Day One Agenda

8:00am - 6:00pm | Boston, MA

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8:00 am Registration Open & Coffee Networking

8:50 am Chair’s Opening Remarks

  • Shon Green Senior Director, Translational Science, Umoja Biopharma

9:00 am Pioneering the In Vivo Cell Engineering Approach

  • Steve Harr President & Chief Executive Officer, Sana Biotechnology

Synopsis

• Explaining Sana’s strategy to use cell-specific delivery to overcome challenges
• Developing the fusogen technology to address multiple diseases
• Exploring the steps needed to bring medicines to patients with unmet clinical needs

9:30 am Industry Leader’s Fireside Chat: In Vivo Engineering of Therapeutic Cells as the Future of Cell & Gene Therapy

  • Andy Scharenberg Chief Executive Officer, Umoja Biopharma
  • Adrian Bot Chief Scientific Officer & Executive Vice President of R&D, Capstan Therapeutics
  • Kiran Musunuru Professor of Cardiovascular Medicine & Genetics, Perelman School of Medicine at the University of Pennsylvania
  • Sid Kerkar Vice President, Research & Development, EXUMA Biotechnology

Synopsis

• Answering the question: what has inspired the move from ex vivo to in vivo therapies?
• Outlining the advantages and opportunities that in vivo cell and gene therapies offer over current generations
• Discussing the next steps to streamline pre-clinical development to fast-track in vivo therapies to the clinic

10:30 am Morning Break & Speed Networking

Generating CAR T In Vivo : Does Pre-Clinical Data Show Efficacy?

11:30 am Advanced Viral Vectors for In Vivo CAR T Cell Generation

  • Christian Buchholz Head of Department, Molecular Biotechnology & Gene Therapy, Paul-Ehrlich-Institut

Synopsis

• Receptor-targeted LV and AAV vectors deliver genes to T cells with >99% precision
• Explain how shielding can overcome immunity barriers
• DARPin selection allows discrimination between closely related target receptors

12:00 pm In Vivo Generated CAR T Cells to Reverse Fibrosis & Restore Cardiac Function

  • Haig Aghajanian Co-Founder & Vice President of Research, Capstan Therapeutics

Synopsis

• Generating transient, functional antifibrotic CAR T cells in vivo by delivering modified mRNA in T cell-targeted LNPs
• Observing efficient delivery and translation of modified mRNA encoding a CAR to T lymphocytes and a reduction in fibrosis
• Leveraging this technology as a therapeutic platform to treat various pathologies beyond cardiac disease

12:30 pm Panel Discussion: Applying In Vivo CAR-T Cell Therapy For Oncology & Beyond

  • Haig Aghajanian Co-Founder & Vice President of Research, Capstan Therapeutics
  • Cory Bentley Co-Founder & Senior Vice President of R&D, Abintus Bio
  • Shon Green Senior Director, Translational Science, Umoja Biopharma

Synopsis

• Addressing the question: how in vivo CAR T cell therapy obviates current ex vivo bottlenecks including access, efficacy and toxicity
• Achieving a transient vs longer term effect for different indications

1.00pm Lunch Break & Networking

Novel Insights to Improve Delivery & Transfection Efficiency

2:00 pm Nanoparticle Single-Cell Multiomic Readouts Reveal that Cell Heterogeneity Influences Lipid Nanoparticle-Mediated mRNA Delivery

Synopsis

• Using Oligo tagged LNPs to discover novel LNPs
• scRNA sequencing of transcriptome and LNP delivery
• Used to discover LNPs that target hard to characterize cell types

2:30 pm Exploring Cell-Specific Viral Entry Approaches to Improve Gene Transduction & Specificity of Delivery

  • Michael Birnbaum Associate Professor, Dept. of Biological Engineering, Massachusetts Institute of Technology

Synopsis

• Deciphering immune recognition with RAPTR (Receptor-Antigen Pairing by Targeted Retroviruses)
• Providing a suite of new approaches for high-throughput interaction screening
• Exploring new approaches for targeted viral entry, including for in vivo engineering of therapeutic

3:00 pm Afternoon Break & Poster Session

4:00 pm A Regulatory Perspective: Implications for the In Vivo Approach

  • Michael Lehmicke Director, Science & Industry Affairs, Alliance for Regenerative Medicine

Synopsis

• Outlining the current regulations around different vectors including AAVs and
lentiviruses
• Exploring the potential regulatory implications for in vivo engineering

Reprogramming Cell Identity to Produce Potent & Durable Responses

4:30 pm Versatile Modular Viral Platform (MVP) for In Vivo Reprogramming for Oncology & Beyond

  • Cory Bentley Co-Founder & Senior Vice President of R&D, Abintus Bio

Synopsis

• Building on 20+ years of technology and clinical advances with in vivo administration of retroviral vectors
• Success factors for in vivo reprogramming
• Leveraging scalable manufacturing to meet the patient need

5:00 pm Chair’s Closing Remarks