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Preliminary clinical trial results of innovative gene therapy to treat fatal childhood immunodeficiency are positive

 Last Update: 2021-11-01
 Source: Internet
 Author: User

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A few days ago, Rocket Pharmaceuticals announced that its research gene therapy RP-L201 has obtained positive interim data in a phase 1/2 clinical trial for the treatment of leukocyte adhesion deficiency type I (LAD-I)

The clinical manifestations of type I leukocyte adhesion deficiency are delayed umbilical cord shedding, repeated soft tissue infections and chronic periodontitis

RP-L201 is an in vitro autologous hematopoietic stem cell gene therapy based on lentiviral vector

This interim data comes from 7 patients with severe LAD-I, whose CD18 expression is less than 2%

Reference materials:

[1] Rocket Pharmaceuticals Announces Positive Clinical Data from RP-L201 Trial for the Treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT).

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