Biomarin Pharmaceutical
Clinical trials sponsored by Biomarin Pharmaceutical, explained in plain language.
A biotechnology company developing therapies for rare genetic diseases.
Trade on eToro (ad)Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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One-shot gene therapy aims to free hemophilia patients from constant infusions
Disease control CompletedThis trial tests a gene therapy called valoctocogene roxaparvovec for people with severe hemophilia A who have developed inhibitors (antibodies that block standard treatment). The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor VIII, so the…
Phase: PHASE1, PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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One-Time gene therapy shows promise for hemophilia a
Disease control CompletedThis study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The treatment aims to help the body produce its own clotting factor VIII, reducing the need for regular infusions. Participants also received steroids to manage immune re…
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC