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BioMarin Reports Encouraging Data In Hemophilia A Gene Therapy Program

BioMarin Pharmaceutical Inc. (BMRN) announced encouraging preliminary data from an ongoing Phase 1/2 clinical trial with BMN 270, an investigational gene therapy treatment for hemophilia A.

The company said that a total of eight patients with severe hemophilia A received a single dose of BMN 270, six of whom have been treated at the highest dose of 6 x 1013 vg/kg, and to date, post-treatment follow-up ranges from five to 16 weeks.

Patients with hemophilia A are not able to produce enough functional Factor VIII to prevent bleeding, and are currently treated with prophylactic or on-demand infusions of plasma-derived or recombinant Factor VIII.

At last observation, patients at the highest dose experienced increasing Factor VIII activity levels ranging between 4 and 60 percent, with five of six patients treated at the high dose showing factor VIII levels above 5 percent and two of six showing increasing levels of factor VIII at over 50 percent.

All the high dose patients improved from severe to either moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.

"We are encouraged by this early data on BMN 270 and the trend we are seeing in increasing Factor VIII levels over time. BMN 270 could have the potential to reduce and possibly eliminate the need for infusions of Factor VIII," said Hank Fuchs, Chief Medical Officer at BioMarin.

BioMarin plans to discuss these findings with UK regulatory authorities prior to dosing the remaining patients.

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