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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

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.

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Epigenetic Editing Explodes on the Heels of Gene Editing Success

BioSpace

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

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Regeneron, Intellia target neurological diseases in expanded gene editing deal

Bio Pharma Dive

The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.

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Gene-editing firm Prime Medicine files $140m public offer

pharmaphorum

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.

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Ginkgo adds gene editing tools in buyout of Feng Zhang-founded startup

Bio Pharma Dive

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.

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The post Moderna partners with Life Edit for mRNA gene editing therapies appeared first on Pharmaceutical Technology.

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Verve partners with Lilly on gene editing therapy for heart disease

Bio Pharma Dive

Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).