Dfn-15

Spinal Muscular Atrophy (SMA)

Hugh McMillanPhase 2Dormant

CCC

53/ 100

Provisional rating · Methodology v0.2

Rating breakdown

22 Active assets in the Spinal Muscular Atrophy (SMA) 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