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Raav9.Cmv.Hnaglu

Mucopolysaccharidosis Type 3B

Explore 2 assets in Mucopolysaccharidosis Type 3B →
Floatz Rating
CCC48/100
Confidence
Indicative
v0.2
Sponsor
Abeona Therapeutics, Inc
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Dormant
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Moderate confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
NCT03315182CT.gov
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
—Terminated———

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Mucopolysaccharidosis Type 3B

AssetSponsorPhaseRating
Raav9.Cmv.Hnaglu (this asset)Abeona Therapeutics, IncP1/2CCC · 48
AAV-2UniQure Biopharma B.V.P1/2CCC
Lesinidase AlfaAlexion Pharmaceuticals, Inc.P1/2CCC
ax 250Allievex CorporationP4NR
Tralesinidase AlfaSpruce BiosciencesP3NR

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Raav9.Cmv.Hnaglu in Mucopolysaccharidosis Type 3B. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/raav9cmvhnaglu-mucopolysaccharidosis-type-3b

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