AGENDA - Specialized Workshop F2 - Friday October 1, 2021: 8am - 5pm

"Gene Therapy, Cell Therapy and Vaccine Bioanalysis: Recent Issues in Scientific Advancements and Regulations"

US FDA & EU EMA expectations for Gene Therapy & Cell Therapy Immunogenicity Monitoring, Anti-AAV assays for patient enrollment/stratification in CAP/CLIA or/and GLP/GCP labs, need/not need to be a CDx, Assay format selection for CAR-T ADA Assay and Triplex CAR-T NAb Assay, Bioanalytical methods for CRISPR/Cas9 genome editing bioanalysis, Vaccine Bioanalysis and Harmonization on validation for Vaccine Assays, qPCR/ddPCR Bioanalytical Method Validation (BMV) and QC to monitor performance for Gene Therapy & Vaccine, Acceptance criteria for Biodistribution/Shedding Assay, Cell-based Aassays for Vaccines, current industry standards for CAR-T evaluation for Flow Cytometry & ELISpot assays

Session 1: Cell Therapy

Chair: Dr. Lina Loo, Director- Bioanalytical Preclinical Development, Vertex
08:00am-08:20am: Dr. Bonnie Wu, Associate Scientific Director Biologics Development Sciences, Janssen
"Overcoming Matrix Interference for a Triplex Biparatopic CAR-T NAb Assay"
08:20am-08:40am: Mr. Bernd Potthoff, Senior Scientist II, Novartis
"Advanced Immunogenicity characterization by Flow Cytometry & ELISpot for CAR-T programs"
08:40am-09:00am: Dr. Timothy Mack, Scientific Director, Bristol Myers Squibb
"Considerations for the Bioanalysis of Cellular Kinetics"
09:00am-09:20am: Dr. Xiaodong Fang, Senior Director of Bioanalysis and Bioassays, AskBio
"ddPCR Assay Development, Validation and Standardization for Cell Therapy"
09:20am-09:50am: Dr. Bonnie Wu, Assoc. Scientific Director Biologics Development Sciences, Janssen
Mr. Bernd Potthoff, Senior Scientist II, Novartis
Dr. Timothy Mack, Scientific Director, Bristol Myers Squibb
Dr. Xiaodong Fang, Senior Director of Bioanalysis and Bioassays, AskBio
"Panel Discussions on Cell Therapy"
09:50am-10:10am: Short Break

Session 2: Vaccines

Chair: Dr. Shannon Harris, Head of Clinical Serology, Seqirus
10:10am-10:30am: Dr. Shabnam Tangri, Chief Scientific Officer, Navigate/Novartis
"Bioanalytical Paradigm for Selecting COVID19 Vaccine Candidates"
10:30am-10:50am: Dr. Katrina Nolan, Principal Scientist Regulated Global PPDM, Merck
"Modernizing Vaccine Bioanalytical Assays: Taking Vaccine Bioanalytical Assays into a New Frontier"
10:50am-11:10am: Dr. Sylvie Bertholet, Scientific Advisor & Director Preclinical Immunology, GlaxoSmithKline
"Immune Monitoring of Vaccine Responses – A Multi-Pronged Approach"
11:10am-11:30am: Dr. Frank Taddeo, Director Clinical Assay Development, Takeda
"Validation of Real Time PCR Assays to Support Vaccine Clinical Trials "
11:30am-12:00pm: Dr. Shabnam Tangri, Vice President / Head of BioPharma Division, Navigate/Novartis
Dr. Katrina Nolan, Principal Scientist Regulated Global PPDM, Merck
Dr. Sylvie Bertholet, Scientific Advisor & Director Preclinical Immunology, GlaxoSmithKline
Dr. Frank Taddeo, Director Clinical Assay Development, Takeda
"Panel Discussions on Vaccines"
12:00pm-12:15pm: Short Break

ASK THE REGULATORS! Interactive Panel Discussion with All the Regulators

Chair: Dr. Lina Loo, Director- Bioanalytical Preclinical Development, Vertex
Chair: Dr. Shannon Harris, Head of Clinical Serology, Seqirus
Chair: Dr. Meena, Vice President Bioanalytical, DMPK and Biomarker Development, Stoke
12:15pm–12:55pm: Regulatory Feedbacks on Submitted Studies and Regulations on Gene & Cell Therapy and Vaccine:
  • Dr. Nirjal Bhattarai (US FDA CBER)
  • Dr. Heba Degheidy (US FDA CBER)
  • Dr. Vijaya Simhadri (US FDA CBER)
  • Ms. Leslie Wagner (US FDA CBER)
  • Dr. Therese Solstad (Norway NoMA/EU EMA)
  • Dr. Dean Smith (Health Canada)
  • Dr. Akiko Ishii (Japan MHLW)
12:55pm-01:10pm: Short Break

Session 3: Gene Therapy

Chair: Dr. Meena, Vice President Bioanalytical, DMPK and Biomarker Development, Stoke
01:10pm-01:30pm: Dr. Wibke Lembke, Associate Director Translational Biomarker & Bioanalysis, UCB
"qPCR Assay Validation for Gene Therapy"
01:30pm-01:50pm: Dr. Luying Pan, Senior Director Head of Bioanalytics - Clinical Biomarker Innovation and Development, Takeda
"Immunogenicity Monitoring Related to AAV Gene Therapy"
01:50pm-02:10pm: Dr. Soumi Gupta, Senior Director / Head of Clinical Immunology, BioMarin
"Mitigation Strategies for Overcoming Pre-existing Immunity to AAV Vectors: Current Approaches, Potential Gaps and Outstanding Questions"
02:10pm-02:30pm: Dr. Wibke Lembke, Associate Director Translational Biomarker & Bioanalysis, UCB
Dr. Luying Pan, Senior Director Clinical Biomarker Innovation and Development, Takeda
Dr. Soumi Gupta, Senior Director / Head of Clinical Immunology, BioMarin
"Panel Discussions on Gene Therapy"
02:30pm-02:50pm: Short Break

Session 4: Evolving Science & Regulations

Chair: Dr. Meena, Vice President Bioanalytical, DMPK and Biomarker Development, Stoke
02:50pm-03:10pm: Dr. Yuanxin Xu, Vice President Early Development and Translational Medicine, Intellia
"Bioanalytical Challenges for CRISPR/Cas9 Genome Editing Therapeutic Development"
03:10pm-03:30pm: Dr. Brian Long, Associate Director Translational Sciences, Immunogenicity Assessment, Biomarin
"AAV5 Mediated Gene Therapy for Hemophilia A: Advances with Clinical Immunogenicity Data Related to Valoctocogene Roxaparvovec"
03:30pm-03:50pm: Dr. Mark Milton, Global Head Gene Therapies PK Sciences, Novartis
"Challenges in Developing an Anti-AAV Ab CDx Assay"
03:50pm-04:10pm: Dr. Yuanxin Xu, Vice President Early Development and Translational Medicine, Intellia
Dr. Brian Long, Associate Director Translational Sciences, Immunogenicity Assessment, Biomarin
Dr. Mark Milton, Global Head Gene Therapies PK Sciences, Novartis
"Panel Discussions on Evolving Science & Regulations"

Session 5: 2021 White Paper in Bioanalysis

04:10pm-04:30pm: Dr. Lina Loo, Director- Bioanalytical Preclinical Development, Vertex
Dr. Shannon Harris, Head of Clinical Serology, Seqirus
Dr. Meena, Vice President Bioanalytical, DMPK and Biomarker Development, Stoke
"Recommendations on Gene Therapy, Cell Therapy and Vaccine Bioanalysis: Recent Issues in Scientific Advancements and Regulations"
04:30pm-05:00pm: "Panel Discussions on 2021 White Paper in Bioanalysis draft Recommendations"
05:00pm-05:20pm: Short Break
05:20pm-06:20pm: Poster Spotlight Sessions with open access to all attendees




Agenda at a Glance Agenda at a Glance