Case Study

Flexibility Drives Progress During A Rare Disease Gene Therapy Clinical Trial


A biotechnology firm specializing in gene therapies for metabolic disorders enlisted the services of Veristat to assist in conducting a clinical study aimed at treating a rare genetic lysosomal storage disorder in humans.

Initially, the sponsor furnished Veristat with a list of potential sites for evaluating the safety, tolerability, and efficacy of the gene therapy via various clinical, molecular, and biochemical assessments. However, regrettably, none of the sites recommended by the sponsor proved to be feasible, necessitating a comprehensive search for alternative locations. This unexpected setback prompted a diligent quest for more suitable venues to conduct the study.

Uncover deployed methods and actions that were used to help the biotech address clinical operations challenges and meet changing requirements.

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