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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.

DNA 142
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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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Artificial Intelligence innovation: Leading companies in in-silico drug discovery

Pharmaceutical Technology

Within the emerging innovation stage, DNA chips, tissue culturing automation, and mass spectrometry analysis are disruptive technologies that are in the early stages of application and should be tracked closely. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189
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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 company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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Oncotarget: Cutaneous apocrine sweat gland carcinoma

Scienmag

Rantala – rantala@misvik.com Oncotarget published “Ex vivo analysis of DNA repair targeting in extreme rare cutaneous apocrine sweat gland carcinoma” which reported a rare metastatic case with a PALB2 aberration identified previously as a […].

In-Vivo 89
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Moderna and Generation Bio partner for genetic medicines development

Pharmaceutical Technology

Under the terms of the deal, Moderna has purchased an option to license ctLNP and closed-ended DNA (ceDNA) technology from Generation Bio. Both companies will use the partnership for advancing in vivo immune cell targeting as a new class of genetic medicines.

Genetics 130
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Verve starts trials of cholesterol drug in test of base editing technique

pharmaphorum

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.