Register Now for the Upcoming Virtual Symposium

Scaling Theory to Therapeutic: Gain insight into clinical development for RNA-based therapeutics

Our fall symposium will focus on the possibilities of genomic medicine across different applications, including scale-up and accelerating the full drug development process, from Discovery through to Process Development and Scale-up for Commercialization, building on a common core of LNP technology. We hope that the attendees will learn about new possibilities, how to build on best practices of LNP formulation and optimization through CMC, analytics, clinical data etc. to gain insight into getting to clinical readiness around RNA-based therapeutics.


This event is free to attend, and featured speakers and their presentation key learnings are listed below.


Date: Thursday, October 27
Time: 6:00-9:30am PT  |  9:00am-12:30pm ET


Register now to save the date!




Akhilesh Bhambhani

Dr. Akhilesh Bhambhani  | Sr. Director, Biologics Drug Product Development at Ultragenyx


CMC Considerations for Developing an Effective mRNA/LNP Therapeutic


mRNA delivery for therapeutic applications in Genetic and Rare Diseases (GARDs) will be discussed. Specifically, the presentation will give an insight into certain

  • - Design principles for formulating LNPs
  • - Approaches to maximizing drug product quality shelf-life
  • - Factors governing efficacy while minimizing reactogenicity


Arun RaturiDr. Arun Raturi  | Chief Scientific Officer, Entos Pharmaceuticals


Proteolipid vesicles: A novel delivery system for safe and effective delivery of nucleic acids


Genetic medicine has the potential to treat a wide variety of diseases however, successful treatment in the clinic has been limited by tolerability, scalability and immunogenicity of current genetic medicine delivery platforms. In order to overcome these limitations, Entos has combined aspects from viral and non-viral delivery platforms to develop a proteolipid vesicle (PLV), which uses fusion-associated small transmembrane (FAST) proteins from fusogenic orthoreoviruses along with well-tolerated lipid formulations using a scalable microfluidic mixing approach. Lipid formulations incorporating the chimeric FAST protein have been optimized for high nucleic acid encapsulation, charge neutralization, and improved tolerability in vitro and in vivo, showing low immunogenicity and maintained activity upon repeat dosing over long periods, at high local or systemic doses. The activity and safety profile of FAST-PLVs makes them a promising alternative platform for redosable gene therapies and genetic medicines.



Ami JoDr. Ami Jo  | Senior Process Engineer, Drug Product Process Development at Omega Therapeutics


Building an LNP process for the Omega platform: a case study


A platform LNP process to encapsulate Omega’s platform epigenomic controller mRNA was developed.  A good understanding of process parameter impact on the product quality attributes is critical to faster development of individual LNP formulations. This presentation focuses on a case study using the Precision Nanosystems’ Blaze to test the impact of selected process parameters on the LNP physical characteristics for two different formulations. Using a DOE, clear trends and significant parameters were determined.



Emily PortEmily Port  | Team Leader, Pharmaceuticals at Centre for Process Innovation


Developing a platform process for LNP manufacture


My presentation details key aspects for developing a platform process for LNP manufacture. It covers the journey CPI took to get to where we are today, including key collaborations and formulation development of the various process parameters, and expands on the limitations of a platform process, but also the future potential applications across other drug delivery systems.