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PharmaShots Weekly Snapshots (January 23 - 27, 2023)

PharmaShots

Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. That comes to an end today however, as Novartis is winnowing down the countries where the gMAP pathway will be available.

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Cannabinoids receptors: popular preclinical target but banned in 137 countries

Pharmaceutical Technology

In 2022, a surge in the pipeline has led to cannabinoid receptors becoming the most popular target in preclinical development. Despite this, the medical use of cannabinoid drugs is heavily restricted, including being banned in 137 countries, according to the United Nations. This is closely followed by CB2 receptors in second place.

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Jolly Good/Teijin Pharma develop VR digital therapeutics for depression

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Technology firm Jolly Good and Teijin Pharma have begun a partnership to develop virtual reality digital therapeutics (VR DTx) for major depressive disorder. Jolly Good is a medical technology company that develops VR solutions and AI-based medical and welfare services that analyse user behaviour in the VR space.

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Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

The commercial investment required to research and develop an innovative drug, prove its safety and efficacy, and bring it to market is staggering. Most famously, the US passed its Orphan Drug Act in 1983, providing innovators with financial motivation to develop orphan drugs and meet the needs of these forgotten patients.

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Oslo Medicines Initiative asks how to balance innovation incentivisation with security of access

pharmaphorum

Access to medicines is “one of the most challenging policy areas in every country in the European region” – it’s time to develop a solution that works for everyone. The outputs of the innovation process are proving prohibitively expensive for high-income countries, and way beyond the reach of low- and middle-income countries.”.

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Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. This includes the first potential approval of a CRISPR-based gene therapy called exa-cel , which is developed by CRISPR Therapeutics and Vertex Pharmaceuticals.