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Conversations from ESMO Targeted Anticancer Therapies Congress 2024 

Drug Discovery World

DDW’s Megan Thomas caught up with Oliver Rausch, Chief Scientific Officer at Storm Therapeutics, following the ESMO Targeted Anticancer Therapies Congress 2024, a global meeting focusing on promising new anticancer targets and agents, focusing on those in early phase clinical development. MT: What’s next for STORM’s research?

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FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA)

The Pharma Data

Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. . More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings.

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

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The Achilles’ heel of cancer

Drug Discovery World

Genoscience Pharma’s Philippe Halfon and Eric Raymond share their expertise on targeting the recycling of unsustainable production of palmitoylated cancer-associated proteins. Palmitoyl transferases are a zinc finger Asp-His-His-Cystype (ZDHHC) family containing 23 distinct mammalian genes (excluding ZDHHC10) that catalyse this reaction 1.

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Nine for 2023, part two: healthcare’s hard problem, the prognosis for diagnosis, and key new pharmacotherapy platforms

pharmaphorum

There were developments in the past 12 months, true – Biogen/Eisai’s lecanemab (now branded Leqembi) showed clear success in the Clarity AD trial and received approval by FDA on 6th January 2023. This is linked to wider questions about the role damaged proteins and their build up plays in ageing across different tissues and organs.

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The Biotech Effect

Pharmaceutical Technology

“The inherent advantages of biopharma are that it allows you to pursue therapies against targets that are not small molecule therapies, for example by targeting protein-protein interactions and targeting GPCRs (G-protein coupled receptors),” he says. “Biotechnology has exploded across the industry. .

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Unlocking the potential of mRNA for the future treatment of rare diseases 

Drug Discovery World

With only 10% of these conditions indicated for an approved therapy, there is a sizable opportunity for scientists and companies to develop treatments for patients with no other options to turn to. Approximately 7,000 rare and ultra-rare diseases have been described, and yet, 90% have no approved therapeutic options 1.