Remove DNA Remove Gene Remove Gene Editing Remove In-Vivo
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Prime Medicine Gets FDA Okay for First Trial of Gene Editing Technique

BioSpace

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

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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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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STAT+: Intellia says CRISPR treatment safely corrects DNA of six patients with rare disease

STAT News

Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells. Participants in the high-dose group have not yet completed the 16-week observation period.

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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., In addition to mRNA, Acuitas LNP can be used to deliver a range of different nucleic acid therapeutics including small interfering RNA (siRNA), antisense oligonucleotides and DNA.

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Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. CRISPR nucleases serve as an important genome editing tool.

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.