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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)

Spinal Muscular Atrophy Type I

Novartis Gene Therapiesgene_therapyPhase 3Active
BB
64/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
49/ 100
Competitive Position
95/ 100

Competitive context

4 Active assets in the Spinal Muscular Atrophy Type I 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 3BB
exg001-307
Guangzhou Jiayin Biotech Ltd
Phase 1/2CCC
Valproic Acid
University of Utah
Phase 1/2CCC
Levocarnitine
University of Utah
Phase 1/2CCC
skg0201
Lanyue Biotech (Hangzhou) Co., Ltd.
Phase 1/2CCC

View all 9 →

Same molecule, other indications

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) is also rated in:

spinal muscular atrophyDormant
A
Indicative ·
80 / 100
View asset →
spinal muscular atrophy, type 1Dormant
BB
Indicative ·
69 / 100
View asset →
Gene TherapyDormant
B
Indicative ·
62 / 100
View asset →

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