Pediatric Gene Therapy Family Decision Companion

Impossibility Negation: 2 years ago a family of a 3-year-old with sickle cell disease could not walk through a $2.2M gene therapy decision at home. FDA expanded Casgevy to age 2+ for SCD in July 2026 — the old impossibility is now gone. AI grounded on the specific label + treatment protocol can hold a multi-week guided decision journey with a family.

This is NOT diagnosis or prescribing. Decision-preparation service operating outside FDA medical device pathway if scoped correctly. Casgevy authorized treatment centers are only 50-70 in the US — rural families face months of decision inertia.

Genome Medical (telehealth genetic counselors) — real competitor if they add a Casgevy decision aid. Vertex Casgevy Cares — manufacturer program, conflicted. SCDAA education-focused, not personalized. SCRHN exists but education only. No independent AI decision aid for pediatric gene therapy families found.

G141 lurks: Vertex has every incentive to build the decision aid itself. Vertex Casgevy Cares already provides financial navigation. Independent version has ~12-18 months window before Vertex ships it bundled and free.

G132 fires: pediatric SCD pool = 5,000-8,000 US families/year eligible. Revenue per family: $30-60/month × 6 months = $180-360. Max revenue: $2.9M/year ceiling. Thin TAM trap.

Kill reason positional (customer segment too narrow). Pivot: expand from pediatric SCD to all pediatric gene therapies (Zolgensma SMA, Luxturna, Casgevy, LentiGlobin, Elevidys Duchenne). Pass 1 estimate: 40-60K families/year = $8-12M ceiling.

Pivoted to all pediatric gene therapies. Independent differentiator: shows alternatives including watchful waiting. Manufacturer programs will never show their own alternatives. Conviction 38%.

Casgevy age 2+ expansion for SCD/TDT confirmed July 2026 (FDA + Vertex press release). US SCD pediatric pool confirmed ~2-3K new eligible families/year (not 5-8K). Genome Medical competitive threat confirmed. Vertex Casgevy Cares financial navigation confirmed.

Pass 1 estimated 40-60K families/year across all pediatric gene therapies. Verified actual: Zolgensma ~300/year (SMA is very rare), Luxturna ~50/year, Casgevy pediatric SCD ~2-3K/year, LentiGlobin ~200/year, Elevidys ~2-3K/year. Real verified total: 8-15K/year — 4x smaller than Pass 1 estimate.

DISTRIBUTION gate failure: Facebook ads to rare-disease parents cost $80-200 per qualified lead (verified in adjacent rare-disease benchmarks). LTV = $180/family. Negative-margin CAC. SCDAA convention 1K attendees — fraction are relevant parents. Single-partner dependency (Sickle Cell 101 or SCDAA) = fatal if partner doesn't convert. Even expanded to all pediatric gene therapies: 8-15K families/year ceiling with negative CAC economics.

Killed at DISTRIBUTION gate. Verified TAM (8-15K families/year) is 4x smaller than Pass 1 estimate. Combined with $80-200 Facebook CAC vs $180 LTV and single-partner dependency, no path to $10M+ ARR. Genuinely useful service, not a venture-scale product.