Shaking-up gene delivery in the neurodegenerative disease world

San Diego, USA-based Ceregene, which is developing a novel neurotrophic factor gene therapy delivery via adeno-associated virus vectors (AAV), was recently listed as among the top 10 next-generation biotech platform by news service FierceBiotech, which noted that Ceregene’s second-generation product will shake up the neurodegenerative disease research field and make Big-Pharma quake in their boots.

Taking up the story, pharmaceutical intelligence firm GlobalData notes that Ceregene’s technology is aimed at reducing symptoms and reversing or slowing progression of degenerating neurons by using nerve growth factors (NGFs). These factors, such as neurturin, play a crucial role in brain and eye development to increase vitality and function. Conventional treatment for diseases such as Huntington’s disease and Parkinson’s disease include oral therapy and deep brain stimulation.

However, says Global Data, since NGFs are large proteins, delivery to the brain is extremely difficult due to low level of absorption and passage through the blood brain barrier. Ceregene novel adeno-associated virus vector aims to address this unmet need that currently exists. The vector uses a strain from the parvovirus family (a non-pathogenic strain to humans) which carries neurturin into the appropriate brain tissue through brain surgery. The desired therapy outcome is that the NGF will remain indefinitely within the targeted brain tissue and emit its therapeutic properties.