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

Drug Discovery World

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 The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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Moderna and Generation Bio partner for genetic medicines development

Pharmaceutical Technology

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

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State of Play: A closer look at hotspots of emerging biotech research

Bio Pharma Dive

In this continuing series, BioPharma Dive examines new areas of biotech investment and startup activity, such as next-generation RNA medicines, TYK2 inhibitors and in vivo CAR-T.

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

XTalks

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

In-Vivo 52
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Intellia, beginning treatment in CRISPR study, secures Gates Foundation backing

Bio Pharma Dive

The biotech won a grant from the foundation two days after dosing the first patient in a study of an in vivo CRISPR medicine, a milestone for the company and the technology.

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