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exg001-307

Spinal Muscular Atrophy (SMA)

Guangzhou Jiayin Biotech LtdEarly Phase 1Active
C
39/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
24/ 100
Competitive Position
95/ 100

Competitive context

21 Active assets in the Spinal Muscular Atrophy (SMA) cohort.

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

View all 40 →

Same molecule, other indications

exg001-307 is also rated in:

spinal muscular atrophy, type 1Dormant
CCC
Indicative ·
54 / 100
View asset →

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