Pre-Conference Workshop Day

Workshop A: How do Non-Viral Vectors Compare with Viral Vectors for Application in Gene Therapy?

Tuesday, June 8 | 11.30am - 1.30pm EDT | 8.30am-10.30am PDT

Viral vectors, and in particular AAV, dominate the gene therapy landscape. However, as more gene therapies enter the clinic, it’s becoming clear that AAV poses significant limitations which could inhibit gene therapy pipelines going forward. Non-viral vectors are in earlier stages of development and not yet widely used in the clinic, but could pose an attractive solution to several of the limitations of viral vectors, including immunogenicity, manufacturability, and genomic load.

Attend this workshop to learn about:

  • Discussing the latest non-viral vector candidates and what stage of development they are at
  •  Comparing and contrasting AAV, alternative viral vectors, and non-viral approaches
  • Are there any particular disease areas/cell types that would benefit from a specific delivery modality?
  • What are the current limitations of both viral and non-viral vectors, and how can they be overcome?

Workshop Leaders:

Russell Monds

Russell Monds
Director, Molecular Biology
Generation Bio

Workshop B: Engineering Vectors to Address the Challenge of Immunogenicity

Tuesday, June 8 | 2.00pm - 4.00pm EDT | 11.00am-1.00pm PDT

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

Workshop Leaders:

Genine Winslow

Genine Winslow
CEO
Chameleon Biosciences

Nicole Paulk

Nicole Paulk
Assistant Professor, Biochemistry & Biophysics
University of California San Francisco

Abraham Scaria

Abraham Scaria
CSO
IVERIC Bio