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Also known as PBGENE-DMD (IV)
PBGENE-DMD · 1 trial · 1 indication
Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment
| Arm | Type | Description |
|---|---|---|
| Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort | EXPERIMENTAL | The trial is planned to enroll participants into 2 parts as follows: * Part 1 (Initial Safety) A total of up to 6 participants may be enrolled. * Part 2 (Expansion) Up to 12 participants |
| Name | Type | Description |
|---|---|---|
| PBGENE-DMD (IV) | BIOLOGICAL | Participants will receive a single dose of PBGENE-DMD |
Inclusion Criteria: 1. Males, 2 to 7 years of age, inclusive, at the time of informed consent/assent 2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \[inclusive\]) 3. Clinical phenotype consistent with DMD in the opinion of the Investigator 4. Ability to co...
PBGENE-DMD is an investigational therapy being developed for Duchenne Muscular Dystrophy in patients with mutations amenable to PBGENE-DMD. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied in a Phase 1/2a trial called FUNCTION-DMD.
PBGENE-DMD targets dystrophin, a protein that is deficient in Duchenne Muscular Dystrophy. By targeting dystrophin, the therapy aims to address the underlying genetic cause of the disease. This mechanism is being evaluated in early-stage clinical trials.
PBGENE-DMD is being developed by Precision BioSciences, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol DTIL. Precision BioSciences is conducting clinical trials to evaluate the safety and efficacy of PBGENE-DMD.
PBGENE-DMD is in Phase 1 clinical development. It is currently being studied in a Phase 1/2a trial, which is recruiting participants. The drug has received Fast Track designation from the FDA, but it is still investigational and not yet approved for commercial use.
PBGENE-DMD is being evaluated in a Phase 1/2a clinical trial with the identifier NCT07429240. This trial, named FUNCTION-DMD, is a safety and preliminary efficacy study in patients with Duchenne Muscular Dystrophy. The trial is recruiting male participants aged 2 years and older in the United States.
Yes, PBGENE-DMD is also known as PBGENE-DMD (IV). The alternative name refers to the intravenous formulation of the drug. Both names refer to the same investigational therapy being developed by Precision BioSciences for Duchenne Muscular Dystrophy.