8.30am - 4.30pm EST | 5.30am - 1.30pm PST

8:30 am Opening Registration & Coffee Networking

Transformative Medicine – Discussing the Potentials of mRNA Protein Replacement Therapies

9:00 am mRNA-Based Approach for Treating Ischemic Heart Disease

  • Lior Zangi Associate Professor, Icahn School of Medicine at Mount Sinai

Synopsis

  • Protein replacement technologies holds a great potential to overcome the lack of therapeutic proteins in different diseases as lung, liver, and heart diseases
  • What are the advantages of mRNA-based over virus-based protein replacement therapies?
  • Explore the wide-ranging diseases currently being targeted through novel strategies

9:30 am Immuno-Modulation & mRNA-based Protein Replacement Therapy Programs for Treatment of Pulmonary Disease

  • Carsten Rudolph Managing Director & Chief Scientific Officer, Ethris GmbH

Synopsis

  • Outlining novel technologies being harnessed for immune modulation
  • Can this be a viable therapeutic for treatment in patients with severe diseases?
  • What are the challenges of this therapy, and is this limited by largescale production?

10:00 am mRNA Applications in Epigenetic Therapy

  • Jay Sarkar Co-founder & Chief Technology Officer, Turn Biotechnologies

Synopsis

  • What is Epigenetic Reprogramming of Aging (ERA) and how does it represent a new and distinct application space of mRNA therapy?
  • Why is mRNA the ideal modality for ERA and what are the accompanying and distinct design considerations?
  • What are the unique delivery challenges and solutions required for this class of therapeutics?

10:30 am Live Panel Q&A – Ask Speakers your Burning Questions

  • Lior Zangi Associate Professor, Icahn School of Medicine at Mount Sinai
  • Carsten Rudolph Managing Director & Chief Scientific Officer, Ethris GmbH
  • Jay Sarkar Co-founder & Chief Technology Officer, Turn Biotechnologies

10:50 am Morning Break & Speed Networking

11:50 am Results of A Phase 1 Trial Demonstrating mRNA Can Encode & Produce Potentially Therapeutic Levels of a Monoclonal Antibody with Neutralizing Activity Against Chikungunya Virus

  • Allison August Vice President, Clinical Development, Infectious Diseases, Moderna, Inc

12:20 pm Panel Discussion: On the Horizon: What is the Future of mRNA Treatments?

  • Norman Begg Independent Vaccine Consultant, Former Chief Medical Officer, GSK Vaccines
  • Lusong Luo Senior Vice President, External Innovation, BeiGene
  • Gal Cafri Immunotherapy & Genetic Engineering Group Leader, Sheba Medical Center
  • Jacob Becraft Chief Scientific Officer & Co-Founder, Strand Therapeutics Inc

Synopsis

mRNA clearly represents a huge potential for prophylaxis across a wide variety of disease indications, and the future looks bright for mRNA-based therapies. As this dedicated community continues working to bring mRNA therapies to patients in need, we enter an exciting era where anything is possible. This panel will consider the future potential of mRNA treatments, debate where the years to come will draw biotech, pharma, and academic leaders, and discuss the continuation of investments and partnerships surrounding

1:00 pm Networking Lunch

Exploring Novel Strategies for mRNA-Based Gene Therapies for Cancer Immunotherapy & Beyond

2:00 pm Advancing Delivery Of mRNA Therapeutics For The Treatment Of Genetic Diseases

  • James Sullivan Senior Vice President Pulmonary Research, Translate Bio

Synopsis

  • Outlining a new approach for the treatment of diseases caused by protein or gene dysfunction
  • Discussing the successful production of desired proteins through delivery of lipid nanoparticle-encapsulated mRNA to various target tissues
  • How proprietary lung delivery system has supported the advancement of our lead program in cystic fibrosis into the clinic
  • Successful application this delivery technology via other routes of administration in multiple target tissues to assess the potential across a broad array of diseases

2:30 pm Transforming Medicine: In Vivo Therapeutic Genome Editing via Transient Expression of CRISPR/Cas9

Synopsis

  • Intellia Therapeutics is a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo
  • Applying modular platform advancements across multiple in vivo targets in the liver and other tissues
  • Leveraging a lipid nanoparticle formulation of mRNA and guide RNA for efficient and transient expression of the genome editing machinery in the target organ
  • NTLA-2001 is the first-ever systemically delivered CRISPR-based therapy, currently in Phase 1 clinical trials
  • Showcasing our in vivo therapeutic platform and pipeline

3:00 pm Reversing the Irreversible: Epigenetic Reprogramming of Aging Through mRNAs

Synopsis

  • Transient expression of mRNAs encoding for nuclear reprogramming factors shows a robust and multifaceted reversal of biological clock in human cells
  • Cellular rejuvenation is characterized by reversal of methylation clock showing on average 5 years of rejuvenation
  • If applied to muscle stem cells it results in 40% of muscle force induction to levels typical of young animals

3:30 pm Live Panel Q&A – Ask Speakers your Burning Questions

3:30 pm End of mRNA Based Therapeutics Summit

4:00 pm Chair’s Closing Remarks