Partnership to advance novel platform tech for lysosomal storage disorders

An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to advance a novel blood-brain barrier-crossing platform technology for those with lysosomal storage disorders.

According to a release from Chiesi Global Rare Diseases, the company will team up with Aliada Therapeutics on research and development of multiple enzyme cargoes modified with Aliada's Modular Delivery (MODEL) platform, which harnesses endogenous brain endothelial cell transport mechanisms to move large molecule therapeutics across the blood brain barrier (BBB) more quickly and effectively.

"Our commitment to the development of new treatment options for people living with [lysosomal storage disorders (LSDs)] is global, as evidenced by recent regulatory approvals," Giacomo Chiesi, head of Chiesi Global Rare Diseases, stated in the release. "With this collaboration, we are expanding our strategy and presence in BBB-crossing technologies and hope to leverage our know-how in LSDs to support the development of an effective and differentiated drug delivery platform.”

Aliada uses its MODEL platform toward differentiated large molecule therapeutics for individuals with neurological disorders, as well as systemic disorders with central nervous system involvement, per the release. These include but are not limited to lysosomal storage disease, which adversely affects enzymes within cells that may lead to disorders such as leukodystrophies.

"We are excited to partner with Chiesi to develop improved therapeutics for patients living with LSDs, who currently lack treatments that can readily access the brain," Adam Rosenberg, CEO of Aliada Therapeutics, said in the release. " This collaboration will allow Aliada to demonstrate the diverse capabilities of our MODEL platform."