Exa-cel, the first CRISPR therapy moves closer to market

Rodger Novak is the president of CRISPR Therapeutics, a company he co-founded with Emmanuelle Charpentier in 2013. Jennifer Doudna was invited to join but declined.

The road from clinical trials to regulatory green light now appears to be downhill for the treatment for sickle cell anemia developed by CRISPR Therapeutics, the company co-founded by Emmanuelle Charpentier. We knew it as CTX001 but it has changed its name to exa-cel (which stands for exagamglogene autotemcel). It was one of the first CRISPR-based gene therapies to enter clinical trials, in 2019. It changed the lives of Victoria Gray and dozens of sickle cell anemia and thalassemia patients enrolled in several countries. Now it also leads the way in the late stage of the regulatory process, both in Europe and the United States, and could come to market first, in 2023. For more information see the press-release by Vertex, that collaborates at exa-cel manufacturing, regulatory and commercialization.

CRISPR crops & Italy elections

Nature Italy is a digital magazine published by Springer Nature, focused on scientific research and science policies in Italy. On 25 September the country goes to the polls, so Marta Paterlini, Fabio Turone and Nicola Nosengo asked some of the major parties about their proposals on climate, science, and health. Unfortunately the right wing Fratelli d’Italia, leading in opinion polls, and its ally, Forza Italia, didn’t respond. Below are the other parties answers about new genomic techniques in agriculture (in Italy they are sometimes called “assisted-evolution technologies”). In brief M5S and UP are quite elusive, while the others are in favor with different nuances.

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Give gene therapy a market chance

From Jakob Kamil Guziak’s website

The scientific renaissance is still there, but the commercial abandonnement is already going on. Patients affected by rare diseases and their families are worried they won’t be able to get treatments that are safe and effective but unprofitable for drugmakers. Take a look at the story of Jakob Kamil Guziak, recently told by Business Insider.

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Seeing the forest for the CRISPR trees

Plant species are threatened by pathogens and pests, and the climate crisis is making things worse. CRISPR could help address some forest health threats, by making trees more resilient. But intervening in a complex ecosystem is a big decision. What are the alternatives? What are the uncertainties? Let’s think about a specific example, the American chestnut, and watch this iBiology video. 

Crispy salads are here!

Berkeley professor Patrick Hsu on twitter: “Delighted to try out the world’s first CRISPR-edited salad”

I must say that I’m a bit envious and eager to taste this kind of Brassica juncea with the “mustard bomb” mechanism prevented by knocking-out multiple copies of the gene responsible for the bitter taste.

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Gender equity meets CRISPR

Navneet Matharu, Jenny Hamilton and Lin Du

The Women in Enterprising Science Program (WIES) is located on the UC Berkeley campus and is supported by the foundation of Solina Chau Hoi Shuen (co-founder of Horizons Ventures in Hong Kong). The initiative, aiming to enhance gender equity in bio-entrepreneurship, was presented last March by IGI, the institute founded by Jennifer Doudna. In the pictures above you can see the inaugural cohort of fellows, announced this month.

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Cholesterol down – ready set edit!

Last week Verve Therapeutics dosed the first patient with a candidate treatment for hypercholesterolemia. This is exciting news for a couple of reasons. First, the technology used: CRISPR 2.0, i.e., base-editing is hitting the clinic (see the news in Nature Reviews Drug Discovery). Second, this is a leap forward into common diseases (“CRISPR for the masses”, says The Washington Post) and a training session for the real challenge, which is to “stop the biggest killer on Earth”, cardiovascular disease (MIT Technology Review).

Happy 10th Birthday CRISPR!

The seminal paper by Doudna & Charpentier was published online at the end of June 2012. The printed issue came out a few weeks later, on August 17 (don’t try to buy it, Science VOLUME 337|ISSUE 6096 is out of stock). No wonder the gene-editing community is in the mood for celebration these days. If you are too, don’t miss the chance to read these articles on CRISPR’s ten-year anniversary!

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The greatest functional map of all

Credit: Jen Cook-Chrysos/Whitehead Institute

Jonathan Weissman and colleagues used a CRISPR-based method to link each expressed human gene to its function in the cell. Here’s our suggested readings to learn more:
the paper in Cell by Josepg Replogle et al.
the Twitter thread by Joseph Replogle
MIT News (by Eva Frederick) and GenEngNews.