Abeona licensing Regenxbio’s NAV tech platform to treat rare diseases

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Texas biopharma Abeona Therapeutics has signed a licensing agreement with Regenxbio to treat four rare diseases using the latter’s proprietary NAV Technology Platform.

The diseases are: Sanfilippo syndrome type A (MPS IIIA), Sanfilippo syndrome type B (MPS IIIB), Infantile Batten Disease, and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 (CLN3 Disease).

As per the agreement, Abeona will pay $20 million up front, $10 million upon signing and a further $10 million by the agreement’s first anniversary. Further, annual payments of $20 million are guaranteed payable on the second anniversary.

“This agreement is an important milestone that underpins the therapeutic potential we see in our Sanfilippo syndrome and Batten disease programs featuring the NAV AAV9 vector, which have the potential to transform the lives of patients,” said Carsten Thiel, Abeona CEO.

“Data from our clinical and preclinical programs and the success of the NAV AAV9 vector observed in other indications strongly positions the platform as a leading technology for investigational gene therapies for the systemic and CNS manifestations of lysosomal storage diseases.”