Phastar helped UK researchers complete SCID gene therapy study
Phastar says pro bono work on a severe combined immunodeficiency (SCID) gene therapy trial treatment is part of a wider effort to ensure researchers can access biometrics.
The trial – which was sponsored by Great Ormond Street Hospital - assessed the efficacy and safety of OTL-101 - cryopreserved stem cells transduced ex vivo with lentiviral vectors encoding for the human ADA gene - for the treatment of children suffering SCID and related conditions.
According to the sponsors “Gene therapy offers an alternative treatment for ADA-SCID whereby a correctly functioning copy of the ADA gene is introduced into hematopoietic stem cells (HSCs) that have been harvested from the patients themselves using a gene transfer vector.
“These transduced cells are then returned to the patient where they initiate immune reconstitution much like HSCs from a healthy donor.”
UK-based Phastar spoke about its participation in the study this month, explaining its specialist statisticians, data managers and programmers worked on the study for no charge.
And the firm played a critical role in the research according to CEO, Andrew MacGarvey.
“It was brought to our attention that Great Ormond Street Hospital needed additional support to ensure their project reached completion. Phastar therefore agreed to provide their services on a pro-bono basis through the Phastar Cares initiative.
He added, “The Phastar Cares pro-bono scheme enables charities to access Phastar’s expertise in biometrics and we are proud to provide support in the form of our industry-leading specialist statisticians, data managers and programmers to this research.”
The pro-bono scheme, which was launched in 2022, has also worked with UK charity Education for Health which provides training and resources healthcare professionals.
Study leader Prof Claire Booth underlined the role Phastar had played in the OTL-101 project, explaining “Great Ormond Street Hospital have been engaging with Phastar for a number of years to deliver this trial.
“They have performed numerous analyses on an ongoing basis and have been professional and knowledgeable throughout. We are grateful for their generous pro bono support that ensured this critical study could be completed.”
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