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Nusinersen

Infantile-onset Spinal Muscular Atrophy

nucleic_acidDormant
D
30/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
15/ 100
Competitive Position
95/ 100

Competitive context

5 Active assets in the Infantile-onset Spinal Muscular Atrophy 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
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

View all 9 →

Same molecule, other indications

Nusinersen is also rated in:

spinal muscular atrophyDormant
A
Indicative ·
82 / 100
View asset →
spinal muscular atrophy, type IIDormant
CCC
Indicative ·
53 / 100
View asset →
spinal muscular atrophy, type IIIDormant
CCC
Indicative ·
53 / 100
View asset →

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