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Restoring lysosomal function to treat Parkinson's disease

Denali Therapeutics is developing a very wide range of small molecules and other compounds to assist in combating several diseases. It has two principal candidates, DTL201 and DTL151, that are in Phase 1 human clinical trials as a therapy against Parkinson’s. The goal of these drugs is to restore lysosomal function, allowing them to better remove Lewy bodies, which kill vital motor neurons and lead to the symptoms of Parkinson’s. As the lysosomes are responsible for destroying degraded proteins., this therapy directly combats the aging hallmark of proteostasis.

Some of Denali’s other work includes a Phase 1 trial of DNL747, which reduces brain inflammation through inhibiting RIPK1, and another Phase 1 trial of DNL343, which is designed to reduce the effects of cellular stress in mutated cells by restoring RNA and protein function. Denali is also in early-stage development of a proprietary technology for getting drugs through the blood-brain barrier, and the company is developing further therapies based on this technology.