Spotlight Bio \USA

A vertically integrated AI-native drug target discovery platform focused exclusively on rare genetic diseases, leveraging patient genomic data, foundation models (AlphaFold3, ESM, GPT-4), and rapid CRO-partnered validation to identify and de-risk novel targets for pharma partnerships. The wedge is hyper-specific: rare diseases with known genetic mutations but no approved therapies, where patient advocacy groups can provide genomic data and pharma partners are desperate for validated targets. Unlike Spotlight's horizontal approach, TargetForge focuses on 10-20 high-value rare diseases (e.g., ALS, Huntington's, certain muscular dystrophies) where genetic causality is clear but druggable targets are unknown. The platform combines: (1) Patient genomic data partnerships (advocacy groups, biobanks), (2) Foundation model analysis (AlphaFold3 for structure, ESM for sequence, GPT-4 for literature synthesis), (3) Rapid in-silico screening (molecular dynamics, docking), (4) CRO-partnered validation (Charles River, Covance for animal models), (5) Pharma co-development deals structured for early milestones. Revenue model: upfront fees ($2-5M per target), milestone payments tied to IND filing and Phase 1 ($10-30M per target), and equity in spun-out therapeutic companies. The modern tech stack dramatically reduces time and cost: AlphaFold3 eliminates months of structure determination, GPT-4 synthesizes decades of literature in hours, cloud compute (AWS, GCP) enables massive parallel screening, and CRO partnerships eliminate the need for in-house labs. The business targets 5-10 validated targets within 3-4 years, each with a pharma partner, creating a portfolio approach that de-risks any single target failure. Exit options: acquisition by pharma (Roche, Novartis, Takeda all acquire rare disease platforms), IPO after demonstrating multiple IND filings, or spin-out of individual therapeutic companies.