ex-vivo cell transfer strategy

After apheresis collection, a functional copy of the therapeutic gene is inserted into the patient’s own HSPCs using a non-replicating lentiviral vector. This is an ex-vivo process called transduction. We believe that LVVs are the first choice for ex-vivo cell therapy in humans because they can efficiently transduce HSPCs with potentially large transgenes that will allow us to expand therapeutic options to a multitude of payloads. Most importantly, there is an abundance of safety data generated using these vectors to develop novel medicines currently in clinical development.


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