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Risdiplam

Spinal Muscular Atrophy Type 2

Scholar Rock, Inc.Small moleculePhase 2Active
CCC
53/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
38/ 100
Competitive Position
89/ 100

Competitive context

6 Active assets in the Spinal Muscular Atrophy Type 2 cohort.

Asset / SponsorPhaseRating
Srk-015
Scholar Rock, Inc.
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 3B
OLESOXIME, E-
Hoffmann-La Roche
Phase 2B
Nusinersen
Scholar Rock, Inc.
Phase 2CCC
gc101
GeneCradle Inc
Phase 1/2CCC
Sodium Phenylbutyrate
Westat
Phase 1/2CCC
Gcb-001
Genecombio Ltd.
N/AD
oav101
Novartis Pharmaceuticals
Phase 3NR

Same molecule, other indications

Risdiplam is also rated in:

spinal muscular atrophyDormant
A
Indicative ·
80 / 100
View asset →
spinal muscular atrophy, type IIIDormant
CCC
Indicative ·
53 / 100
View asset →
neuromuscular diseaseDormant
CCC
Indicative ·
53 / 100
View asset →

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