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Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of geneediting.
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.
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.
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.
While the biotech will not develop the geneediting therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish.
The findings build on early evidence that geneediting inside the body could safely and effectively treat disease, and suggest the effects of Intellia's medicine might last.
The startup, which launched last year with $315 million in funding and plans to advance a new form of geneediting, is one of the first big venture-backed biotechs in several months to seek an IPO.
Expert advisers are reviewing the companies’ case for approval of what could be the first medicine based on the geneediting technology. Follow their discussion here.
A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.
A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.
The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.
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.
The geneediting company will focus on “in vivo” medicines, while seeking to license out or find a development partner for its clinical-stage treatment reni-cel.
The findings provide early proof that multiple doses of a geneeditingmedicine can be safely administered with additive effects, overcoming a key constraint of the complex therapies.
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. The genomic medicine journey. Zinc fingers.
Adrian Hernandez In this Friday’s PCT Grand Rounds, Adrian Hernandez of Duke University will present “Precision Health to Population Health: Opportunities and Challenges for GeneEditing Therapies.” ” The Grand Rounds session will be held on Friday, August 2, 2024, at 1:00 pm eastern. Join the online meeting.
Biology is opening up new frontiers in medicine. We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics.
Formed as a diagnostics developer, Mammoth Biosciences, which Doudna started with two of her former students at the University of California, has accelerated plans to make geneeditingmedicines as well.
The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with geneediting and RNA medicines.
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.
The company is one of three preclinical biotechs to successfully go public since last summer, but it pulled in significantly less than others who’ve debuted with more advanced medicines.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA geneediting therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on geneeditingmedicines.
By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry geneediting with a fast-emerging form of cancer immunotherapy.
Gene-editing therapy aimed at two targets—HIV-1, the virus that causes AIDS, and CCR5, the co-receptor that helps the virus get into cells—can effectively eliminate HIV infection, new research from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) shows.
The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editingmedicines.
The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” geneeditingmedicines.
The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo geneediting programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD).
Shape Therapeutics is evolving programmable RNA medicines that push the limits of current geneediting capabilities. BioSpace spoke with CSO David Huss.
Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA. On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.
In a new episode of the pharmaphorum podcast, Rahul Kakkar, CEO of geneediting company Tome, speaks with web editor Nicole Raleigh about programmable genomic integration (PGI) technology.
The geneediting biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.
Gene-editing biotech Prime Medicine has priced its IPO, hoping to raise $140m to advance its pipeline of one-shot therapies for severe genetic disorders.
Editas Medicine’s new leadership team on Wednesday reiterated a promise to deliver clinical updates on two CRISPR-based treatments before the end of the year — data the troubled biotech hopes will ease investor doubts about its gene-editing technology.
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