Abeona bags rights to Regenxbio AAV9 vector in rare lysosomal storage disorders

Abeona is making the outlay to secure the rights to an AAV9 vector in indications that are important to its plans. (LionFive/Pixabay)

Regenxbio has granted Abeona Therapeutics a global license to use its AAV9 vector in the treatment of four rare lysosomal storage disorders. Abeona will pay at least $40 million for the right to use its vector in gene therapies against forms of Sanfilippo syndrome and Batten disease. 

Maryland-based Regenxbio has built up a portfolio of adeno-associated virus (AAV) vectors that it thinks can unlock the potential of gene therapies by improving gene expression and cutting the risk of immune attacks. These vectors, notably AAV8 and brain-penetrating AAV9, underpin Regenxbio’s internal pipeline and have proven attractive to companies including Novartis-owned AveXis and Ultragenyx.

Abeona has become the latest company to secure the right to use Regenxbio’s AAV9 vector in certain therapeutic areas. The deal gives Abeona near-exclusive rights to use the vector in treatments of Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB), and infantile (CLN1) and juvenile (CLN3) forms of Batten disease. The exception to the exclusivity is MPS IIIA. Regenxbio granted Laboratorios Dr. Esteve the nonexclusive right to use the vector in the indication in 2014.


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In return for its rights to the vector, Abeona has agreed to a financial package of payments that could ultimately be worth $180 million. Regenxbio will receive a guaranteed $40 million, starting with $10 million upon signing and continuing with subsequent annual fees. And it could receive up to $80 million more in contingent annual fees. The deal also features $60 million in potential commercial milestones.

Abeona is making the outlay to secure the rights to an AAV9 vector in indications that are important to its plans. The New York-based biotech is running early-phase trials of AAV9 gene therapies in MPS IIIA and MPS IIIB, and has another pair of AAV9 gene therapies against CLN1 and CLN3 in preclinical development. 

Regenxbio is active in areas just adjacent to the indications covered by the agreement. The biotech has AAV9 gene therapies against MPS I and MPS II in early-phase development, and a third candidate targeting CLN2 disease that should be ready for IND submission next year. 

Analysts at Jefferies said in a note to clients: "Deal today unlocks potential for four programs that use AAV9 including MPS IIIA, MPS IIIB, CLN1, and CLN3. Recall, AAV9 is administered IV and has tropism to CNS, enabling delivery of functioning gene (i.e., SGSH for IIIA); the co's use of IV administration has the potential to leverage blood supplies that reach deep areas of the brain that may be more critical and we believe could increase chances of efficacy.

"Currently, MPS IIIA is their most advanced program, followed by IIIB, but ABEO is making progress w/CLN1 and CLN3 too; CLN1 and CLN3 could move quickly for ABEO given their experience w/IIIA and IIIB. CLN3 could be a large mkt oppty (est 5,000 pts in the U.S.) w/competitor interest (recall, FOLD acquired several programs including CLN3 from Celenex 9/20/18 for $100M upfront and ~$350M in milestones)."

Editor's note: Abeona is based in New York. A previous version of this story identified its base as Texas.

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