RE:News Alert This knockout mouse model of Hunter Syndrome (MPS II) showed that the Transcend fusion protein, MTfp-I2S (MTfp-Iduronate-2-sulfatase), was able to:
1) cross the blood-brain barrier,
2) reached therapeutic levels in the CNS,
3) and significantly decreased the number of lysosomal storage vacuoles in the cytoplasm of brain cells.
The study also showed that MTfp-I2S:
1) normalizes the GAG level in the periphery (liver & urine) showing that the recombinant enzyme is active and distributed throughout the body.
2) The MTfp-I2S treatments were safe, showing no toxicity,
3) and the activity of the enzyme was not affected by the MTfp peptide carrier.
Dr. Maurizio Scarpa, President of the Brains for Brain Foundation stated, "The study that we have recently performed in collaboration with biOasis produced some exciting data. I am very encouraged that after some adjustments regarding dosing, we might be able to move this program into the clinic. My goal and I am sure the goal of biOasis as well, is to help children with this devastating disease. I am very encouraged that based on these results, Transcend may play an important role in helping us achieve our goals."