BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases – Endpoints News

BioMarin has just struck a deal to back a Swiss biotech startup with some deep ties to top research institution UCL in London as it beefs up the swelling gene therapy portion of the pipeline.

We dont have any terms to deal with, just the knowledge that BioMarin CEO JJ Bienaim saw enough of DiNAQORs work to invest in the company as it licenses their lead preclinical program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy, while collaborating on the rest of the pipeline.

DiNAQOR barely rippled the pond with its launch a year ago with its base in Pfffikon, Switzerland and operations in London and Boston. Part of those ties belong to UCL, where CMO Valeria Ricotti is an MD in pediatrics with experience running gene therapy studies. It set up a manufacturing pact with Lonzas cell and gene therapy unit in Houston last fall as it looked to jump into the clinic with treatments for monogenic cardiomyopathies.

The pact marks a significant expansion in the gene therapy portion of the pipeline at BioMarin. Just days ago the biotech reported that the FDA is providing an accelerated review for the hemophilia A drug valrox, which will likely speed the arrival of a new drug that will test the limits of pricing in the field.

Bienaim has said the company is considering a price range of $2 million to $3 million for valrox, even though there are lingering doubts on just how long it can remain effective.

BioMarin built its rep on rare diseases, and clearly sees a much bigger future in gene therapy.

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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases - Endpoints News