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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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Data shows new therapy successfully targets solid tumours

Drug Discovery World

Avacta Group has presented pre-clinical data describing the novel pre|CISION proteasome inhibitor, AVA3996, at the 2023 American Association for Cancer Research (AACR) Annual Meeting in Florida, US. While the global proteasome inhibitors market is expected to reach nearly $2.3

In-Vivo 97
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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147
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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1.

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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver.

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Taking a new approach to tackle neurodegenerative diseases

Drug Discovery World

Using its proprietary compound screening platform, Oligomerix discovers and develops differentiated, oral, small molecule inhibitors of tau self-association that are easy to administer and cost-effective. . OLX-07010 has been well characterised through an extensive program of pre-clinical toxicology and metabolism testing. .

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Evusheld long-acting antibody combination approved in the EU for pre-exposure prophylaxis (prevention) of COVID-19 in a broad population

The Pharma Data

Evusheld significantly reduced the risk of developing symptomatic COVID-19 in PROVENT Phase III trial, with protection lasting at least six months Evusheld retains neutralising activity against the Omicron BA.2 6,7 This study also showed Evusheld reduced viral burden and limited inflammation in the lungs (in vivo) across all Omicron variants.6.