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Raav2/5-Hnaglu

Sanfilippo Syndrome B

UniQure Biopharma B.V.gene_therapyPhase 1/2Dormant
CCC
52/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
37/ 100
Competitive Position
95/ 100

Competitive context

2 Active assets in the Sanfilippo Syndrome B cohort.

Asset / SponsorPhaseRating
LESINIDASE ALFA
Alexion Pharmaceuticals, Inc.
Phase 1/2CCC
Raav9.Cmv.Hnaglu
Abeona Therapeutics, Inc
Phase 1/2CCC
ax 250
Allievex Corporation
Phase 4NR
Tralesinidase Alfa
Spruce Biosciences
Phase 3NR

Same molecule, other indications

Raav2/5-Hnaglu is also rated in:

hemophilia ADormant
CC
Indicative ·
43 / 100
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