
FDA Approves First Gene Therapy for Leukemia - perseusprime11
http://www.npr.org/sections/health-shots/2017/08/30/547293551/fda-approves-first-gene-therapy-treatment-for-cancer
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jfarlow
Kite's $12b acquisition by Gilead [1] on Monday may be a harbinger of
anticipated success of these kinds of biotherapeutics.

Given the modularity of these CAR systems, I'd expect to see a cascade of more
approvals as the therapeutic scaffold is perfected. It's not just giant pharma
working on these systems either. Younger companies like Cell Design Labs,
Chimera bio, Nkarta are taking very novel approaches to further refining the
specificity and precision of these kinds of treatments.

The cool thing about the CAR system is that it's amenable to rational
engineering from modular, biological components. The CAR approved here was the
product of low-throughput painstaking labor, but many companies are trying to
boost the rate of R&D.

We're building digital infrastructure to facilitate the rational design of
these kinds of biotherapeutics in hopes of speeding up the R&D cycle through
an intelligent high-throughput development.

You can dig into the structure of the approved CAR therapy here:
[https://serotiny.bio/notes/proteins/car19/](https://serotiny.bio/notes/proteins/car19/)

[1] [https://uk.reuters.com/article/us-kite-pharma-m-a-gilead-
sci...](https://uk.reuters.com/article/us-kite-pharma-m-a-gilead-sciences-
idUKKCN1B810Y)

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perseusprime11
I just hope these kinds of innovations become accessible to people while not
replicating the big pharma greed of high prices that dominated the better part
of this century leaving people I'm a developed nation like ours rely on animal
antibiotics.

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jfarlow
These kinds of genetically designed therapies will require some major
retooling of infrastructure, capital, regulation, etc., but are fundamentally
cheap and curative - for mis/dis-regulation of the body's own systems.
Ultimately, the material to be delivered is extraordinarily cheap to produce.
There is a LOT of capital that must be recouped to get these innovations up
and running, but in time they should dramatically drop in price and
accessibility.

