To Scale or not to scale? That’s the question!

Last week we shared a small but meaning thing that shows how much work is necessary to produce 1 billion human heart cells. This is a significant number because estimations by some researchers (doi:10.1038/nature10147) have pointed to 1 billion heart cells being number of cells that could potentially have a meaningful impact in patients with ischemic heart failure.

We have been advocating for a need of industrial scale processes to cultivate and differentiate human cells because we Do NOT see a future in which producing human cells at industrial scale is NOT in it. I doesn’t matter how different we look into the future, it seems very unlikely that we will not have mastered human cell cultivation and differentiation in industrial scale somehow. We think it will become something like producing recombinant protein today. You would be hard pressed to find a relevant pharmaceutical company not developing a product using recombinant protein technology. Cell therapy will become mainstream and LizarBio is working on this problem today.

Source: Reptile8488/Getty Images

I want to do some simple math to show the problem the industry face today in manufacturing and why scaling manufacturing processes are relevant.

• A heart indication would require, potentially 1 billion cells/patient
• There are currently, around 6m patients already diagnosed with heart failure just in in the US
• From the 6 million, 1% of them, that is 60k people, die every year from the diseases (likely the most severe cases)
• If we were to treat only the most severe cases every year, we would need a production capacity of 60.000 X.1.000.000.000 cells = 60 trillion heart cells/year
• A very good process can give you up to 10b cells/liter of culture
• Therefore, if this is to move the needle for patients, we need to be able to produce 6k liters of heart cells every year just to treat 1% of patients in the US (if nothing fails, right?). This is already industrial scale territory

Treating 1% of a patient population should not be our end goal, but this gives us a little perspective on where we should aim and the size of the scalability problem. And this rationale also works for other diseases such as diabetes, muscle wasting diseases and others. There are game changing medicines being approved with curative potential (just like Novartis Kymriah) that are so expensive and complex that over 5 years have been able to treat only about 5k patients worldwide (https://www.novartis.com/sites/novartis_com/files/novartis-annual-report-2021.pdf). So are plenty of patients in need of a curative treatment.

Therefore, why people don’t scale their current manufacturing process? This is key point: in biotech, the process IS the product. Therefore, once settled for your manufacturing, it will be very challenging to change it later on (specially if it is working fine). Therefore, companies tend to sacrifice scale, in order to obtain a small, but sure revenue stream. Hardly a bad economic decision. However, we are then left with high end therapies that can benefit only a small number of patients.

LizarBio is working to democratize cell therapy and be able to help as many patients as possible.

We keep pushing!

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