Remove RNA Remove Science Remove Small Molecule
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Novartis Acquires Regulus Therapeutics, Expanding its RNA-Based Drug Pipeline

The Pharma Data

By integrating Regulus’ expertise in microRNA science with its own deep capabilities in clinical development, regulatory affairs, and commercialization, Novartis is well-positioned to accelerate farabursen’s clinical path and potentially bring a first-in-class treatment to ADPKD patients.

RNA 52
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RNA processing in health and disease: challenges and opportunities of the field

Drug Target Review

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing.

RNA 96
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Ribometrix and Genentech Partner in Potential $1 Billion+ RNA Deal

The Pharma Data

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins.

RNA 52
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Reaching cruising altitude: New discovery tools to target RNA

Dark Matter Blog

The majority of small molecule drugs induce their therapeutic effects by seeking out and binding to their intended target while avoiding most other molecules in the dense milieu of the cell interior. Our overall mission at Arrakis is to expand the set of “druggable” targets for small-molecule medicines to include RNA.

RNA 52
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From Impossible to Inevitable: Transforming Undruggable Targets

DrugBank

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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The rising impact of biomarkers in early clinical development

Drug Target Review

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

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Women in STEM with Juliet Williams

Drug Target Review

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Pushing the boundaries of science always opens the door to new, impactful developments, although there are always challenges along the way.