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Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on geneediting treatments for rare diseases.
A new alliance with Mammoth Biosciences marks the latest evidence of Vertex's interest in geneediting, which has already been the focus of multiple deals with emerging biotechs.
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR geneediting inside the body can be safe and effective, a culmination of years of scientific research.
Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of geneediting.
The IPO is several times larger than what CRISPR Therapeutics, Intellia and Editas raised several years ago, and ranks well ahead of a more recent offering from geneediting startup Beam.
The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering geneediting tools into the body, dates back to December 2020, but has recently begun to advance.
The biotech has identified geneediting as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions.
Capitalizing on investor interest in geneediting, the high-profile startup has secured one of the richest biotech initial public offerings priced this year.
The announcement, which follows recent regulatory setbacks for some of Intellia’s peers, is a “big win” for the geneediting field, according to one analyst.
Founded by University of California, Berkeley scientists, Rewrite is studying ways to expand the reach of geneediting beyond limitations of current technologies, including Intellia's.
Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo geneediting treatment for sickle cell disease.
Shares in LogicBio had lost nearly all their value since a 2018 IPO, bringing the company to the brink of Nasdaq delisting. The buyout deal represents a premium of over 600%.
A federal patent board ruled Broad Institute scientists were first to a key geneediting advance, weakening the patent position of Intellia and CRISPR Therapeutics, which hold licenses through the University of California.
Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.
Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in geneediting research in recent years.
The regulator will issue separate verdicts on the pioneering treatment’s use in sickle cell disease and beta thalassemia, with the first expected late this year.
Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership.
In addition to clearing Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, the FDA also granted an early OK to Bluebird bio’s sickle cell treatment Lyfgenia.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
Results from 10 patients with beta thalassemia or sickle cell disease show CRISPR Therapeutics and Vertex's pioneering treatment to be effective, building on earlier study data.
The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data.
Using technology from Caribou Biosciences, AbbVie hopes to engineer "off-the-shelf" CAR-T cells that can better withstand attacks from the immune system.
Verve, a buzzy geneediting startup, unveiled its first drug candidate on Tuesday, while Eli Lilly's and Pfizer's CEO gave updates on Alzheimer's and dealmaking.
Formed around research from the Broad Institute's David Liu and Andrew Anzalone, Prime has attracted significant funding from a wide range of high-profile biotech backers.
Cutting edge’ is, for once, a truly apt description when it comes to geneediting – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.
The next six months could feature clinical milestones for CRISPR geneediting, the treatment of COVID-19, microbiome drugs and gene-targeted cancer therapy.
Since halting work on a sickle cell treatment early this year, Graphite Bio has laid off staff, shed assets and, now, agreed to be subsumed via a merger with Lenz Therapeutics.
Verve didn’t say what led to the FDA’s decision, but claimed it hasn’t observed any safety issues in an ongoing trial in New Zealand and the U.K, where enrollment will continue.
Moonwalk Biosciences, the latest biotech cofounded by the geneediting scientist, joins other startups aiming to alter gene expression without changing DNA.
Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.
The geneediting startup is one of less than two dozen biotech companies to debut on Wall Street this year, as the pace of IPOs remains far off last year’s trajectory.
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