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  • Kaitlyn Choi

The delivery is a key

In the list of upcoming IPOs, I saw a biotech company called Taysha Gene Therapies. The company's gene therapy programs haven't entered Phase 1 safety trials yet, but its valuation is "well above the typical range for even a clinical stage firm." Then, what could explain this high valuation?


I looked into their S-1 registration statement and learned that the company is using machine learning to "develop a novel AAV (Adeno-Associated Virus) capsid platform." Capsid is to gene therapy as an envelope is to a mail delivery. Capid not only encapsulates the genetic material we'd like to deliver to a defective tissue, but also ferries the gene(s) to a specific tissue. In other words, AAV4 targets cells in the eye, whereas AAV5 targets lung cells (click here to see more details). The ability to find and engineer the best candidate capsids for specific deliveries is paramount for the success of gene therapies. In addition, Taysha could develop a library of AAVs with distinct capsids and sell them to other gene therapy companies.


Many business articles covering Taysha's IPO talk about the pipeline of gene therapy candidates and the partnership with UT Southwestern. I wonder whether a turbo engine for the company's growth is actually its capsid platform development.


(This post was originally published in my personal blog on 9/19/2020)

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