Genetic Medicine: The Next Generation

遺伝子治療 The Next Generation レポート 英語版




Evaluate Vantageは、新しい遺伝子治療に取り組む企業の幹部に、第一世代が直面した課題をどのように克服しているのか、最前線からの見解を聞いた。

Huge investment in the first generation of genetic medicine has, by and large, led to incredibly high valuations for companies, and some in the market began to wonder whether gene therapy has been “a little overhyped”.  

The US biotech bear market has been particularly tough on gene therapy and editing groups, whose multi-billion valuations always looked rich considering the technologies involved were mostly unproven. Other issues the field has had to contend with include growing worries about toxicity and lack of durability, not to mention eye watering price tags.

However, the next generation of gene therapies are starting to gain some attention, albeit with more caution this time round. 

For a view from the frontlines, Evaluate Vantage spoke to executives at companies that are working on new gene therapies about how they are overcoming the challenges that beset the first generation.



The new delivery methods that may improve durability
A “plug and play” approach to genetic medicine
How gene therapy is moving beyond rare diseases
The thorny issue of price for these exceptionally expensive treatments