Busulfan – VJRegenMed

ARU-1801, a novel lentiviral gene therapy for SCD

Simon Ng — Thu, 03 Feb 2022 12:25:15 +0000

Current investigational therapies for sickle cell disease (SCD) require conditioning chemotherapy such as busulfan to administer the genetically-modified stem cells in the patient’s bone marrow. Will Chou, MD, Aruvant, New York, NY, provides an overview of ARU-1801, for patients with SCD. The lentiviral gene therapy uses fewer doses of an alternative chemotherapy, melphalan, which results in fewer side effects such as thrombocytopenia and veno-occlusive liver disease, as well as shorter hospital stays. This interview took place at Advanced Therapies Week 2022.

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.