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PM359 · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| PM359 | EXPERIMENTAL | PM359 is an autologous CD34+ hematopoietic stem cell (HSC) suspension that is Prime Edited at the NCF1 locus resulting in expression of the p47phox protein. |
| Name | Type | Description |
|---|---|---|
| PM359 | BIOLOGICAL | Single dose of PM359 administered autologously by intravenous (I.V.) infusion following myeloablative conditioning with busulfan |
Inclusion Criteria: * Autosomal recessive Chronic Granulomatous Disease due to the delGT mutation in NCF1 causing dysfunction of p47phox * Treated and followed for at least the past 2 years in a specialized center * Willingness to participate in this study as well as a long-term follow-up study wit...
PM359 is an investigational gene therapy being developed for Chronic Granulomatous Disease (CGD), specifically p47phox autosomal recessive CGD. It is currently in Phase 1 clinical development and is not yet approved by the FDA.
PM359 targets the NCF1 gene, which encodes the p47phox protein. It uses prime editing technology to correct mutations in NCF1, aiming to restore function in patients with p47phox autosomal recessive Chronic Granulomatous Disease.
PM359 is being developed by Prime Medicine, Inc., a biopharmaceutical company. The company's stock trades under the ticker symbol PRME on the NASDAQ.
PM359 is in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The drug has been granted RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations by the FDA.
PM359 is being studied in a Phase 1 clinical trial with the identifier NCT06559176. This trial is enrolling by invitation and aims to evaluate the safety and efficacy of PM359 in participants with p47phox autosomal recessive Chronic Granulomatous Disease.
PM359 is a prime editing gene therapy specifically designed for p47phox autosomal recessive Chronic Granulomatous Disease. It is being studied in a clinical trial enrolling patients aged 6 years and older in the United States, Canada, and the United Kingdom.