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BMN 307 · 1 trial · 1 indication
| 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 |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923 |
| 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 |
BMN 307 is an investigational gene therapy being developed for the treatment of phenylketonuria (PKU), a metabolic disorder. It is designed to deliver a functional copy of the gene for phenylalanine hydroxylase, the enzyme that is deficient in PKU, to help restore normal phenylalanine metabolism.
BMN 307 targets the gene for phenylalanine hydroxylase, the enzyme responsible for breaking down the amino acid phenylalanine. In phenylketonuria (PKU), mutations in this gene lead to enzyme deficiency, and BMN 307 aims to provide a working copy of the gene to correct the underlying defect.
BMN 307 is being developed by BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a biopharmaceutical company focused on rare genetic diseases. The company is conducting clinical research to evaluate the safety and efficacy of this gene therapy for phenylketonuria (PKU).
BMN 307 is currently in Phase 1 clinical development. It is an investigational gene therapy for phenylketonuria (PKU) and has not been approved by regulatory authorities. The ongoing Phase 1 trial is active but not recruiting participants, and it is designed to assess safety and preliminary efficacy.
BMN 307 is being studied in a Phase 1 clinical trial with the identifier NCT04480567, titled 'AAV Gene Therapy Study for Subjects with PKU.' This trial is active but not recruiting, with an estimated enrollment of 100 participants. The study is being conducted in the United States and the United Kingdom, enrolling individuals aged 15 years and older.
BMN 307 is a distinct investigational gene therapy developed by BioMarin Pharmaceutical Inc. for phenylketonuria (PKU). It is not known to be marketed under any other name, and it is being evaluated as a unique treatment approach for this metabolic condition.