Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Bmn 307 Human Liver-Specific Transcription Regulatory Element Plus Human Phenylalanine 4-Hydroxylase Codon-Optimized Gene Hpah Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks)
Phenylketonuria
Explore 18 assets in Phenylketonuria →Floatz Rating
CC47/100
Confidence
Indicative
Sponsor
BioMarin Pharmaceutical
Modality
gene_therapy
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04480567CT.gov AAV Gene Therapy Study for Subjects with PKU | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Phenylketonuria
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Bmn 307 Human Liver-Specific Transcription Regulatory Element Plus Human Phenylalanine 4-Hydroxylase Codon-Optimized Gene Hpah Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks) (this asset) | BioMarin Pharmaceutical | P1/2 | CC · 47 |
| Sapropterin Dihydrochloride | Sohag University | P4 | A |
| Phenylase | BioMarin Pharmaceutical | P4 | A |
| Ravpal-Peg | BioMarin Pharmaceutical | P2 | BB |
| Repinatrabit | Otsuka Pharmaceutical Development & Commercialization, Inc. | P3 | BB |
| Escherichia Coli Strain Synb1618 | Synlogic | P2 | BB |
| L-Carnitine | Mansoura University | P4 | BB |
| Bmn 165 | BioMarin Pharmaceutical | P3 | B |
| Sepiapterin | PTC Therapeutics | P3 | B |
+18 more in the Phenylketonuria cohort
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. Dna (Recombinant Replication-Deficient Adeno-Associated Virus 5 Vector Bmn 307 Human Liver-Specific Transcription Regulatory Element Plus Human Phenylalanine 4-Hydroxylase Codon-Optimized Gene Hpah Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks) in Phenylketonuria. 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/dna-recombinant-replication-deficient-adeno-associated-virus-5-vector-bmn-307-human-liver-specific-transcription-regulatory-element-plus-human-phenylalanine-4-hydroxylase-codon-optimized-gene-hpah-plus-adeno-associated-virus-2-inverted-terminal-repeat-flanks-phenylketonuria
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