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Gene Therapy Clinical Trial Update 2022

Researchers give an update on the FANCA Gene Therapy Clinical Trial in the US and Spain


Rocket Pharma has sponsored gene therapy clinical trials to study the use of autologous (from the patient) stem cells transduced with a lentiviral vector carrying the wild type (normal) FANCA gene. The goal of the study is to determine safety and preliminary efficacy of autologous gene correction for the FANCA gene in hematopoietic stem cells without the use of harmful conditioning agents. This Q&A session was aired in February 2022 as a part of the Fanconi Anemia Research Fund FA Connect Series.

ES: Rocket Pharma ha patrocinado ensayos clínicos de terapia génica para estudiar el uso de células madre autólogas (del paciente) transducidas con un vector

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Rocket Pharma has sponsored gene therapy clinical trials to study the use of autologous (from the patient) stem cells transduced with a lentiviral vector carrying the wild type (normal) FANCA gene. The goal of the study is to determine safety and preliminary efficacy of autologous gene correction for the FANCA gene in hematopoietic stem cells without the use of harmful conditioning agents. This Q&A session was aired in February 2022 as a part of the Fanconi Anemia Research Fund FA Connect Series.

ES: Rocket Pharma ha patrocinado ensayos clínicos de terapia génica para estudiar el uso de células madre autólogas (del paciente) transducidas con un vector lentiviral que lleva el gen FANCA de tipo salvaje (normal). El objetivo del estudio es determinar la seguridad y la eficacia preliminar de la corrección genética autóloga del gen FANCA en células madre hematopoyéticas sin el uso de agentes acondicionadores nocivos. Esta sesión de preguntas y respuestas se emitió en febrero de 2022 como parte de la serie FA Connect del Fanconi Anemia Research Fund.

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