Pre Conference Workshop Day

Tuesday May, 7 | Boston, MA

Morning Welcome Coffee

08:00 AM

Workshop A

Optimizing Delivery Platforms for Next Generation RNA Therapeutics & Overcoming Targeted Delivery Challenges to Achieve Their Full Therapeutic Potential

9:00 am - 11:00 am 

In recent years, RNA-based therapeutics have shown remarkable promise in treating a wide range of diseases, from cancer to genetic disorders. However, ensuring efficient and targeted delivery of these therapies remains a prevalent challenge in realizing their full potential. In this session, we will explore the delivery vehicles and mechanisms available for RNA therapeutics, including circular RNA, siRNA, and saRNA, and discuss their unique challenges. We will also delve into strategies to address and overcome these challenges to unlock the full potential of RNA-based treatments.

Join this session to discuss:

  • The range of delivery options currently available, such as lipid nanoparticles, viral vectors, and chemical modifications
  • The challenges in delivering RNA therapeutics, including stability, cellular uptake, and tissue specificity
  • Understand the latest data in targeted delivery, and how to enhance this without compromising efficacy and safety

Workshop Leaders:

Anthony Salah Headshot

Anthony Saleh
Chief Executive Officer
MiRecule

Dinesh Chandra Headshot

Dinesh Chandra
Associate Director, Interventional
Oncology
Johnson & Johnson

vignesh

Vignesh Hariharan
Senior Postdoctoral Associate
University of Massachusetts Chan Medical School

Workshop B

Enhancing CMC Process Development to Streamline Next Generation RNA Therapeutic Development in Line with Regulatory Expectations

11:30 pm - 1:30 pm 

As the unprecedented efficacy of RNA in the treatment of various diseases has catapulted RNA-based therapeutics into the spotlight, it is crucial to design robust protocols for scaling and manufacturing these drugs to ensure regulatory approval and enable commercial use. With streamlined chemistry, manufacturing and control strategies, next generation RNA-therapeutics can be manufactured more efficiently and efficaciously to accelerate the development and manufacturing timeline.

Join this workshop to discuss:

  • Obtaining a comprehensive understanding of which steps and variables need to be controlled in the manufacturing process
  • Ensuring consistency of specificity, safety, stability and strength of the therapeutic drugs in both a clinical trial and commercial setting
  • Driving R&D success by minimizing bottlenecks in process development, manufacturing scale-up, qualification and validation, quality assurance and quality control approachesImproving CMC strategies to increase chances of regulatory success

Workshop Leaders:

jim nolan

Jim Nolan 
Vice President of Tech Ops
Sali Biomedicines

Gilles Besin Headshot

Giles Besin
Chief Scientific Officer
 Orbital Therapeutics 

Dharini Shah Headshot

Dharini Shah
Senior Director
Sanofi

DAY ONE
DAY TWO