Tackling the Gene Therapy Challenge and Keeping Up with China

Thursday - November 2 2023, 05:26 UTC - 1 year ago

Researchers at Fudan University in Shanghai were able to restore hearing in patients suffering from inner ear hair cell loss. In order for gene therapy to become a successful medical treatment, more research needs to be done in order to make the product cost effective. The Chinese government has committed to improving the gene therapy industry, for example through subsidies and grants. However, despite this progress, gene therapy remains expensive and inaccessible.

On Friday, Shu Yilai, a professor and practicing surgeon at Fudan University in Shanghai, shared results of the trial, in which young patients received injections of a virus that added replacement DNA to the hair cells in their inner ear. Four out of five participants have since developed the ability to hear. As we wrote, "Scientists in China say [they] are the first people ever to have their natural hearing pathway restored … The feat is even more remarkable because until now, no drug of any kind has ever been able to improve hearing .

" But this remarkable result faces the same challenging calculations that plague a lot of medical solutions, both gene therapies and traditional medicine: they are used to treat a very specific and rare disease, so the market demand doesn’t justify the spending in R&D and mass production. While costs aren’t a factor for experimental trials like the one Yiyi took part in, they will become more salient if the technology is to be introduced to the mass market .

One thing that may help is government subsidies. In a bid to develop Shanghai’s competitiveness in the gene therapy industry, for instance, the municipal government has offered local companies sizable subsidies for developing new treatments; a company can receive up to $13 million a year for manufacturing an approved gene therapy product locally in the city. Other local governments in China have announced similar policies, which may attract more biomedicine companies into the field of gene therapy .

Nevertheless, today gene therapy remains a relatively expensive and inaccessible option. Nova Liu, the founder of Shanghai Refreshgene Therapeutics, which financially backed the Chinese research on this gene therapy, estimated that a commercialized product could cost between $125,000 and $250,000. That’s cheaper than other gene therapies currently available (CAR T-cell therapy, which treats cancer, can cost more than $1 million) but significantly more expensive than a cochlear implant (which in China costs just over $10,000) .

As Antonio summarized it for me: "Demonstrating that a gene therapy works is not the same as creating a successful medicine that helps people in the real world. For that, companies would need to carry out more studies, get approval, and finally sell the product for a profit. None of that is easy, especially in rare diseases where only a few patients can benefit from the treatment." Some previous products that have shown to be effective in trials have indeed languished before they can be brought to market .

This is the next battle that gene therapy will need to fight.Catch up with China1. Li Keqiang, China’s former second-ranking leader, died of a sudden heart attack on Friday. But public mourning has been stifled online to avoid social unrest. (The Guardian)2. California governor Gavin Newsom visited China last week to promote collaboration on climate actions. He took a ride in an autonomous SUV from BYD, China’s top EV maker .

(CNN)3. Hugging Face, the popular open-source AI platform, was blocked in China. (Semafor)4. Nvidia was asked by the US government to immediately halt its export of advancive AI chips into China. (Bloomberg)Researching and developing a gene therapy that helps people in the real world is a difficult challenge due to limitations of the size of the market and the cost of the treatments. However, with new initiatives from the Chinese government to prioritize the manufacturing of approved gene therapies, together with subsidies to motivate international companies, gene therapy might eventually become more accessible .