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First gene therapy trial to treat severe pyruvate kinase deficiency (PKD) is now open for enrollment!

First gene therapy trial to treat severe pyruvate kinase deficiency (PKD) is now open for enrollment!

2019-11-25
First gene therapy trial to treat severe pyruvate kinase deficiency (PKD) is now open for enrollment!

An open-label, phase 1 gene therapy study consisting of autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding for the PK enzyme is now enrolling patients

An open-label, phase 1 gene therapy study consisting of autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding for the PK enzyme is now enrolling patients. 

PKD patients with a confirmed PKLR mutation and severe, transfusion dependent anemia despite splenectomy may be eligible to enroll. The trial will initially enroll adult patients (≥18 and <45 years old) at Hospital Universitario Fundación Jiménez Díaz in Madrid, Spain. Once safety has been established in the adult population, the trial will next enroll older pediatric patients (≥12-17 years), followed by younger pediatric patients (≥8-11 years) at Hospital Infantil Universitario Niño Jesús.

Financial support, including travel and housing accommodations for patients and a family member, both for the treatment and follow-up visits, will be provided. 

If you are interested to learn more about the trial please email Dr. Gayatri Rao at PKDclinicaltrial(ELIMINAR)@rocketpharma.com, visit www.clinicaltrial.gov (NCT04105166), or click on https://bit.ly/2Qi6I6d for direct link.


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