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AAV Analytical Characterization Workshop

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AAV Analytical Characterization Workshop

Back to ARM Events

AAV Analytical Characterization Workshop

We invite you to join the Alliance for Regenerative Medicine and US Pharmacopeia for the AAV Analytical Characterization Workshop taking place March 8-9, 2023. This one and a half day workshop will take place in-person at the USP offices in Rockville, MD. If you cannot join us live, you are able to attend virtually via the livestream. This event is free for ARM members and it is also open to the public for a fee.

About This Workshop

Adeno-associated virus (AAV)-based gene therapies have shown promise for the treatment of many diseases, including rare diseases with unmet medical needs. Despite their extensive clinical application and licensure as marketed products, further efforts are required to increase product understanding with regard to their characteristics that may have an impact on clinical efficacy, safety, or immunogenicity. This workshop will cover various topics on AAV analytical characterization, including full / empty capsid characterization, potency assay, and titer measurements. This workshop aims to review state-of-the-art methodologies, share learnings on approaches for different products, with the ultimate goal of moving towards harmonized methods and agreements on potency expectations.

Registration Information

ARM Member

In-Person: Free to attend
Virtual: Free to attend

Industry & General Admission

In-Person: $350
Virtual: Free to attend

Hotel Information

If you are attending in-person, a room block has been reserved at the Hilton Washington D.C./Rockville Hotel. Use the link below to book your accommodations.

Agenda

Wednesday, March 8

8:00 – 9:00am: Breakfast
9:00 – 9:10am: Opening Remarks

Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)

9:10 – 9:50am: General Overview: Methods and Challenges on Full / Empty Capsid Characterization

Khandan Baradaran, Ph.D., Vice President, Regulatory CMC, Ultragenyx Pharmaceutical

9:50 – 10:20am: Case Studies and Landscape of Analytical Methods for the Characterization of AAV Full-To-Empty Particle

Jerome Jacques, Principal Scientist, USP

10:20 – 10:50am: Removal of Empty AAV Capsids to Undetectable Levels Using Orthogonal Purification Steps – Product Quality and Process Robustness Considerations

Rahul Sheth, Ph.D., Senior Scientist 2, Purification Process Development, BioMarin Pharmaceutical

10:50 – 11:20am: Characterization of Defective – Interfering Particles through Next Generation Sequencing

Lauriel Earley, Ph.D., Senior Scientist, Shape Therapeutics

11:20 – 11:50am: Evaluating PTMs in Relation to AAV Capsid Integrity

Adriana Kita, Ph.D., Associate Director, Analytical Development, Ultragenyx Pharmaceutical

11:50am – 12:30pm: Panel Session

All Morning Speakers

12:30 – 1:20pm: Lunch Break
1:20 – 1:50pm: Overview of Potency Assays for AAV

Meghan Brown, Ph.D., Executive Director, Regulatory Affairs CMC, Sarepta Therapeutics

1:50 – 2:20pm: Development of a Functional Potency Assay for the Characterization of a Novel Krabbe Gene Therapy

Erandi De Silva, Ph.D., Co-founder, Forge Biologics

2:20 – 2:50pm: Multi-Attribute Potency Assay for AAV Gene Therapy Drug Product

Connie Tsai, Ph.D., Associate Director, Analytical Development Bioassay, Novartis Gene Therapies

2:50 – 3:20pm: Success Story of Luxturna Potency Assay Development and Validation

Ravindra Kumar, Ph.D., Director, Bioassay Lead, Analytical Sciences, Biologics, Vaccines, Cell and Gene Therapy Analytics, Spark Therapeutics

3:20 – 3:35pm: Afternoon Break
3:35 – 4:05pm: Manufacturing Yields and Impact to Patient Access

Pierre Caloz, Chief Operating Officer, uniQure

4:05 – 4:50pm: Panel Session

All Afternoon Speakers

4:50 – 5:00pm: Day 1 Concluding Remarks

Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)

Thursday, March 9

8:00 – 9:00am: Breakfast
9:00 – 9:10am: Opening Remarks

Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)

9:10 – 9:40am: Quality of AAV Vectors: Impact on Induction and Detection of Immune Responses

Roberto Calcedo, Ph.D., Vice President, Preclinical and Immunology, Affinia Therapeutics

9:40 – 10:10am: Success Stories: Conversion from qPCR to ddPCR Targeting the GOI and Reduction in %CV to <15%

Matthew Hewitt, Ph.D., Executive Director, Scientific Solutions, Cell and Gene Therapy, Charles River Laboratories

10:10 – 10:40am: Product Comparability and the Transition from qPCR to ddPCR for Characterization of a Novel Krabbe Gene Therapy

Adam Davis, Ph.D., Senior Director, Analytical Development, Forge Biologics

10:40 – 11:10am: Moving from Vector Genome Titer to Quantification of Vector Genome Integrity by Multiplex dPCR

David Dobnik, Ph.D., Co-founder and Chief Scientific Officer, Niba Labs

11:10 – 11:20am: Break
11:20 – 11:50am: Approaching Complexity of Transition from qPCR to ddPCR for Particle Titer Measurements and the Use Orthogonal Particle Titration Methods

Andrea Hamilton, Ph.D., Associate Director, Quality Control, Gyroscope Therapeutics

11:50am – 12:20pm: Title TBD

Andrew Harmon, Ph.D., Lead Biological Reviewer, Gene Therapy CMC, U.S. Food and Drug Administration (FDA)

12:20 – 1:00pm: Panel Session

All Day 2 Speakers

1:00 – 1:30pm: Day 2 Concluding Remarks

Josephine Lembong, Ph.D., Manager, Scientific and Industry Affairs, Alliance for Regenerative Medicine (ARM)

Date:
March 8–9, 2023

Location:
USP Rockville Office

12601 Twinbrook Pkwy
Rockville, MD 20852

In Partnership With:

Questions?

Please contact Josephine Lembong at jlembong@alliancerm.org