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Scribe and Sanofi expand genetic therapy development deal

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have expanded partnership to progress the development of in vivo genetic therapies to treat genomic diseases.

Genetics 246
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Single-course in vivo base editing therapy proven to lower cholesterol

Drug Discovery World

Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. “Of

In-Vivo 52
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FDA clears in vivo gene editing therapy for trials in US

Drug Discovery World

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW). The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE).

In-Vivo 52
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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52
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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 92
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ELRIG meeting will focus on genome engineering in drug discovery

Drug Discovery World

The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for target validation, and its applications in therapeutic genome editing. Register here.

Genome 52
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Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Roots Analysis

The global genome editing market is anticipated to grow at a CAGR of 12.6% The global genome editing market is anticipated to grow at a CAGR of 12.6% Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. during the forecast period 2023-2035. during the forecast period 2023-2035.

Genome 40