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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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haematological malignancies
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AstraZeneca to advance haematology cell therapy
The new acquisition grants AstraZeneca rights to a new CAR-T cell therapy with a differentiated manufacturing process that could provide a potential best-in-class blood cancer treatment.
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FDA approves new therapy for myelodysplastic syndromes
The first targeted therapy for certain adults with the rare blood cancer myelodysplastic syndromes, has been approved by the US Food and Drug Administration (FDA).
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Astellas to advance oncology CAR-T therapy
An investment of $50 million by Astellas Pharma in Poseida Therapeutics’ Phase I allogeneic CAR-T product candidate for solid tumour indications is set to advance the cancer immunotherapy field.
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$1.7b acquisition to develop rare haematology therapy
When CTI BioPharma is acquired by Swedish Orphan Biovitrum AB (Sobi), the companies intend to advance an oral kinase inhibitor treatment for a rare haematological disease.
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FDA approves allogeneic stem cell transplant therapy
The first allogeneic stem cell transplant therapy to be approved based on Phase III study results has been granted by the US Food and Drug Administration (FDA).
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GOSH and Leucid to manufacture novel CAR T therapy
Great Ormond Street Hospital and Leucid Bio will manufacture a novel lateral CAR targeting NKG2D ligands, under a first-of-its-kind manufacturing agreement.
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UK ATMP clinical trials report 2022 published
T cells were revealed as the most commonly investigated cell type continuing to increase, found CGT Catapult's 2022 UK ATMP clinical trials report.
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Immunotherapy shows benefits for hospitalised COVID-19 patients
Apogenix’s asunercept demonstrates efficacy in Phase II trial for the treatment of hospitalised COVID-19 patients.
