Gene therapies currently available

We should test the non viral vector using hTERT therapy in mice and move this into human…

Another good article by Bill Haseltine:

Longevity biotech company Gordian Biotechnology launches their in vivo screening platform…

Forbes magazine coverage:

When developing new therapies for diseases, biotech researchers are often limited by two time-consuming steps: first, screening thousands of drug candidates in test tubes and second, taking the best candidates and testing them on multiple animals to make sure it’s safe and effective. Combined, these steps can be slow and expensive.

Today, a startup called Gordian Biotechnology debuted a technology that could make this process better for both animals and people. The San Francisco-based company has developed a new animal screening platform which allows multiple gene therapies to be tested at the same time with just one animal. Instead of the gene treatment being given to the animal and affecting an entire area of its body, Gordian’s innovation enables it to test a drug inside of a single cell. That means one mouse could potentially support the evaluation of hundreds of possible new gene therapies in a way that’s faster and impacts fewer animals with less risk of harm to them.

Full story: This Biotech Startup Aims To Speed Up Drug Testing On Animals

The company website information on their platform:

More details in this Twitter / X thread:

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I assume that the boy gets the treatment for free as a “human guinea pig” discount.

Seems like a treatment only the top 0.1% can afford.

Gordian Biotechnology is introducing a method of screening hundreds of gene therapies simultaneously in an individual animal model - its co-founders explain

“Every time we approve a new gene therapy, and then the company announces the price of the gene therapy, the commissioner sends me a note and it’s usually [short]—I love short emails,” Marks told the crowd at the American Society of Gene and Cell Therapy annual meeting Wednesday morning. Marks serves as director of the Center for Biologics Evaluation and Research (CBER).

“The last one was a $4.25 million exclamation point," Marks recalled. "It was like a lot of exclamation points after. I can’t remember how many, and that was the email. And I knew what it meant.”

CBER does not consider prices in approval decisions for the therapies it reviews, Marks said. It’s just not part of the group’s remit.

“On the other hand, it’s my job to look at the entire ecosystem," he commented Wednesday. "Step back independent of a given product and say, is this sustainable? In other words, can we ultimately have the field of gene therapy mature and start to treat more common diseases if we keep having to charge a million or $2 million for a dose?”

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Every single technology starts expensive and gets cheaper to benefit all.

If you ban expensive technology you don’t get cheaper versions - you get no technology at all. Price caps lead to empty shelves, not cheap goods.

The only way we can “ultimately have the field of gene therapy mature and start to treat more common diseases” is by letting them charge as much as they want like all other free market products.

Not to mention - the only regulation that should exist is for safety, fraud prevention - and even that is debatable - how many people’s lives will be shorter with more disease because you need an authority approval to buy rapamycin?

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Promising Initial Results From a Phase II Trial of VEGF Gene Therapy