Branaplam

Spinal Muscular Atrophy

Novartis PharmaceuticalsSmall moleculePhase 1/2Dormant

CCC

50/ 100

Provisional rating · Methodology v0.2

Rating breakdown

22 Active assets in the Spinal Muscular Atrophy cohort.

Asset / Sponsor	Phase	Rating
Nusinersen Scholar Rock, Inc.	Phase 4	A
Risdiplam Scholar Rock, Inc.	Phase 4	A
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 Pharmaceuticals	Phase 4	A
oav101 Novartis Pharmaceuticals	Phase 4	BBB
Srk-015 Scholar Rock, Inc.	Phase 3	BB

View all 40 →

Branaplam is also rated in:

Huntington diseaseDormant

CCC

Indicative ·

53 / 100

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