[mlyle]

> that making new nanoparticles

I hate all the buzzwords around "nanoparticles", but here we're talking about "nanoparticle" encapsulated mRNA-- like is used in the mRNA-based COVID vaccines.

Here, the formulation is intended for inhalation instead of injection.

And it's carrying CRISPR machinery to do gene-editing.

[HPsquared]

The conspiracy theorists are going to love this one, I think.

[Gatsky]

The point still stands, there are thousands of papers demonstrating preclinical utility of nanoparticles for many applications, and extremely few actual medicines.

[mlyle]

> there are thousands of papers demonstrating preclinical utility of nanoparticles for many applications, and extremely few actual medicines.

For nanoparticle delivered mRNA, things are relatively early and relatively little is to market. But I don't think we can reasonably dismiss it as being niche or not too useful, given that we just dosed hundreds of millions of people for the biggest emergent public health issue in recent memory.

[DoreenMichele]

Adult cystic fibrosis patients account for like a third of all lung transplants in the US and children with CF account for like half of all pediatric lung transplants. Lung transplants cost like a million dollars, can lead to a gruesome death if rejection sets in and require lifelong drugs.

The latest, greatest drugs for CF are extremely expensive (around $250k annually last I checked), don't work for everyone and must be taken forever as well.

Standard treatments for CF are extremely expensive and the condition is extremely debilitating.

It's a good candidate for new gene editing therapies in part because there is a simple, identified issue: a defective cell channel. Some genetic disorders are more complicated than that.