Day Two Agenda

8:00am - 5.00pm | Boston, MA

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8:00 am Registration Open & Coffee Networking

8:50 am Chair’s Opening Remarks

  • Adrian Bot Chief Scientific Officer & Executive Vice President of R&D, Capstan Therapeutics

Utilizing In Vivo Editing to Target the Liver

9:00 am In Vivo Gene Editing to Treat Monogenic Blood Disorders

Synopsis

• Moving from traditional gene therapy to in vivo gene editing approaches
• Combining CRISPR/Cas9 with second generation factor VIII transgene
• Targeting the liver to treat hemophilia and beta-thalassemia

9:30 am In Vivo Liver-Directed Gene Editing to Treat Highly Prevalent & Life-Threatening Disease

  • Kiran Musunuru Professor of Cardiovascular Medicine & Genetics, Perelman School of Medicine at the University of Pennsylvania

Synopsis

• Outlining program to safely and durably turn off genes in the liver
• Managing the challenges of assessing human-directed therapies in non-human pre-clinical studies
• Expanding to other indications to achieve to true “in vivo” potential

In Vivo Approaches for Enhanced Target Cell Specificity Beyond the Liver

10:00 am In Vivo Engineering of B Cells

  • Adi Barzel Senior Lecturer, Dept. of Biochemistry & Molecular Biology, Life Sciences Faculty, Tel-Aviv University

Synopsis

• Targeting and engineering B cells in vivo with dual AAV, one coding for CRISPR/ Cas9 and another coding for bNAb donor cassette
• Showing memory retention and bNAb secretion at high titres
• Providing the opportunity to treat different indications: HIV, oncology and beyond

10:30 am Morning Break & Networking

11:00 am Developing Modular CRISPR-based RNPs to Edit Selected Cell Types In Vivo

  • Mary Haak-Frendscho President, Chief Executive Officer & Board Member, Spotlight Therapeutics

Synopsis

• Developing TAGE (Targeted Active Gene Editors), a new class of in vivo cell-targeting CRISPR RNP-based biologics
• Applying TAGE to expand the target space in immuno-oncology (IO), as well as for monogenic diseases therapies
• IO TAGE, targeting selected immune cells, reprogram the tumor microenvironment to facilitate a systemic anti-tumor response

11:30 am In Vivo Approaches to Generate CAR T Cells Engineered to Mediate Durable Antitumor Responses

  • Ryan Larson Vice President, Head of Immunology, Umoja Biopharma

Synopsis

• Autologous CAR T cell products have been transformational in hematological malignancies however manufacturing complexities limit patient access
• Umoja’s VivoVec off-the-shelf lentiviral vector platform enables efficient generation of functional CAR T cells in vivo
• Applying these approaches to target both hematological and solid tumor cancers

12:00 pm Fusogen Technology: Improving Targeting for Multiple Indications

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

Synopsis

• Overcoming the fundamental challenges of intracellular delivery
• Delivering nucleic acid and protein payloads to specific cell types effectively
• Exploring the application to and pre-clinical studies from oncology

12:30 pm Lunch Break & Networking

Roundtable Sessions: Addressing the Unanswered Questions to Unleash the In Vivo Potential

1:30 pm Optimizing the In Vivo Approach: Considerations for Safety & Tolerability

  • Sid Kerkar Vice President, Research & Development, EXUMA Biotechnology

Synopsis

• How do we improve the safety and tolerability of in vivo therapies?
• To what extent do we have to worry about off-target effects?
• How do we circumnavigate the issue of immunogenicity and integration?

2:00 pm Optimizing the In Vivo Approach: Considerations for the Clinic

  • Dharini Shah Senior Director, Immunology, Tidal Therapeutics - Sanofi

Synopsis

• How do we provide a durable response without lymphodepletion?
• Is multiple dosing the answer?
• What are the safety considerations of chronic dosing?

Advancing Gene Engineering Strategies to Improve the Payload

2:30 pm Advancing CRISPR-based Genetic Medicine for Highly Efficient and Specific Genome Modification

  • Brett Staahl Co-Founder & Head of Platform Technologies, Scribe Therapeutics

Synopsis

• Pioneering novel viral and non-viral delivery technologies to truly enable in vivo genetic medicine
• Outlining X-editing (XE) technology to provide greater activity, specificity and deliverability
• Utilizing XE to address diseases of significant unmet medical need

3:00 pm Afternoon Break & Networking

3:30 pm Novel CRISPR-Associated Gene-Editing Systems from Metagenomic Samples Enable Efficient & Specific Genome Engineering In Vivo

Synopsis

• Assembly and interrogation of a metagenomic database reveals new gene-editing systems with attractive properties
• Metagenomi nucleases can be used to create a wide variety of engineered primary immune cells
• Metagenomi nucleases effect extremely efficient gene editing in the mouse liver

4:00 pm New Tools for the Development of In Vivo Gene Editing Therapeutics

  • Tedd Elich Chief Scientific Officer, Life Edit Therapeutics

Synopsis

• Leveraging a proprietary microbe collection to develop next generation gene editing systems
• Enabling a broad array of editing modalities including deletion, insertion and base editing
• Applying these tools for the development of next generation in vivo therapies

Planning for the Future to Move from Ex Vivo to In Vivo

4:30 pm Identifying Bioproduction Challenges to Accelerate Therapies into the Clinic

Synopsis

• Addressing the advantages and disadvantages of ex vivo and in vivo manufacturing
• Overcoming current limitations when manufacturing vectors
• Adapting the corresponding quality control to fulfill regulatory agencies expectations

5:00 pm Chair’s Closing Remarks