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Use of MAPK Pathway Inhibitors for the Treatment of Friedreich Ataxia

University of Pennsylvania Clinical Small Molecule
Probability of Success

49% · grade B · Medium data confidence

Model PoS 49% (grade B, medium data confidence). Strongest signals: Data maturity, IP position. Biggest drag: Translational evidence.

Data maturity (22%) 76

Clinical; clinical reference.

IP position (16%) 72

Patent filed/pending -- prosecution ongoing.

Modality feasibility (14%) 55

Cell/gene therapy -- high upside, heavy manufacturing and regulatory load.

Indication pull (14%) 78

Neurology, Rare Disease -- active buyer appetite.

Competitive whitespace (12%) 86

Under-served segment; multi-modal differentiation.

Translational evidence (12%) 62

Public evidence mentions: clinical, patient.

Licensing readiness (10%) 94

Listed as available for licensing; established tech-transfer office; 1 public source link(s).

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