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 (PKU)
BioMarin Pharmaceuticalgene_therapyPhase 1/2Active
CC
47/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 33/ 100
- Competitive Position
- 89/ 100
Competitive context
18 Active assets in the Phenylketonuria (PKU) cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Sapropterin Dihydrochloride Sohag University | Phase 4 | A |
PHENYLASE BioMarin Pharmaceutical | Phase 4 | A |
Ravpal-Peg BioMarin Pharmaceutical | Phase 2 | BB |
Repinatrabit Otsuka Pharmaceutical Development & Commercialization, Inc. | Phase 3 | BB |
ESCHERICHIA COLI STRAIN SYNB1618 Synlogic | Phase 2 | BB |
View all 29 →
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