Targeting Brain Delivery of Biotherapeutics for CNS Diseases

Rapid progress in identifying human genetic risk associated with neurodegenerative disease has revealed numerous genes involved in neurodegeneration. Degenogenes are genes that when mutated cause, or are major risk factors for, neurodegenerative disease. These degenogenes highlight important disease pathways for therapeutic discovery, including lysosomal function, glial biology and cellular homeostasis.

Advances in the genetics, pathology and cell biology underlying chronic neurodegenerative disease have identified pathways that trigger neurodegeneration and contribute to disease onset and progression.

Novel delivery approaches being developed at Denali are being used to deliver both small and large molecules across the blood brain barrier to address the high unmet need in Parkinson’s Disease, Frontotemporal Dementia-Granulin, ALS, Multiple Sclerosis, and other diseases.

Joe Lewcock will speak about developing therapeutics involved in lysosomal function, glial biology, cellular homeostatis for neurodegenerative diseases.