exg001-307

Spinal Muscular Atrophy Type I

Guangzhou Jiayin Biotech LtdPhase 1/2Active

CCC

54/ 100

Provisional rating · Methodology v0.2

Rating breakdown

4 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
Levocarnitine University of Utah	Phase 1/2	CCC
Valproic Acid University of Utah	Phase 1/2	CCC
gb221 Gemma Biotherapeutics	Phase 1/2	CCC

View all 9 →

exg001-307 is also rated in:

spinal muscular atrophyDormant

Indicative ·

39 / 100

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