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Valoctocogene Roxaparvovec

Gene Therapy

BioMarin Pharmaceuticalgene_therapyPhase 1/2Dormant
CC
47/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
33/ 100
Competitive Position
89/ 100

Competitive context

4 Active assets in the Gene Therapy cohort.

Asset / SponsorPhaseRating
NON-REPLICATING RECOMBINANT SELF-COMPLEMENTARY (SC) ADENO-ASSOCIATED VIRUS SEROTYPE 9 (AAV9) VECTOR CONTAINING THE CDNA OF THE SURVIVAL OF MOTOR NEURON 2 (SMN2) GENE UNDER THE CONTROL OF THE HYBRID CYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN BETA-ACTIN PROMOTER (CBA)
Novartis Gene Therapies
Phase 3B
Spk-8016
Spark Therapeutics, Inc.
Phase 1/2CCC
Kln-1010
Kelonia Therapeutics, Inc.
Phase 1CC
bmd003
Peking Union Medical College Hospital
Early Phase 1C
Clozapine
Ruijin Hospital
Early Phase 1C
Of Gs001
Institute of Hematology & Blood Diseases Hospital, China
N/AD

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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