Express | Solving the three major challenges of gene therapy delivery, what is unique about this cutting-edge platform?

▎WuXi AppTec Content Team Editor

Today, Versant Ventures, a venture capital firm, announced the launch of Vector BioPharma. This is a biopharmaceutical company dedicated to the development of precision gene delivery platform technology. Versant has committed to a $30 million Series A investment in the company. According to the press release, the company’s technology platform is expected to overcome three major challenges currently facing gene therapy delivery: tissue-specific delivery payloads, limited gene payload size for packaging, and adverse immunogenic responses.

Vector’s differentiated gene delivery platform is based on research in the laboratory of Dr. Andreas Plückthun at the University of Zurich. His team used engineered virus-like particles as a tool for precise delivery of gene payloads.

The delivery system, called SHREAD, has the following features:

It does not carry viral genes, thus reducing the immunogenicity of viral challenge.

Vectors designed based on adenovirus protein capsids have higher capacity can package genomes up to 36 kb compared to traditional adeno-associated viruses (AAV) vectors can only package 4.7 kb genomes.

By decorating the surface of virus-like particles with adapter proteins that can target different antigens with high affinity, this system can precisely deliver transgenes to tissues or tissues expressing specific antigens. cells.

Furthermore, the carrier capsid is covered with an “armor” of antibody fragments that prevents the capsid from being recognized and degraded by the immune system.

These features combine to give this platform the potential to revolutionize the safety, efficacy and specificity of cell and gene therapies, as well as biologics.

▲Illustration of SHREAD delivery system (Image source: Reference [2])

“It’s great to see that research we started 10 years ago can now be translated into new treatments,” said Dr. Plückthun. “I look forward to working closely with Vector’s team to bring these treatments to life. Choose to bring to the patient.”

The company expects to receive technical proof-of-concept data this year in key immuno-oncology and gene editing areas, and in the first half of 2023, the company expects to receive in vivo data for its lead investigational therapy.

As a CTDMO of WuXi AppTec focused on cell and gene therapy, WuXi Biosciences is committed to accelerating and transforming the development, testing, manufacturing and commercialization of gene and cell therapy and other high-end treatments. WuXiXi can help global customers bring more innovative therapies to the market as soon as possible for the benefit of patients. If you have relevant business needs, please click the picture below to fill in the specific information.