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CGTs address rare and complex diseases at the root cause with increasing use in cancer, genetic disorders, and autoimmune diseases. While clinicalsupply is essential to any successful trial, autologous cell therapy trials occupy the far end of the spectrum regarding risk tolerance.
By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.
Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. Cell and gene therapies are not your typical clinicalsupply chain.
Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.
Similarly, Abecma’s boxed warning includes a description that outlines occurrences of T cell malignancies observed after treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies like Abecma. In addition, the companies are also gunning for a first-line setting approval.
“Africa offers effective, innovative and cost-effective ways to execute clinical trials,” explains Rob van den Bergh, Managing Director of Oximio’s sub-Saharan Africa division, which delivers comprehensive services in clinical trial logistics. “It’s
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