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Merck KGaA and C4 Therapeutics enter protein degrader partnership worth $756m

The collaboration will utilise C4T’s TORPEDO platform to target and degrade disease-associated proteins

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Novartis shares positive results for spinal muscular atrophy gene therapy Zolgensma

The company said the results support the use of the one-time therapy in older and heavier children

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GSK’s Jemperli combination recommended by NICE as first-line endometrial cancer treatment

Approximately 580 patients will now be eligible for treatment with the PD-1 inhibitor

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Sanofi’s Rezurock fast-tracked by NHS for rare chronic graft versus host disease

Around 400 stem cell or bone marrow transplant patients will develop the condition each year in England

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First UK cancer patients receive Moderna’s experimental mRNA therapy

The Mobilize trial is evaluating mRNA-4359 in cancers including melanoma and lung cancer

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Radboud researchers use new method to improve diagnosis of rare diseases

Approximately 7,000 rare diseases collectively impact up to 5.9% of the global population

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OneChain-led consortium granted €1.9m to advance CAR-T therapy for rare leukaemia

Acute lymphoblastic leukaemia has an incidence of around 1.5 cases per 100,000 people

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Clinigen launches ‘What is Possible?’ campaign on Rare Disease Day to accelerate access to medicines

There are over 7,000 rare diseases impacting more than 300 million people globally

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BeiGene’s tislelizumab receives CHMP recommendation for non-small cell lung cancer

Lung cancer is the second most common type of cancer and the leading cause of cancer-related death globally

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ICR study reveals AstraZeneca’s ceralasertib sensitises tumours to immunotherapy

Results showed that the drug stopped tumour growth in more than half of patients

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Novo Nordisk and Neomorph enter molecular glue degrader partnership worth over $1.4bn

The companies will work on therapies for cardiometabolic and rare diseases

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AstraZeneca’s Voydeya receives CHMP recommendation for rare blood disease PNH

Up to 20% of PNH patients treated with a C5 inhibitor experience clinically significant extravascular haemolysis

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