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.

turtle — Gene therapies are making slow progress commercially • Source: Shutterstock

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.

Complex administration and reimbursement of the expensive therapeutics remain barriers to broad uptake, but a growing pipeline of patients are moving closer to receiving infusions

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