Day 1 - Gene Delivery Systems Public Workshop

8:30 – 8:45 am 

Welcome & Opening Remarks

• Charles Romine, Associate Director for Laboratory Programs (NIST)
• Heather Lombardi, Director, Office of Cellular Therapy and Human Tissue, Office of Therapeutic Products, CBER (FDA)

8:45 – 9:35 am

Workshop Overview

• "NIST Role in Standards for Emerging Biotechnology and advanced therapy" – Sheng Lin-Gibson (NIST)
• “Standards Recognition Program for Regenerative Medicine Therapies” – Judy Arcidiacono (FDA)
• “Regulatory perspective: CMC considerations for viral vectors” – Anurag Sharma (FDA)
• "NIST efforts on Gene Delivery Systems and ISO standards" – Ed Kwee (NIST)

9:35 – 10:05 am

Complementary Programs

• “Accelerating Medicines Partnership Program – Bespoke Gene Therapy Consortium” – Courtney Silverthorn (FNIH)
• “NIIMBL Program in Viral Vector Manufacturing and Analytics” – Kelvin Lee (NIIMBL)

10:05 – 10:20 am

Break

10:20 – 11:35 am

Common challenges and difference of gene delivery systems and ongoing efforts

• “Enabling Affordable and Accessible Advanced Medicines” – Boro Dropulic (Caring Cross)
• “Common Analytical Challenges with Adeno Associated Virus Mediated Gene Therapy” - Will Fountain (Andelyn Bioscience)
• “Analytical Challenges Associated with Lipid Nanoparticle mediated Gene Delivery and Gene Editing” – Carl Co (Arbor Bio)
• “Extracellular Vesicles (EVs) as a Novel Drug Delivery Platform" – Haiying Zhang (Weill Cornell Medical)
• “Analytical challenges for adenovirus-based vectors” – Nancy Sajjadi (Sajjadi Consulting)

11:35 am – 12:00 pm             

Panel Discussion – commonalities and differences

12:00 – 1:00 pm

Lunch Break

1:00 – 2:10 pm

Breakout Sessions (in-person only)

• Viral vector functional measurements
• Viral vector physical measurements
• Non-viral vector functional measurements
• Non-viral vector physical measurements

2:10 – 2:30 pm

Break

2:30 – 4:30 pm

New Technology Showcase (Measurements and Systems)

• “NibaPlex® assay platform for AAV vector genome integrity quantification” – David Dobnik (Niba Labs)
• “Viral vector capsid characterization using mass photometry” – Gabriella Kiss (Refeyn)
• “How super resolution can helps you gain better insights into your samples”  –Ricardo Nunes Bastos (Oni)
• “Image cytometry technology and applications for viral infectivity/transduction analysis” - Bo Lin (Revvity)
• “Label-free single cell PAT precision analytics for cell and gene therapies and vaccines” - Renee Hart (Lumacyte)
• “Insights into LNP quantification and their therapeutic application”- Carsten Russ (Intellia)
• “Focused ultrasound modulation of the blood-brain-barrier permeability for delivery of gene therapy to the brain” - Frederic Padilla (Focused Ultrasound Foundation)
• “Therapeutic Applications of Novel Q5 Exosomes” - Javed Siddiqi (Exom BioPharma)

4:30 – 5:00 pm

Closing

• Report of breakout sessions
• Summary with polling
• Next Steps for NIST Gene Delivery Systems Working Group and opportunities for participation.

Jamie Almeida (NIST)

5:00 pm

Adjourn