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

Drug Discovery World

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. FH is one of the most common genetic conditions, affecting around one in 300 people globally.

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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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Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

pharmaphorum

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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Astellas teams up with UK biotech Mogrify on hearing loss project

pharmaphorum

Cell therapy specialist Mogrify has struck a deal with Japanese drugmaker Astellas to look at ways to deploy regenerative medicine to treat hearing loss caused by factors such as chronic exposure to loud noises. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide.

In-Vivo 109
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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147
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Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.