Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond
Highlights From the ESGCT 28th Annual Congress

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Alessio Cantore, PhD
San Raffaele Telethon Institute for Gene Therapy
University Vita-Salute San Raffaele
Milan, Italy

Key Data Points

Lentiviral Vector (LV) for Liver Gene Therapy

The construct of the lentiviral vectors designed for liver-directed gene therapy and used to package the human FIX or FVIII transgenes that were infused into non-human primates in order to transduce hepatocytes with the vector-transgene construct.

Normal to Supranormal Human Coagulation Factor Activity in NHP

The expression of hFIX and hFVIII in non-human primates infused with the lentiviral vector containing the corresponding transgene. The LV-FIX vector-transgene was infused at doses of 2.5 x 10-9 TU/kg (n = 1) and 7.5 x 10-9 TU/kg (n = 3). The LV-FVIII vector-transgene construct was infused at doses of 1 x 10-9 TU/kg (n = 2) and 3 x 10-9 TU/kg (n = 3).

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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