Chiesi partners with Key2Brain to develop therapies for rare neurodegenerative diseases
Ella Day | June 12, 2025 | News story | Manufacturing and Production, Mergers and Acquisitions, Research and Development | Chiesi Group, Key2Brain, Neurology, Rare Diseases, lysosomal storage disorders, neurodegenerative disease, partnership, ultra-rare disease
Italian biopharma, Chiesi Group, and Key2Brain, a Swedish biotech, have agreed to advance the development of two blood-brain barrier (BBB)-crossing recombinant enzyme replacement therapies (ERT) for lysosomal storage disorders. These are ultra-rare diseases, including alpha-mannosidosis and Krabbe disease, that affect the central nervous system and have limited treatment options.
Key2Brain will provide Chiesi with a licence to develop and commercialise two BBB-crossing ERTs. This is built on a partnership initiated in 2022 that aimed to develop the production of alpha-mannosidase, a BBB-crossing recombinant. Key2Brain will receive an upfront payment and is eligible to receive milestone payments upon development and sales, while Chiesi will fund all research, development and commercialisation.
The companies intend to expand the licence for the development of additional BBB-crossing ERTs. Such advancement will both enhance Chiesi’s BBB-crossing ERT portfolio and support Key2Brain’s ambition to become a leading platform provider of the therapy.
The development will be led by Chiesi global rare diseases, the group’s dedicated business unit focused on the development of therapies for rare and ultra-rare conditions.
“Expanding our relationship with Chiesi allows us to build on a clinically validated foundation to address the neurological complications of lysosomal storage disorders,” commented Elisabet Sjöström, founder and CEO of Key2Brain.
“We aim to enhance the biodistribution, efficacy and tolerability of promising therapies that have historically faced challenges,” said Mitch Goldman, senior vice president R&D, Chiesi global rare diseases.
Ella Day
12/6/25
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