Phastar Supports Great Ormond Street Rare Disease Study

Phastar has supported a Great Ormond Street Hospital-sponsored study into new treatments for children with severe combined immunodeficiency (SCID). The company provided pro bono support from its specialist statisticians, data managers and programmers for the study, testing efficacy and safety of cryopreserved haematopoietic stem cells for transplant.

OTL-101 is a cell suspension comprising autologous CD34+ HSCs that are transduced ex vivo with Elongation Factor 1α Short form lentiviral vectors encoding for the human ADA gene. These transduced cells are then cryopreserved until such time of infusion back to the subjects.

The objective of this trial, supported by Phastar, was to determine the efficacy and safety of cryopreserved OTL-101 treatment.

Reference

Phastar Supports Great Ormond Street Rare Disease Study Through Pro Bono Scheme. (2023, April 13). Phastar.