Srk-015

Spinal Muscular Atrophy Type 3

Scholar Rock, Inc.Phase 3Active

67/ 100

Provisional rating · Methodology v0.2

Rating breakdown

4 Active assets in the Spinal Muscular Atrophy Type 3 cohort.

Asset / Sponsor	Phase	Rating
Pyridostigmine UMC Utrecht	Phase 2	B
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	B
Pyridostigmine Centre Hospitalier Régional de la Citadelle	Phase 2	B
OLESOXIME, E- Hoffmann-La Roche	Phase 2	B
Risdiplam Scholar Rock, Inc.	Phase 2	CCC