Chiesi Enters Licenses Key2Brain’s Blood-Brain Barrier-Crossing Therapies for LSDs

Rare Daily Staff

Chiesi Group and Key2Brain have entered into a worldwide license agreement to advance the development of two blood-brain barrier-crossing recombinant enzyme replacement therapies for alpha-mannosidosis and Krabbe disease, ultra-rare lysosomal storage disorders that affect the central nervous system.

The agreement is built on an existing research collaboration to develop the production of a blood-brain barrier-crossing recombinant alpha-mannosidase.

Key2Brain’s technology enables efficient brain uptake and broad distribution of therapeutics. The technology provides opportunities for the development of next-generation brain targeting across a diverse range of therapeutic areas. The technology can be efficiently integrated into or combined with therapeutic molecules without impacting the disease target specificity. The platform is being applied to peptides, proteins, enzymes, and oligonucleotides.

Under the new agreement, Key2Brain will receive an undisclosed upfront payment. It is eligible to receive development and sales-based milestone payments, and tiered royalties on potential sales. Chiesi Group will fund all research, development, and subsequent commercialization worldwide.

Upon mutual agreement, the two companies may expand the license to Key2Brain’s technology platform for the development of additional blood-brain barrier-crossing enzyme replacement therapies.

Chiesi Global Rare Diseases, the Chiesi Group’s dedicated business unit focused on research, development, and commercialization of therapies for rare and ultra-rare conditions, is spearheading the programs.

“We are deeply committed to building a sustainable pipeline in rare diseases by embracing emerging technologies that can enhance the treatment landscape,” said Giacomo Chiesi, executive vice president of Chiesi Global Rare Diseases. “This agreement exemplifies that vision—working to address areas of profound unmet medical need, including neurodegenerative manifestations of ultra-rare diseases like alpha-mannosidosis and Krabbe disease.”