Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04480567 | AAV Gene Therapy Study for Subjects with PKU | PHASE1 | ACTIVE NOT_RECRUITING | 100 | — | — | Sep 24, 2020 | Dec 1, 2027 | Dec 12, 2024 | 3 | United States, United Kingdom |
| Arm | Type | Description |
|---|---|---|
| Dose 1 of BMN 307 | EXPERIMENTAL | - |
| Dose 2 of BMN 307 | EXPERIMENTAL | - |
| Dose 3 of BMN 307 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BMN 307 | DRUG | AAV Gene Therapy Infusion |
Inclusion Criteria: * Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency * Ability and willingness to maintain dietary protein intake consistent with baseline intake * Willingness to abstain from hepatotoxic substances post-BMN 307 administration * W...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase, BMN 307 |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923, Sepiapterin |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | — | HMI-102 |