SLDB

Solid Biosciences Inc.

BS score 43.2LOWPHASE3 · mkt cap $797.0M · rev ttm $0

drug hypothesis

Unknown - microdystrophin gene therapy (specific product/company name not provided in filing) modulates Dystrophin deficiency (genetic replacement therapy - not a traditional druggable target) to treat Duchenne Muscular Dystrophy (DMD).

moa:AAV-vector mediated delivery of a truncated microdystrophin gene to skeletal and cardiac muscle cells, enabling production of a functional but shortened dystrophin protein to compensate for the mutated endogenous gene in DMD patients.

score breakdown

trial design70

base rate disconnect8

language red flags60

composite43.2

valuation analysis

market cap$797.0M

revenue ttm$0

phasePHASE3

historical base rate55%

disconnect ratio0.3x

lead trialNCT07148089

meta

cik0001707502

first discovered2026-04-09

last updated2026-04-10

statusACTIVE

llm model—

extracted at2026-04-10

trial design

Phase 1, non-randomized, single-arm, open-label study enrolling 12 pediatric patients with DMD to evaluate safety and exploratory efficacy endpoints including microdystrophin protein expression in muscle biopsies and functional assessments (NSAA, 6MWT, PUL, respiratory function, cardiac function).

primary endpoint:Number of Participants with Treatment Emergent Adverse Events (TEAEs) (Up to 5 years)

claimed differentiation

Not stated - no SEC filing text provided, only clinical trial metadata

language red flags

No SEC filing text provided to analyze for red flags
Trial design shows primary endpoint is safety-focused (TEAEs) rather than efficacy - typical for early Phase 1 but indicates very early stage of development
Small enrollment (n=12) limits statistical power for efficacy conclusions

company-stated risks

Not explicitly stated in provided data - typical gene therapy risks include immune response to vector, potential for insertional mutagenesis, limited durability of expression, and immune clearance of transduced cells

upcoming catalysts

2026-10-15Primary completion date