With a growing number of complex cell and gene therapies approved globally, mostly for rare disease populations, manufacturers are looking at how to make these products more accessible by delivering available treatments through a greater number of health care centers and by developing new treatments for larger patient populations.
Manufacturers Eye Challenges Of Broadening Cell And Gene Therapy Access
Potential Expansion From Academia To Community
With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.
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The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.
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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.
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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.
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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.