Toronto, Canada, May 7, 2024 – Biopharma Services, a leading contract research organization specializing in early clinical trials, is pleased to announce that it has been awarded a groundbreaking First-in-Human (FIH) study. This milestone follows a significant partnership development where BioPharma Services executed an MSA with a top 10 pharmaceutical company specializing in rare diseases, underscoring our expanding influence in cutting-edge pharmaceutical research. 

The study aims to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of a new approach to improve gene therapies particularly focusing on patients who might not be eligible for traditional therapies due to pre-existing antibodies.  

The study, titled “A Randomized, First-In-Human, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity” will be conducted with healthy adult participants, under the guidance of BioPharma Services’ team of experts in scientific affairs and clinical operations. 

Dr. John Oldenhof, Executive VP of Scientific Affairs at BioPharma Services, expressed his excitement about the study: “This study represents a significant milestone in our commitment to advancing Gene Therapy treatments. By evaluating the new strategies to enhance gene therapies for safety and efficacy, we aim to contribute to the development of effective strategies for patients including those with pre-existing antibodies.” Dr. Oldenhof, who has a personal connection to the potential benefits of this research, shared, “As a parent of a child who may one day benefit from gene therapy, I am particularly excited by the prospect that this approach could increase his chances of being eligible for innovative therapies currently in development.” 

Renee John, Executive VP of Clinical Operations, added, “We are thrilled to lead this study, which has the potential to transform gene therapy approaches. Our experienced team is dedicated to ensuring the highest standards of safety and efficacy throughout the study.” 

Gene therapy has emerged as a promising approach for treating genetic disorders. However, pre-existing antibodies against viral vectors used in gene therapy can pose challenges. The study’s results will inform further clinical development and the design of Phase 2/3 studies for gene therapies in pre-existing antibody positive gene therapy patients. 

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