Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II), is being targeted for treatment with RGX-121, a product candidate utilizing the AAV9 vector to deliver the IDS gene encoding the I2S enzyme to the CNS. This innovative approach aims to establish a continuous source of I2S beyond the blood-brain barrier, facilitating long-term correction of […]
Category: Orphan Drug Status
Leronlimab: Promising Therapy for Metastatic Triple-Negative Breast Cancer
CytoDyn’s investigational therapy, leronlimab, has shown promising outcomes in treating metastatic triple-negative breast cancer (mTNBC), a disease known for its poor prognosis. Survival rates at 12, 24, and 36 months post-treatment with leronlimab compare favorably to current approved therapies, with some patients surviving over 36 months with no ongoing disease. After resolving a dispute with […]
