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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. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza.

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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. . Global dementia prevalence is expected to increase to 152 million in 2050, a 204% increase compared to 2018.

Disease 130
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Three trends in the antibody-drug conjugate (ADC) marketĀ 

Drug Discovery World

Antibody-drug conjugates (ADCs) are biopharmaceutical products in which a monoclonal antibody (mAB) is linked to a small molecule drug with a stable linker 1. According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1.

Marketing 245
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Metabolism of 2023 FDA Approved Small Molecules ā€“ PART 1

Metabolite Tales Blog

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals.

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Metabolism of 2022 FDA approved small molecule drugs PART 2

Metabolite Tales Blog

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. Dermavantā€™s tapinarof is one such friend.

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Breaking C-F bonds in drugs

Metabolite Tales Blog

In 2021, almost one third of FDA approved drugs contained at least one fluorine. Even newer from the pharma benches are 12 small molecule drugs highlighted by Chris de Savi [4], whose structures were first disclosed at the ACS and AACR meetings in Q1 2023. Of these 12 drugs, 9 contain at least one fluorine! Elizabeth A.

Drugs 52
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The evolution of the RNA therapeutics landscapeĀ 

Drug Discovery World

With the knowledge that RNA can adopt a diverse range of structural shapes, we can now develop therapeutics using RNAs 3D structure, similar to creating antibody therapies or small molecules that can target surfaces on proteins.

RNA 147