Western Blot Assay

Chair: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
11:10am-11:30am: Mr. Jason DelCarpini, Sr. Manager Biomarkers and Clinical Assay Development, Bluebird Bio
"Characterization of a Single Cell Western Assay to Measure the Proportion of Red Cells Expressing b-globin Transgene"
11:30am-11:50am: Dr. Lisa Kierstead, Executive Director Vaccine Sciences, PPD
"What Gene & Cell Therapy ELISPOT can Learn from the Vaccine ELISPOT Experience?"
11:50am-12:10pm: Dr. Arno Kromminga, Chief Scientific Advisor, BioAgilytix
"ELISpot Method Development & Validation for Gene Therapies: Building on 2019 WRIB Recommendations, Practical Case Studies, Lessons Learnt, Evolving Trends"
12:10pm-12:40pm: Social Networking - Time to "Visit" Exhibitors and Posters
12:40pm-01:10pm: Mr. Jason DelCarpini, Sr. Manager Biomarkers and Clinical Assay Development, Bluebird Bio
Dr. Lisa Kierstead, Executive Director Vaccine Sciences, PPD
Dr. Arno Kromminga, Chief Scientific Advisor, BioAgilytix
Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
"Panel Discussion on ELISPOT & Single Cell Western Blot Assay"

Immunogenicity, Safety Risk Assessment and NAb/CDx Assays

Chair: Dr. Nirjal Bhattarai, Principal Investigator and Product Reviewer Division of Cellular and Gene Therapies, CBER, US FDA
01:10pm-01:30pm: Dr. Jim McNally, Chief Scientific Officer, BioAgilytix
"Application of Current Immunogenicity Guidance/Guideline (FDA/EMA) to Gene/Cell Therapy: Parallels and New Thoughts"
01:30pm-01:50pm: Dr. John Kamerud, Director BioMedicine Design, Pfizer
"Considerations on Development and Validation of AAV Capsid Nab (Transfection Inhibition) Assays"
01:50pm-02:10pm: Dr. Swati Gupta, Director Immunology, Allergan
"Gene & Cell Therapy Safety Risk Assessment and its Direct Influences on Bioanalytical Strategy"
02:10pm-02:40pm: Dr. Jim McNally, Chief Scientific Officer, BioAgilytix
Dr. John Kamerud, Director BioMedicine Design, Pfizer
Dr. Swati Gupta, Director Immunology, Allergan
Dr. Nirjal Bhattarai, Principal Investigator and Product Reviewer Division of Cellular and Gene Therapies, CBER, US FDA
"Panel Discussion on Gene Therapy Immunogenicity"
02:40pm-03:10pm: Social Networking - Time to "Visit" Exhibitors and Posters

2020 White Paper on Cell & Gene Therapy Bioanalysis

03:10pm-03:30pm: Dr. Heba Degheidy, Staff Scientist, Division of Cellular and Gene Therapies, CBER, US FDA
Dr. Nirjal Bhattarai, Principal Investigator and Product Reviewer Division of Cellular and Gene Therapies, CBER, US FDA
"Consensus & Recommendations on Cell & Gene Therapy Bioanalysis"
03:30pm-04:00pm: Cell & Gene Therapy White Paper Working Experts Group
"Panel Discussion on Cell & Gene Therapy Bioanalysis"




Final Agenda Agenda at a Glance