The main rule of the cell and gene therapy market is there are few hard and fast rules when it comes to how to manufacture, distribute, administer and price these potentially curative treatments for cancer and rare diseases.
Cell And Gene Therapies: Where Few Standard Rules Apply
Manufacturing, distribution and pricing challenges for cell and gene therapies have been mostly hypothetical – until now. But with no industry standards, the first products to win approval are dictating the complex path forward.
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Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.
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Kyoto-based venture moves HQ to California to expand R&D and business outreach for its regulatory T-cell technology, as it raises around $46m in public and private funding.
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The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
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Miltenyi’s leadership calls for a streamlined regulatory framework as India gears to scale in cell and gene therapy. Executives from the German group also talked to Scrip about helping advance local capabilities, delivering point-of-care CAR- T cell therapy to hospitals and pricing dynamics.
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The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
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The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
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Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.