biOasis releases quantitative results: siRNA-Transcendpep reduces target gene expression in the brain

The company announces that it has achieved effective knock-down of the expression of a target gene in the brain in a sequence-specific manner. Independent analysis of brain tissue revealed that, not only did Transcendpep deliver the siRNA to the brain tissue, but it also reduced the expression of the target gene by 40% to 50% after a single dose.

"Injection of a small bioconjugate of siRNA coupled to Transcendpep achieved a significant reduction in target gene expression in the brain," says Dr. Wilfred Jefferies, Founding Scientist of biOasis. "This represents a potential advancement for reducing disease-associated gene expression that often leads to the pathology present in a variety of brain diseases."

"These findings expand the preeminent standing of biOasis in the area of BBB carriers and provides vast opportunities for Transcendpep to act as a platform to significantly advance the treatment of brain diseases," says Rob Hutchison, CEO.

"Due to the vast market potential of siRNA delivery to the brain, we are focused on advancing this program to the IND stage and commencing Phase I Clinical Trials as soon as possible. This adds to biOasis’ development programs in the delivery of therapeutics across the BBB, which includes our metabolic disease programs for lysosomal storage diseases such as Hunter Syndrome and Sandhoff Disease and our oncology program (Transcendpep-Herceptin) for the treatment of brain metastases of HER2+ breast cancer. In addition to the aforementioned programs, we continue to further our partnership with MedImmune and develop relationships with other pharmaceutical companies."