The development of a commercial market for gene therapies is unfolding in real time, and progress remains slow. Several developers of new gene therapies – bluebird bio, Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc. and BioMarin Pharmaceutical Inc. – updated investors on launch progress in connection with first quarter sales and earnings results.
Gene Therapy Q1 Roundup: The Struggle Is Real
Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.
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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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Amgen’s AI model to predict proteins in the “bright Goldilocks zone of viscosity” with over 80% accuracy minus a wet lab experiment holds immense potential, VP Research Haldar said at an event as he spoke about a “hinge moment” for AI in pharma
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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.
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Amgen’s AI model to predict proteins in the “bright Goldilocks zone of viscosity” with over 80% accuracy minus a wet lab experiment holds immense potential, VP Research Haldar said at an event as he spoke about a “hinge moment” for AI in pharma