In the story from the Bible King Solomon ruled between two women who both claimed to be the mother of a child. In the CRISPR saga the contention is between biotech companies over foundational patents, and the next crucial episode will unveil the consequences for the first CRISPR therapy, Casgevy.
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Recently David Liu announced that Prime Medicine will likely submit the first human trial application in 2024. The standard version of CRISPR uses an RNA guide to find the editing site in the genome. Prime editing, on the other hand, also uses the same RNA molecule to direct the correction, in short, to specify what to do as well as where to go.
This insight blossomed in Andrew Anzalone’s mind a few years ago during his PhD at Columbia University. The first practical demonstration came with a paper published in Nature in 2019 after joining the Liu’s Lab at the Broad Institute. Since then, this platform has been used in hundreds of experiments to fix all kinds of mutations in vitro and in animal models.
Meanwhile, the company co-founded by Anzalone and Liu has begun work on 18 treatments, the most advanced for chronic granulomatous disease. To learn more, from the eureka moment to the latest developments, we suggest listening to the Close to the Edge podcast and reading Alex Philippidis’ article in GEN.
The news of the week is definitely this: the first clinical trial with base editing (the CRISPR platform used to chemically change single DNA letters without double-strand breaks) hit the goal of lowering cholesterol in patients but raised questions about the risks (with two serious adverse events, including one death), as Nature reports.
But we also recommend reading two other articles. Nature Biotechnology takes a look at experiments using CRISPR to eliminate viruses that manage to hide from the immune system, such as HIV and hepatitis. While Genetic Literacy Project publishes an analysis of the problems that could cripple the new regulation on edited plants proposed by the European Commission and delay (even until 2030) the arrival of the first products on the EU market.
Once upon a time there was a field of research that had been dormant for over a century, well now it has awakened. Approved clinical trials are in dozens, scientific journals are publishing reviews of the state of the art, the first books are coming out, chronicling unexpected individual successes, and even the first inquiries from major publications such as the Economist are popping up.
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After half a century of experimentation, biotech tools have advanced to the point that many treatments capable of changing the lives of people with rare and ultrarare diseases would be achievable, at least in theory. However, the harsh reality is that, in the vast majority of cases, the costs of research and production are too high, while the number of beneficiaries of each treatment is too small, to attract and then maintain the interest of the pharmaceutical industry.
After the first biomedical “valley of death” in which flawed drugs fatally strand, a second economic “valley of death” is looming for treatments that work but are not profitable enough, ss Michele De Luca and Giulio Cossu wrote in EMBO Reports. It is in this context that the Italian charity Fondazione Telethon last week announced the transfer of the marketing authorization for Strimvelis, a gene therapy for the immunodeficiency ADA-SCID developed by their reserachers (at SR-Tiget in Milan) and then abandoned by the manufacturer (Orchard Therapeutics) despite the excellent results.
“We are the first non-profit organization to take on the commercialization of a gene therapy. This step is necessary to remain true to our mission and continue ensuring access to this important therapy,” said Telethon’s general manager Francesca Pasinelli. Will others follow suit? To learn more about the background of this decision you can read our post from a year ago; for an updated insight we recommend Telethon’s press release.
Four questions to Luigi Naldini (San Raffaele Telethon Institute for Gene Therapy, Milan) about the Nature Biotechnology study that revealed limitations and risks of gene and prime editing.
Continue readingThe first is a CRISPR innovator (base editing and prime editing came out of his lab). The second has a genetic disease that causes him to age prematurely (progeria) and has taken his destiny into his own hands by becoming a biologist. They are each other’s inspiration and in this video they tell us why.
Jennifer Doudna’s Innovative Genomic Institute has received $70 million to explore a bold idea: combating climate change and other emergencies by modifying the microbial communities that live outside and inside us.
Bacteria are the true masters of the planet, for better or worse. Besides affecting our health in many ways, they are responsible for much of the methane emissions. This gas traps heat far more than carbon dioxide and is produced in large quantities by microbes that proliferate in environments associated with human activities (farms, landfills, rice paddies). The good news is that methane is short-lived, so reducing its emissions would have a rapid and substantial effect on global warming. What tools do we have at our disposal to try to pursue such an ambitious goal?
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The progress of the new therapies of the CRISPR era can be told by interweaving two stories. The first is the one featuring Victoria, Carlene, Patrick, Alyssa, Terry and many others. There are over two hundred patients who have so far undergone some experimental treatment based on genome editing, i.e. the targeted correction of DNA instead of the addition of extra genes as in classical gene therapy. These women and men suffering from serious diseases had to face increasing pain and sacrifice until they decided to pin their hopes on a new type of experimental therapy, which is promising but not without risks. For the unluckiest of them, this act of courage and faith in science was not enough, but for many of these pioneers, life really did change. In fact, there are already dozens of people who have managed to free themselves (hopefully in the long term) from the burden of a rare genetic disease or, in some cases, leukaemia. Along with genetically edited cells, a new normalcy has arrived for them and the chance to finally think about the future.
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The death of pioneer patient Terry Horgan is a warning about the risks of viral vectors but the focus is now on the first gene therapy being approved in the US
On the chellenging frontier of advanced therapies, every death is a pain from which everything possible must be learned. The inauspicious outcome of the individual treatment for Duchenne muscular dystrophy developed by the non-profit Cure Rare Disease for Terry Horgan, and tested solely on this American boy, can teach little about the specifics of CRISPR. Indeed, the death occurred before the molecular editing machine could get into action. But the information on the case, circulated in May on a preprint archive awaiting peer-reviewed, is nonetheless a valuable contribution to the advancement of knowledge in an area where science has no intention of giving up.
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CRISPeR FRENZY 