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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

FH is one of the most common genetic conditions, affecting around one in 300 people globally. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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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New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing

BioTech 365

New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting … Continue reading →

In-Vivo 40
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Start-up aims to advance transformative genetic medicines

Drug Discovery World

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).

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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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Evotec and Variant Bio partner to develop fibrosis treatments

Drug Discovery World

The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery. The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery.