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Find Clinical Drug Pipeline Developments & Deals for Valoctocogene Roxaparvovec
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions.
Roctavian (valoctocogene roxaparvovec) also received orphan drug designation from the EMA and FDA for severe hemophilia A. Phase 1/2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors.
The GENEr8-1 Phase 3 study for valoctocogene roxaparvovec demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate.
Overall, to date, a single 6e13 vg/kg dose of BMN 270 (valoctocogene roxaparvovec) has been well tolerated with no delayed-onset treatment related adverse events.
Overall, single 6e13 vg/kg dose of Roctavian (valoctocogene roxaparvovec) has been well tolerated with no delayed-onset treatment related adverse events.
European Commission has granted conditional MA to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for treatment of severe hemophilia A in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5.
BMN 270 (valoctocogene roxaparvovec) (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
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