[Gatsky]

I was listening to a talk from a leading nanoparticle researcher. He basically said that making new nanoparticles and characterising them is great for writing papers but they all fail actual testing as therapeutic delivery devices. In oncology there are kinda only two nanoparticle therapies - Abraxane, which is a well known chemotherapy drug bound to albumin; liposomal doxorubicin, an encapsulated form of another well known chemotherapy drug. They have been around a while and they are not exactly game changing, extending survival a modest amount at best. Nanoparticles are overrated from where I sit, as are University press releases.

On the other hand, antibody based therapies are amazing revolutionary drugs in oncology. Chief among them is Keytruda which has improved the lives of so many patients and has $2billion in sales every month and rising. Most recently, sticking chemotherapy drugs or radioisotopes on the end of antibodies (or smaller antibody like proteins) has shown great results as a delivery vehicle. Are these ‘nanoparticles’? No, just actual drugs that work.

[DoreenMichele]

Oncology is about treating tumors (aka cancer). Cystic fibrosis is a genetic disorder with a well-known mechanism, a defective channel in the cell membrane.

It's already well established that improving the function of the CFTR or increasing the number of them is effective in mitigating the seriousness of the condition.

[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.