Day One

7:45 am Morning Networking Coffee & Registration

8:35 am Chairperson’s Opening Remarks

Jacob Becraft Co-Founder & CEO, Strand Therapeutics

Navigating the Future Outlook of Successful mRNA-Based Vaccines and Therapeutics

8:45 am mRNA-1273 (Spikevax) – The Past, Present, & Future?

Rituparna Das VP – COVID Vaccines , Moderna

Synopsis

Adult vaccine development
Pediatric vaccine development
Variant vaccine development

9:15 am Pharma Leaders Fireside Discussion

Jacob Becraft Co-Founder & CEO, Strand Therapeutics
Kathy Fernando VP, Head of Worldwide Research, Development and Medical Operations; Head of mRNA Scientific Planning, Pfizer
Regis Gervier Global Head mRNA Center of Excellence , Sanofi
Hao Chen Global Head of Cell & Viral Drug Substance, GlaxoSmithKline

Synopsis

With the next wave of mRNA on the rise and with large pharma that were involved in prepandemic now having a validated platform on their hands to develop new therapeutics and vaccines, this leads to the inevitable question: What comes next? It is now important to realize the opportunities of such mRNA- based therapeutic applications, the
opportunities to develop global partnerships and collaboration as we hear from leading experts on the future of this technology

10:00 am Session Details To Be Confirmed

Tomas Kostelec Product Manager, mRNA, BIA Separations

10:30 am Morning Structured Networking

Track A: Discovery

Engineering Next-Generation mRNAs to Expand
Therapeutic Applicability

Chair: Gregory Fiore, CEO, Exacis Biotherapeutics

11.30 Engineering an mRNA Platform for Durable Expression of Vectors

Exogenous vs endogenous regulation of durability
Regulatory switches on RNA molecules
Future advancements in RNA designs

Vittorio Sebastiano, Co-Founder & Scientific Advisory Board Chairman, Turn Biotechnologies

12.00 Exploiting Diversity Found in Nature for the Development of Next Generation srRNA Vectors

Four pillars drive the bioactivity of self-replicating RNA: design of the protein insert(s), choice of vector, drug delivery system, and manufacturing process
Mining the diversity present in viruses enables origination of next generation self-replicating RNA vectors
There is no “one size fits all” with each pillar being a fully independent variable in the development of new products

Shigeki Miyake-Stoner, Director - R&D & Head - Technology, Replicate Bioscience

12.30 Onco-Selective mRNA Therapeutics for Cancer Immunotherapy

Cancer ribosomes present unique opportunity for novel, onco-selective mRNA designs
Onco-selective mRNAs encoding immunogenic cell death proteins results in highly effective oncolytic activity in vitro
Onco-selective KR-333 mRNA LNPs enable robust antiactivity in syngeneic tumor models

Yusuf Erkul, CEO, Kernal Biologics

Track B: Pre- & Clinical Development

Advancing Clinical Development of mRNA Therapeutics for Rare Diseases

Chair: Danilo Casimiro, CSO, Sanofi Pasteur

11.30 Strategy & Rationale for Development of mRNA for Metabolic Disorders

Overview of mRNA therapy, its potential applications with a focus on targeting liver disorders
Background and pre-clinical development of an mRNA therapeutic for Propionic Acidemia
Path to the clinic

John Marshall, Senior Director – Rare Disease, Moderna

12.00 The Benefits of mRNA as a Therapeutic Modality for Rare Genetic Diseases

Putting mRNA into perspective: viewing mRNA as an important part of the integrated ecosystem of therapeutic modalities and identifying parallels between mRNA, enzyme replacement therapy, and gene therapy
Case study of UX053 for Glycogen Storage Disease Type III (now in Ph 3 study): why mRNA was the ideal modality, study design considerations, and lessons learned
The future of mRNA therapeutics for rare genetic diseases: summary of benefits of the modality, what the industry still needs to learn, and what’s on the horizon for mRNA-based therapeutics

Camille Bedrosian, CMO & Executive VP, Ultragenyx

12.30 Pre-clinical Strategies for Evaluating & Mitigating Safety Parameters for Better Clinical Outcome

Current status of safety evaluation
What additional evaluations need to be included to current evaluation process
How to mitigate any potential safety parameters

Arunan Kaliyaperumal, VP - Preclinical Biology, ReCode Therapeutics

Track C: Manufacturing

Unlocking Early Process Developing for Efficient Downstream Manufacturing & Improving the mRNA Ecosystem

Chair: Manmohan Singh, SVP - Pharmaceutical
Sciences & Delivery Technologies, Beam Therapeutics

11.30 mRNA-Based Vaccine Development for Infectious Diseases: Opportunities & Challenges

mRNA platform technology has potential to prevent and control of future epidemics and pandemics
Current pipeline of mRNA-based vaccines is very diverse
There are number of challenges to be addressed including thermostability, durability of immune response, IP restrictions, availability of raw materials
CEPI is committed in supporting a number of programs for improving ecosystem of mRNA technology development

Arun Kumar, Pre-clinical Vaccines Development Lead, Coalition for Epidemic Preparedness Innovations (CEPI)

12.00 Plan Now or Fail Later: Why Raw Materials Matter

Session Details to Be Confirmed

Darwin Asa, Executive Director - RNA Technology & Manufacturing, Verve Therapeutics

12.30 mRNA Drug Substance Attributes & Manufacturing Considerations for Gene Editing Therapeutics

GMP manufacture of mRNA drug substance
mRNA process development and scale-up challenges
mRNA attributes and impurities that affect gene editing performance in non-human primate

Christopher Cheng, Executive Director - RNA Technology & Manufacturing, Verve Therapeutics

1:00 pm Networking Lunch & Tech Slam

Track A: Discovery

New & Emerging Delivery Technologies for mRNA- Based Vaccines & Therapeutics

2.00 Effective Delivery of mRNA Vaccines and Therapeutics with PharmaJet Needle-Free Injection Systems

Effective immune and clinical response of using PharmaJet needle-free injection systems compared to traditional needle/syringe
Potential for dose-savings and cost-effectiveness when using needle-free compared to needle/syringe
High patient and healthcare worker acceptability of needle-free injection systems, leading to elimination of needle-phobia and needle-sticks and increased vaccination rates

Carmen Ledesma- Feliciano, Clinical Device Specialist, PharmaJet

2.15 Identification of Novel LNPs for mRNA Delivery Using In Vivo Screening

In vivo LNP-barcoding allows us to screen hundreds of nanoparticles at once
Identifying novel delivery vehicles using this screening method
Adapting this screening method to improve its efficiency and robustness

Kalina Paunovska, Postdoctoral Scholar, Dahlman Lab - Georgia Institute of Technology

2.45 Novel Lipid Nanoparticle Platform Enables RNA Delivery for Different Applications in Genetic Medicine

RNA can be designed to silence, express, and edit specific genes providing a flexible and powerful approach to treating disease
RNA are large molecules that require nanoparticles for delivery to target tissues and cells
We have developed the GenVoyTM lipid nanoparticle platform, comprised of proprietary ionizable lipids and compositions designed for specific applications in genetic medicine
We showcase data in the areas of vaccines, gene therapies and cell therapies to demonstrate the versatility of the GenVoyTM platform to accelerate development of different genetic medicines

Samuel Clarke, Director - R&D, Precision NanoSystems

3.15 Panel Discussion Revealing the Latest Advances in Novel mRNA Delivery Systems for LNPs & Beyond to Expand the Therapeutic Applicability of mRNA-Based Therapeutics

Rationally designing novel mRNA delivery systems, from molecular structure through to tissue specific targeting
Harnessing the power of biophysical modeling to improve mRNA delivery of the future
Standardizing mRNA delivery systems for various disease treatments

Moderator: Russel Johnson, VP – Formulation Research, RVAC Medicines

Linda Marban, CEO, Capricor Therapeutics
Daniel G. Anderson, Professor of Medical Engineering & Science; Harvard-MIT Division of Health Sciences & Technology; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology
Gaurav Sahay, Associate Professor, Oregon State University
Manmohan Singh, SVP - Pharmaceutical Sciences & Delivery Technologies, Beam Therapeutics
Vittorio Sebastiano, Co-Founder & Scientific Advisory Board Chairman, Turn Biotechnologies
Muthiah (Mano) Manoharan, SVP - Innovation Chemistry, Alnylam Pharmaceuticals

Track B: Pre- & Clinical Development

Translating Pre-clinical to Clinical Success in the Context of mRNA- Based Therapeutics & Vaccines

2.15 Pre-clinical Considerations for Programmable mRNA Therapies for Enabling Bench to the Clinic Transition

Know your platform
Uncouple various parts of the drug product to conduct thorough risk assessment
Execution of the risk assessment

Prashant Nambiar, SVP - Research & Translational, Development, Strand Therapeutic

2.45 Model Informed Dose Optimization for mRNA Therapeutics & Vaccines

Overview of Pharmacometric approaches for clinical development of mRNA therapeutics and vaccines
Translational PK/PD modeling to predict first in human responses for mRNA therapeutics
Immunostimulatory/immunodynamic modeling for guide dose selection for mRNA vaccines

Husain Attarwala, Director & Head, Pharmacometrics & Clinical Pharmacology, Moderna

3.15 Panel Discussion Deep Diving into Dose Frequency & Local or Systemic Modes of mRNA Administration to Suit the Treatment of Different Diseases

Benchmarking challenges faced during intravenous, intrathecal, intraventricular, intramuscular mRNA modes of administration, and revealing innovative approaches to overcome them
Comparing the therapeutic risk-reward profile of each mode of delivery along with combination therapies to understand which diseases can withstand which side effects
Overcoming the difficulties of efficacious mRNA payloads to penetrate deep tissue
Do we fully understand kinetics of IV injections and off-target effects of lipid compositions?

Moderator: Metin Kurtoglu, COO, Cartesian Therapeutics

Husain Attarwala, Director & Head, Pharmacometrics & Clinical Pharmacology, Moderna
Camille Bedrosian, CMO & Executive VP, Ultragenyx
Arunan Kaliyaperumal, VP - Preclinical Biology, ReCode Therapeutics
Lior Zangi, Associate Professor of Cardiology; Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai

Track C: Manufacturing

Translational & Scalable Manufacturing of mRNA Based Vaccines & Therapeutics

2.00 Reserved for Trilink Biotechnologies

Session Details to Be Confirmed

2.15 mRNA Manufacturing: Throughput, Innovation, Scaling

Influence of mRNA design on manufacturability
Robust mRNA manufacturing platform: Challenges and perspectives
State-of-the-art process development for throughput and scalability

Rachit Jain, Associate Director - mRNA Process Development, Tessera Therapeutics

2.45 Strategy for Overcoming Upscaling & Analytical Challenges of Nanomedicines for mRNA Delivery

Clinical development for scalable manufacturing of nanomedicines
Critical quality attributes of nanomedicines
GMP manufacturing of mRNA delivery vehicles

Mark van Eldijk, Principal Scientist – Nanomedicine; Development & Manufacturing, Ardena

3.00 Panel Discussion Addressing Regulatory Perspective on Requirements for Effective mRNA Development & Manufacturing

What are the requirements from regulatory agencies for manufacturing mRNA therapies?
Exploring how to streamline development of mRNA therapies to ensure rapid and effective approval
Can companies leverage opportunities within regulatory guidelines for mRNA, siRNA, oligonucleotides and other methods?

Moderator: Hao Chen, Global Head - Cell & Viral Drug Substance, GlaxoSmithKline

Arun Kumar, Preclinical Vaccines Development Lead, Coalition for Epidemic Preparedness Innovations (CEPI)
Charles Cabral, Drug Substance Manufacturing; Technology & Tech Transfer Director, Arcturus Therapeutics
Akhilesh Bhambhani, Senior Director- Biologics Drug Product Development; Tech Dev/Tech Ops, Ultragenyx

4:00 pm Afternoon Networking Break

4:30 pm Reserved for Applied DNA Sciences

Synopsis

Session Details To Be Confirmed

5:00 pm Diversity, Equality & Inclusion Round Table: Prioritizing Diversity, Equality & Inclusion in the mRNA Industry

Linda Marban CEO, Capricor Therapeutics
Hamideh Parhiz Research Assistant Professor of Medicine, Perelman School of Medicine, University of Pennsylvania
Tamar Grossman Global Head - RNA & Targeted Therapeutics , The Janssen Pharmaceutical Companies of Johnson & Johnson
Jane True VP - mRNA Commercial Development, Pfizer

Synopsis

What impacts are there on talent strategy in organizations today?
What is something actionable people can do today to make the scientific field more inclusive?
What developments and practices have organizations made or put in place that other leaders should consider adopting?

July 26-28 | Boston, MA

July 26-28 | Boston, MA

Day Two

7:45 am Morning Networking Coffee & Registration

8:35 am Chairperson’s Opening Remarks

Navigating the Future Outlook of Successful mRNA-Based Vaccines and Therapeutics

8:45 am mRNA-1273 (Spikevax) – The Past, Present, & Future?

Synopsis

9:15 am Pharma Leaders Fireside Discussion

Synopsis

10:00 am Session Details To Be Confirmed

10:30 am Morning Structured Networking

1:00 pm Networking Lunch & Tech Slam

4:00 pm Afternoon Networking Break

4:30 pm Reserved for Applied DNA Sciences

Synopsis

5:00 pm Diversity, Equality & Inclusion Round Table: Prioritizing Diversity, Equality & Inclusion in the mRNA Industry

Synopsis

5:30 pm An Overview of the mRNA Landscape & Future Directions

Synopsis

6:00 pm Chair’s Closing Remarks & End of Conference Day One

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