This week’s hot topic: prime editing moves closer to patients

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A multi-billion dollar collaboration between Prime Medicine Inc. and Bristol-Myers Squibb brings this important technology closer to patients. 

At the end of 2023, the FDA already approved Casgevy (exagamglogene autotemcel), developed jointly by CRISPR Therapeutics and Vertex Pharmaceuticals for sickle cell disease and beta thalassemia, and bluebird bio’s Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease. 

It was proof of concept that this technology could be used as a viable therapy, instead of just an editing tool. 

But Prime’s technology takes the concept that bit further – the approved therapies can carry out one certain task, which is to edit a patient’s genes at a certain point to produce a cellular work-around to counter the disease. 

In the case of Casgevy, it edits a patient’s hematopoietic stem and progenitor cells to produce foetal haemoglobin, which carries oxygen around the body in place of the defective adult haemoglobin. 

Lyfgenia works differently by adding a functional beta-globin gene that aims to reduce the proportion of red blood cells that are sickle-shaped and relieving the debilitating and dangerous symptoms such as pain crises and organ damage. 

Prime editing is a technology that acts like a DNA “word processor,” with the power to search and replace genetic sequences at their exact location in the genome. 

This is achieved without making double-strand breaks in DNA, which can threaten the integrity of a cell’s genome. 

For Cambridge Mass.-based Prime Medicine, the $3.61 billion deal gives the company a desirable financial cushion that will allow it to continue its research and bring this important technology closer to the market. 

The involvement of BMS will give development oversight and manufacturing and marketing expertise for the reagents for ex vivo T-cell therapies. The deal could help bring this important technology to patients, potentially correcting a wealth of serious hereditary diseases. 

 

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