Pre-Conference Workshop Day

Tuesday, November 29

Workshop A

8.30am - 10.30am

From Bench to Clinic: Successfully Translating Basic Research to a Viable Therapeutic Product

There are a plethora of gene editing therapies in all stages of development, many of which are currently navigating the late preclinical and translational development stages. As such, it has never been so crucial to discuss how to appropriately translate your drug substance into a successful drug product. This workshop aims to overcome current challenges in translational development and leverage the most relevant insight conducive to successfully progressing your CRISPR-based therapy into a viable therapeutic product.

Attend this workshop to learn about:

  • Generating robust and relevant preclinical models
  • Evaluating current limitations in animal models to successfully predict CRISPR therapeutic outcomes
  • Assessing the relevance of surrogate animal models
  • Understanding current delivery challenges and the impact this has on transability of your therapeutic product
  • Outlining how to develop safety packages for assessing unwanted off-target and long-term side effects
  • Exploring the potential in using CRISPR technology as a diagnostic tool

Workshop Leader:

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Danilo Maddalo
Group Leader – Translational Oncology

Workshop B

11am - 1pm

Characterizing CRISPR-Based Therapies

It is important to characterize your developed gene editing therapeutic tobunderstand the impact of off-target impacts such as gene editing induced mutations. This workshop aims to assess current and novel methods, assays and analytical tools to help you better understand your own gene editing

Attend this workshop to learn about:

  • Novel tools, analytics and assays for full characterization of your CRISPR therapeutic products
  • Understanding the latest molecular assays and analytical tools to characterize CRISPR-based therapy efficiency and preciseness, particularly for off-target impacts
  • Reviewing the impact of different off-target effects and their implications for the safety profile of your CRISPR-based therapy
  • Top tips for success when developing base editing analytical platforms
  • Refining potency assays for LNP base editing technologies

Workshop Leader:

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Carlo Zambonelli
Senior Director, Analytical Sciences
Beam Therapeutics

Workshop C

2pm - 4pm

Non-Viral Approaches to In Vivo Delivery

Whilst there are currently over 300 CRISPR-based gene editing programs Workshop Leaders: active in trials worldwide, fewer than a fifth of these are in clinical development. A common challenge in bridging this gap lies within the delivery of CRISPR cargo in a safe, efficacious and tissue-targeted manor, with numerous established and novel delivery platforms obfuscating the choice for drug developers. Here, we dive deep into emerging non-viral delivery platforms to help you maximize the in vivo delivery potential of your CRISPR-based therapy.

Attend this workshop to learn about:

  • Improving eVLP architectures to enable more efficient packaging of CRISPR-based cargos
  • The in vivo therapeutic efficacy across multiple tissues, and minimal off-target editing effects of eVLP delivery methods
  • An overview of the current polymeric nanoparticle delivery systems being used by industry
  • Comparing the benefits of polymeric nanoparticles over traditional LNP delivery methods, including the potential ease of manufacturing scale-up
  • Optimizing shuttle peptides for delivering Cas9, Cas12 RNPs and ABECAS9 RNPs
  • The unique benefits and challenges of this novel delivery method

Workshop Leader:


Rammohan Devulapally
Principal Scientist – Drug Delivery Chemistry and Nanoparticle Formulation Development

David Guay - Feldan Therapeutics

David Guay
Vice President Innovation and Technology
Feldan Therapeutics


Aditya Raguram
PhD Student, David Liu Lab
Harvard University