8:45 am Coffee & Networking

9:15 am Chairman’s Opening Remarks

  • Adrian Bot Chief Scientific Officer, Capstan Therapeutics

Accelerating the Next Frontier of Cell Therapy with Next Generation Gene Editing

9:30 am Cell-Specific In Vivo Gene Delivery & Genome Editing via Retargetable Fusogens

  • Jagesh Shah Vice President - Gene Therapy & Technologies, Sana Biotechnology


• Highlight the in vivo delivery platform that Sana has developed for gene therapy

• Discuss progress in targeting T Cells and HSCs

• Provide examples of pre-clinical studies with delivery of integrating vectors and Gene editing machinery

10:00 am AAV-Mediated In Vivo Base Editing Prevents Cardiomyopathy in Mice

  • Gregory Newby Assistant Professor, Johns Hopkins Department of Genetic Medicine


• One dose of AAV encoding base editors could modify the disease-causing genomic nucleotide in ~80% of left ventricular cardiomyocytes

• This editing prevented the onset of hypertrophic cardiomyopathy, even in mice that were just two months away from developing symptoms

• Delivery of Cas9 nuclease to destroy the dominant mutant allele could also prevent cardiomyopathy but appeared to have a more narrow therapeutic window

10:30 am Roundtable Discussion- Transforming the Cell Therapy Space Using Precision Delivery Platforms with Next Generation Editing Machinery

  • Gregory Newby Assistant Professor, Johns Hopkins Department of Genetic Medicine


• Developing safer and more precise genetic modifications with complex gene editing strategies such as base

editing/prime editing

• Overcoming the challenge of unpredictable off-target edits

• Improving cellular viability with reduced possibility of double-strand breaks

11:00 am Morning Break & Refreshment

Evaluating Therapeutic Payloads Being Utilized for In Vivo Delivery

11:30 am Development and Evaluation of Non-Viral RNA Delivery Platforms

  • Owen Fenton Assistant Professor, University of North Carolina


• Commentary on the advantages and applications of RNA based medicines

• Commentary on the challenges associated with delivering RNA based medicines to the correct location in the body for the right amount of time and at the proper dose

• Commentary on synthetic non-viral delivery vectors for controlled RNA delivery to cell populations in vitro and in vivo

12:00 pm Panel Discussion: War of the Payloads – Addressing the Advantages & Disadvantages of Transient VS Direct Integration

  • Cory Sago CEO, Stealth Mode Biotech
  • Sheena Smith Head of Program Management, Vector BioPharma
  • Owen Fenton Assistant Professor, University of North Carolina
  • Adrian Bot Chief Scientific Officer, Capstan Therapeutics


• Discussing the use of non-stable integration with a DNA payload for greater safety and control

• Discussing the challenges associated with stable integration, including lack of control in the context of proliferated cells

• Re-thinking re-dosing strategies for enhanced efficacy

• Evaluating the implications of removing lymphodepletion for in-vivo approaches, and associated effects on persistence/durability

12:45 pm Lunch & Networking

Unveiling the Full Potential of In Vivo Engineering Therapeutics to Treat Patients in Oncology and Beyond

1:45 pm Delivering Genetic Cargoes to Cells Of Choice In Vivo Using Non-Viral Delivery Vectors


  • Development of non-viral delivery vectors, and the delivery of multiple different payloads to cells.
  • In vivo delivery to immune cells
  • Exploring the use of these vectors for diverse indications.

2:15 pm Mastermind Session: Discussing the Translatable Applications of In- Vivo Therapeutics in Oncology, Autoimmunity, Infectious Disease & More

  • Adrian Bot Chief Scientific Officer, Capstan Therapeutics
  • Bakul Gupta CEO, Co-Founder, ImmTune Therapies


• Deep dive session comparing and contrasting in vivo engineering of therapeutic cells to treat a wider range of disease indications including solid tumor, cardiovascular condition, IBD lupus, HIV and more

• Discussing the blockbuster potential for in vivo approaches to bring lost lasting treatments to areas of high unmet need

3:00 pm Chairs Closing Remarks

3:15 pm Close of Summit