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bmd003

Gene Therapy

Peking Union Medical College Hospitalnucleic_acidEarly Phase 1Active
C
39/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
24/ 100
Competitive Position
95/ 100

Competitive context

3 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
Valoctocogene Roxaparvovec
BioMarin Pharmaceutical
Phase 1/2CC
Kln-1010
Kelonia Therapeutics, Inc.
Phase 1CC
Clozapine
Ruijin Hospital
Early Phase 1C
Of Gs001
Institute of Hematology & Blood Diseases Hospital, China
N/AD

Same molecule, other indications

bmd003 is also rated in:

cystic fibrosisDormant
C
Indicative ·
38 / 100
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cystic fibrosisDormant
C
Indicative ·
36 / 100
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