Valproic Acid

Spinal Muscular Atrophy Type I

University of UtahSmall moleculePhase 1/2Dormant

CCC

52/ 100

Provisional rating · Methodology v0.2

Rating breakdown

5 Active assets in the Spinal Muscular Atrophy Type I cohort.

Asset / Sponsor	Phase	Rating
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 3	BB
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 3	BB
exg001-307 Guangzhou Jiayin Biotech Ltd	Phase 1/2	CCC
Levocarnitine University of Utah	Phase 1/2	CCC
skg0201 Lanyue Biotech (Hangzhou) Co., Ltd.	Phase 1/2	CCC