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Valoctocogene Roxaparvovec

Blood Disorder

BioMarin Pharmaceuticalgene_therapyPhase 1/2Dormant
CCC
48/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
33/ 100
Competitive Position
95/ 100

Competitive context

32 Active assets in the Blood Disorder cohort.

Asset / SponsorPhaseRating
Cyclophosphamide
Baptist Health South Florida
Phase 2/3BBB
Fludarabine
Baptist Health South Florida
Phase 2BB
Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA) introduced transiently as ribonucleoprotein (RNP) complex, targeting the erythroid lineage-specific enhancer region of BCL11A (B-cell lymphoma/leukemia 11A). The site-specific cleavage by Cas9 forms a double strand break (DSB), which is subsequently repaired by nonhomologous end-joining (NHEJ), leading to the transcriptional repression of BCL11A, a repressor of ?-globin gene transcription
Vertex Pharmaceuticals Incorporated
Phase 3BB
Busulphan
Baptist Health South Florida
Phase 2BB
Kogenate
Bayer
Phase 4BB

View all 50 →

Same molecule, other indications

Valoctocogene Roxaparvovec is also rated in:

hemophilia ADormant
BB
Indicative ·
64 / 100
View asset →
severe hemophilia ADormant
CCC
Indicative ·
52 / 100
View asset →
blood coagulation diseaseDormant
CCC
Indicative ·
48 / 100
View asset →

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