Pre-Conference Workshop Day

Monday, July 18

*All times are in EDT

8:00 - 10:00AM | Workshop A: An AI & Machine Learning Primer

Technological advancements in the fields of machine learning & AI present perhaps one of the greatest avenues of innovation and expedited vector development in the gene therapy space. Despite this, little is known in regards to leveraging this technology and what its capabilities actually mean for the vector development now and in the future.

Join this workshop to better understand how:

  • Using machine learning to generate capsid types offers the promise of speedy and precise vectors
  • Exploring the optimal methods for computational approaches to be successful
  • Predicting the function of capsids and making selections can be influenced by certain priorities, including immunogenicity, manufacturability, and specificity

Workshop Leaders:

Eric Kelsic

Eric Kelsic
CSO
Dyno Therapeutics

10:30 - 12:30PM | Workshop B: Immunogenicity in the Context of Next Gen Vectors

One of the biggest concerns with current gene therapy vectors is the immune response they induce when they are administered. Immune responses not only may make gene therapy less efficacious, but also trigger adverse events, which ultimately put patients at risk. With safety top of the priority list for any company operating in this space, investigating how next generation gene therapy vectors could help solve immunogenicity challenges is integral to delivering a safer, more efficient gene therapy to patients.

Attend this workshop to learn about:

  • Retaining functionality of AAV but removing the possibility of neutralising antibodies responding
  • Understanding that immune response can be from the capsid but also the transgene itself
  • Developing new AAV serotypes which humans may not have been exposed to for increased population coverage
  • Modifying vectors to ultimately achieve a lower vector dose to minimize overloading of the immune system
  • Here, we demonstrate that depletion of CpG motifs from the AAV expression cassette substantially reduces cross-priming of capsid specific CD8+ T cells in mice

Workshop Leaders:

Pierre Burg

Pierre Burguiere
Independent Consultant

1:00 - 3:00PM | Workshop C: Tissue Targeting Beyond the Liver for Next Generation Vectors

Gene therapy has the potential to treat a wide variety of devastating diseases, but one of the biggest challenges in doing this effectively is reaching the target cells  and achieving sufficient gene expression in those cells to make a meaningful impact. This is particularly true when working on systemic delivery, and in challenging cell types, such as the CNS or muscle. By developing next generation vectors that are better able to access difficult to reach areas of the body, more patients can be treated effectively.

Attend this workshop to learn about:

  • Choosing the right AAV serotype for different cellsChanging the capsid, promoter and transgene properties to transfect cells more easily
  • Case study on treating the CNS using gene therapy
  • Case study on treating muscle cells using gene therapy
  • More efficient targeting of specific cells can be achieved through capsid engineering
  • Increasing expression in desired targets leads to lower doses required which is key in reducing toxicity in systemic delivery
  • Sharing data on engineered capsids with increased liver tropism

Workshop Leaders:

Dwaipayan_Sen

Dwaipayan Sen
Associate Director Vector Development & Production
Astellas Gene Therapies

Li Oh

Li Oh
Associate Director
Capsida Biotherapeutics

3:30 - 5:30PM | Workshop D: Optimizing Transgene Expression with Next Generation Vectors

Once a viral vector successfully delivers its therapeutic gene to the cells in question, the efficacy of the gene therapy depends on the quality of transgene expression. Specifically, the transgene must be expressed at the appropriate level, in the appropriate cells, and for the appropriate duration to mediate the desired clinical effect. For therapeutic uses, the transgene may need to be expressed permanently if the gene therapy is to serve as a one-time cure. To maximize chances of success, early viral-vector gene therapies have opted to include regulatory elements that have been selected to drive high levels of transgene expression in all cell types. However, overexpression of the transgene or its expression in the wrong cells may contribute to inflammation and other toxicities Moreover, current gene therapies, once administered, cannot be controlled or turned off by clinicians should the need ever arise.

As such this workshop will look to cover:

  • How transgene expression can be further programmed by engineering regulatory elements into the vector sequence
  • Turning on transgene expression in certain cell types or tissues to prevent potentially toxic expression
  • Such cell-type- or tissue-specific regulatory elements have become relatively common in viral-vector gene therapies
  • Incorporating regulatory elements, such as microRNA-target sites, that reduce expression in specified cells
  • Engineering inducible vectors where transgene expression can be controlled using an additional signal to turn on and off a gene therapy after it is administered, delivering a personalized course of treatment

Workshop Leaders:

tam

Lawrence Tam
Group Leader Vector Engineering Group
Gyroscope Therapeutics