Tag Archives: gene therapy

CRISPR trials: the 2024 update

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The recent approval of Casgevy represents the first official success of gene editing-based therapies. The treatment for sickle cell anemia and thalassemia came in record time, only 11 years after CRISPR was invented. “Two diseases down, 5,000 to go,” commented Fyodor Urnov, Director of Technology & Translation at the Innovative Genomics Institute. Among the many diseases awaiting a cure, what will be the next to benefit from CRISPR? At what rate can we expect new treatments to arrive? The periodic update published by IGI is a must-read to navigate through hope and hype, papers and press-releases. The picture is overwhelmingly positive, but there is also some cause for disappointment. Here is an excerpt from the introduction:

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Better than Casgevy? Three paths to explore

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Credit: Bing Image Creator

Not only sickle cell anemia, now thalassemia as well. On January 16, the Food and Drug Administration completed the approval process for the second type of hemoglobinopathy too, while the European Medicines Agency is expected to give the green light in the coming months. It took just over a decade to go from the invention of the Cas9 genetic scissors to the first approved treatment, and the excitement over the milestone achieved in record time is more than justified. Yet an article in MIT Technology Review has already turned the spotlight on the next challenges. The title is, “Vertex developed a CRISPR cure. I’ts already on the hunt for something better”. Gentler conditioning for ex vivo gene editing, new vectors for in vivo delivery, and maybe even a pill mimicking the Casgevy mechanism without modifying DNA. This is how CRISPR researchers try to out-innovate themselves.

Charity saves life-saving therapy

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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.

Brilliant Minds: David Liu & Sammy Basso

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The 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.

CRISPR from bench to bedside

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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 lesson of Terry, the pioneer patient who didn’t make it

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Terry and Richard Horgan (Courtesy of Cure Rare Disease)

He was the first patient to get a CRISPR therapy for muscular dystrophy. The first to receive a CRISPR treatment made specifically for him. And also the first to try a CRISPR approach that did not aim to change a DNA sequence but only its expression (epigenetic editing). Six months after Terry Horgan’s passing, his brother Richard disclosed the first information on the cause of death.

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Chronicles from the London editing summit

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CRISPR patient Victoria Gray talking at the summit (credit The Royal Society)

The third – and perhaps final – act of the Human Genome Editing Initiative ended last week. The first summit (Washington 2015) was held amid enthusiasm for the invention of CRISPR, with the aim of fostering a constructive dialogue between science and society. The second edition (Hong Kong 2018) was dominated by the birth in China of the first edited human beings. The main points in the agenda of geneticists and bioethicists meeting a few days ago (London 2023) was to overcome the shock and focus on the next challenges: broadening the range of treatable diseases, reducing the costs of therapies, simplifying them so they can be administered anywhere in the world, and reach as many sick people as possible.

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CRISPR aims straight for the heart

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Photo credit Singularity Hub

The latest challenge is protecting damaged tissue immediately after a heart attack with the help of base editing (see the paper published in Science by Eric Olson’s group at the University of Texas Southwestern Medical Center). But there are hundreds of devastating diseases that affect the heart or other muscles and are caused by mutations that could be fixed by CRISPR-based tools (see this paper in Science Trsnslational Medicine for example). From Duchenne dystrophy to cardiomyopathies, some preliminary results are very encouraging.
Learn more reading the article on the Science paper published by El Pais and watching this video with Olson explaining his studies, especially on Duchenne muscular dystrophy.

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

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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.