AAV immunogenicity and safety concerns - industry-wide scrutiny following adverse events in gene therapy trials could trigger FDA clinical holds or require costly protocol modifications
Manufacturing scalability challenges for clinical-grade AAV vectors with limited CDMO capacity industry-wide, potentially delaying trials
Regulatory pathway uncertainty for CNS gene therapies with limited precedent for approval standards and biomarker acceptance
Crowded Alzheimer's landscape with 150+ programs including established antibody therapies (Leqembi, Kisunla) and competing gene therapy approaches from better-capitalized competitors
Friedreich's ataxia competition from Reata Pharmaceuticals (Skyclarys approved), PTC Therapeutics, and other gene therapy developers with more advanced programs
Platform risk if competing AAV capsid technologies or alternative delivery modalities (lipid nanoparticles, exosomes) demonstrate superior CNS penetration or safety profiles
Significant dilution risk with 18-24 month cash runway requiring $75-150M equity raise by Q4 2026, likely at depressed valuation given -31% three-month performance
High cash burn rate of $100M annually with no near-term revenue, creating dependency on capital markets access during uncertain biotech funding environment
Minimal debt cushion (0.07 D/E) provides no alternative financing flexibility if equity markets become inaccessible
StructuralCompetitiveBalance Sheet