RP-L301 – VJRegenMed

RP-L301 lentiviral gene therapy for PKD

admin_vjr_user — Mon, 27 Dec 2021 12:22:59 +0000

Jose-Luis López-Lorenzo, MD, University Hospital Jimenez Diaz, Madrid, Spain, describes ongoing research into RP-L301, an investigational lentiviral gene therapy for pyruvate kinase deficiency (PKD). This approach involves autologous hematopoietic stem cells being transduced ex vivo with a lentiviral vector carrying a functional copy of the deficient PKLR gene. Based on preclinical studies in a murine model of PKD, a Phase I clinical trial (NCT04105166) evaluating RP-L301 is currently underway. While results obtained from two patients treated so far are promising, Dr López-Lorenzo notes that data from the treatment of more patients with a longer follow-up time is needed and highlights the need for a less complex and safer treatment protocol in the future. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

An update on the Phase I trial evaluating RP-L301 in PKD

admin_vjr_user — Mon, 27 Dec 2021 12:22:57 +0000

Jose-Luis López-Lorenzo, MD, University Hospital Jimenez Diaz, Madrid, Spain, provides an update on a Phase I clinical trial (NCT04105166) evaluating the safety of RP-L301, an investigational lentiviral gene therapy, in patients with pyruvate kinase deficiency (PKD). Dr López-Lorenzo outlines the study design, which involves the collection of peripheral blood hematopoietic stem cells (HSCs) which are transduced with a lentiviral vector carrying a functional copy of the deficient PKLR gene. Patients then receive a busulfan-containing conditioning regimen and then intravenous infusion of the RP-L301 HSC product. In the first two patients treated, hemoglobin levels were restored to normal levels, both report improved quality of life and no serious adverse events have been attributed to RP-L301. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Lentiviral gene therapy for pyruvate kinase deficiency

admin_vjr_user — Fri, 14 May 2021 15:12:26 +0000

Jose-Carlos Segovia, PhD, CIEMAT, CIBERER & IIS- FJD, Madrid, Spain, describes ongoing research into the use of gene therapy technologies for pyruvate kinase deficiency (PKD), a rare inherited hemolytic anemia. He describes an ongoing global Phase I clinical trial (NCT04105166) of RP-L301, an investigational ex vivo lentiviral gene therapy, in which two patients with PKD have been treated thus far. Preliminary data obtained from Patient 1 and 2 at 6- and 3-months follow up, respectively, demonstrated the normalization of erythrocyte counts and hemoglobin levels, and that the treatment was well tolerated. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.