Science Matters: Targeting Heme Trafficking In A Variety Of Diseases
A better understanding of heme trafficking could lead to drug targets in metabolic diseases, anemias, porphyrias, hemolytic diseases such as sickle cell disease – even parasitic infections.
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Givosiran was approved by the US FDA for the treatment of acute hepatic porphyria, a rare generic blood disorder, nearly three months ahead of the action date.
Higher success rates in rare cancers, where biomarkers can better direct therapy, characterize the oncology treatment landscape. Deal-making is favoring alliances over acquisitions as drugmakers focus on establishing the clinical benefit of new targets, particularly for rare indications.
The 2021 collaboration between Abbvie and Regenxbio to develop an anti-VEGF gene therapy for chronic retinal diseases is a noteworthy deal in the gene therapy space. But for both clinical and commercial reasons, it is a one-off opportunity and not an indicator of the overall potential of gene therapies to address eye diseases. Interest in optogenetics, however, which is the introduction of light-sensing genes into cells in the eye to restore vison, may be on the rise.