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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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BioMed X and Sanofi partner to leverage AI for drug development

Pharmaceutical Technology

The companies will jointly create a virtual patient engine for the drug candidates’ clinical translation. The partnership intends to address a 90% failure rate of new therapy candidates in clinical development, which is a key bottleneck of the pharmaceutical industry.

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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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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 99
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Evotec and Variant Bio partner to develop fibrosis treatments

Drug Discovery World

Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.

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New research highlights potential of combination cancer treatment 

Drug Discovery World

iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumour survival pathways, has published new research data in the peer-reviewed journal Cancer Research, that supports a strategy of combining autotaxin inhibitor IOA-289 with TGF-β pathway inhibitor IOA-359.

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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

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.