Abeona Therapeutics Licenses RegenxBio’s AAV9 Vector to Develop Gene Therapies for Four Rare Lysosomal Storage Disorders

November 5, 2018

Rare Daily Staff

Abeona Therapeutics, a company focused on developing novel gene and cell therapies for life-threatening rare diseases, received an exclusive global license to RegenxBio’s proprietary adeno-associated virus  vector NAV AAV9 for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA), Sanfilippo syndrome type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 disease), and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 (CLN3 disease).

RegenxBio’s gene delivery platform consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.

“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 central nervous system manifestations of lysosomal storage diseases,” said Carsten Thiel, CEO of Abeona.

Under the terms of the agreement, Abeona will pay RegenxBio $20 million in an 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; potential commercial milestone payments of up to $60 million; and low double-digit royalties on net sales of products incorporating the licensed intellectual property.

“This license agreement further validates the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of lysosomal storage diseases, as well as the strength of our intellectual property portfolio,” said Kenneth T. Mills, president and CEO of RegenxBio.

Sanfilippo syndromes, also known as mucopolysaccharidosis or MPS type III, is a group of four inherited genetic diseases each caused by a single gene defect. The four forms of MPS III are identified as type A, B, C or D. Each causes enzyme deficiencies that result in the abnormal accumulation of metabolic waste in cells from improperly broken down carbohydrates. An estimated 70 percent of children with MPS III do not reach age 18.

Infantile and juvenile forms of Batten disease, known as CLN1 and CLN3, are rare autosomal recessive genetic disorders with no approved treatments. Batten disease is fatal, and most people with the disease do not live past their twenties or thirties. The underlying cause of the disorder is a deficiency in proteins critical to lysosomal function that lead to abnormal buildup of lipopigments, and result in neuroinflammation and neurodegeneration.

November 5, 2018

Photo: Carsten Thiel, CEO of Abeona Therapeutics


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