Chiesi partners Aliada on brain-penetrating CNS therapies

Rare disease specialist Chiesi Pharma has forged an alliance with US biotech Aliada Therapeutics that will apply a technology designed to get drug molecules across the blood-brain barrier (BBB).

The BBB is a microenvironment in the brain, designed to protect it from noxious substances and pathogens in the blood, and is a major obstacle to getting large-molecule therapeutics into the central nervous system.

Chiesi is interested in Aliada's modular delivery (MODEL) platform, which harnesses endogenous brain endothelial cell transport mechanisms – including transferrin and CD98 receptors – to usher therapeutics across the BBB and into the CNS.

It is the second partnering deal focused on technologies that allow drugs to pass the blood-brain barrier in the last few months, coming after Biogen teamed up with Denali on its ATV technology, in the hope of boosting the activity of amyloid-targeting drugs for Alzheimer's disease.

Chiesi said in a statement that it hopes to apply the MODEL approach to "multiple enzyme cargoes" in the hope of not only elevating levels of the therapies within the brain, but also optimising "CNS delivery and downstream functionality".

The aim will be to develop improved therapies for lysosomal storage diseases (LSDs), a group of disorders characterised by inborn errors of metabolism that are often treated with enzyme replacement therapies.

Chiesi's portfolio of medicines for LSDs includes Elfabrio (pegunigalsidase alfa) for Fabry disease - which has just been approved in the UK - and Lamzede (velmanase alfa) for alpha-mannosidosis, with the rare disease portfolio as a whole contributing €227 million out of total turnover of €1.5 billion in the first half of this year.

"Many LSDs have CNS involvement," said Giacomo Chiesi, who is head of Chiesi's rare diseases unit. "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."

This is the first partnership announced for Boston-based Aliada, which was formed in 2021 and raised $32 million in a financing round last December. The biotech counts Johnson & Johnson Innovation Capital, Sanofi Ventures, OrbiMed, and RA Capital among its backers.

It says its BBB technology can be applied to monoclonal antibodies, enzymes, oligonucleotides, peptides, and other proteins.