Bristol-Myers Squibb Buys Into Gene Therapy

Bristol-Myers Squibb and uniQure, a Dutch specialist in gene therapy, have formed a collaboration in which BMS will gain exclusive access to uniQure’s technology for use on multiple targets in cardiovascular disease.

Gene therapy is a technique for correcting defective or missing genes by inserting a functional gene into the cells of a patient.

The pact includes uniQure’s gene therapy program for congestive heart failure, which is intended to restore the heart’s ability to synthesize S100A1, a calcium sensor and regulator of heart function. Beyond cardiovascular disease, the agreement includes potential collaboration on gene therapy for up to 10 targets.

Under the terms of the agreement, uniQure will manage discovery efforts and be responsible for manufacturing clinical and commercial supplies using its insect-cell-based production technique. BMS will take charge of development and regulatory activities and of commercialization.

BMS will make a preliminary payment of about $100 million, which will cover the selection of three targets for research in addition to S100A1. UniQure could receive further R&D and regulatory milestone payments of hundreds of millions of dollars.

Collaborating with uniQure “further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases, including cardiovascular diseases,” says Carl P. Decicco, head of discovery research at BMS.

Gene therapy has garnered the attention of other major pharmaceutical firms in recent months. Earlier this year Genzyme, the rare disease division of Sanofi, signed with Voyager Therapeutics to develop gene therapies for central nervous system diseases. And Pfizer began work in December with Spark Therapeutics on a gene therapy treatment for hemophilia B.