Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel gene and cell therapies for life-threatening rare diseases, has been granted new exclusive licenses to develop gene therapies for treatment of four rare lysosomal storage disorders using Regenxbio’s NAV AAV9 vector platform technology. 

The global licenses will help support development of gene therapies for treatment of Sanfilippo syndrome types A and B (MPS IIIA and MPSIIIB), infantile Batten disease (CLN1), and juvenile Batten disease (CLN3).

Under the terms of the agreement, Regenxbio has granted Abeona an exclusive worldwide license (subject to certain non-exclusive rights previously granted for MPS IIIA), with rights to sublicense, to Regenxbio’s NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of MPS IIIA, MPS IIIB, CLN1 Disease and CLN3 Disease.

In return for the rights, Regenxbio will receive a guaranteed $20 million upfront payment, $10 million of which will be paid upon signing and $10 million of which will be paid within 12 months of the effective date. In addition, Regenxbio will receive a total of $100 million in annual fees, payable upon the second through sixth anniversaries of the agreement, $20 million of which is guaranteed. Regenxbio is also eligible to receive potential commercial milestone payments of up to $60 million. Regenxbio will receive low double-digit royalties on net sales of products incorporating the licensed intellectual property.

Sanfilippo syndrome is a group of rare genetic lysosomal storage diseases with no approved treatments. Infantile and juvenile forms of Batten disease are rare autosomal recessive genetic disorders with no approved treatments.

(Source: Abeona Therapeutics Inc.)