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A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. Using gene therapy, their approach was beautifully straightforward – “Missing an important gene? The agent, onasemnogene abeparvovec, was approved by the FDA in 2019 and is marketed as Zolgensma.

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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Sosei Heptares and Biohaven Enter Global Collaboration and License Agreement to Advance Novel Small-Molecule CGRP Antagonist Portfolio

The Pharma Data

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. We look forward to working on this new collaboration with Biohaven, a world leader in the clinical development of CGRP-targeted therapies.” TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,

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Antibody-drug conjugates payloads: then, now and next

Drug Target Review

Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 6 Combining the effect of payloads with different mechanisms of action – an approach that revolutionised small molecule chemotherapy – also holds the promise of enhanced therapeutic activity for ADCs.

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The new approach in cancer therapy with innovative mechanism-of-action for ferroptosis induction

The Pharma Data

The new approach in cancer therapy with innovative mechanism-of-action for ferroptosis induction A team of researchers led by Dr. Marcus Conrad from Helmholtz Munich discovered a novel anti-cancer drug, called icFSP1, which sensitizes cancer cells to ferroptosis.

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Johns Hopkins Researchers Identify CRISPR Dimmer

The Pharma Data

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. In particular, Editas is known to be exploring tunable CRISPR-Cas9 systems for therapies has developed self-inactivating Cas9 adeno-associated virus vectors.

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Sumitovant to Snap Up Development Partner Urovant Sciences

The Pharma Data

As its name aptly suggests, Urovant is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for urologic conditions such as overactive bladder (OAB) and maladies involving adjacent areas of the human anatomy. . The condition is most prevalent in men. .

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