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ABO-102 (formerly ABX-101, ABX-A)

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Company Abeona Therapeutics Inc.
DescriptionAdeno-associated viral serotype 9 (AAV9) vector that delivers the N-sulfoglucosamine sulfohydrolase (SGSH; HNS) gene
Molecular Target N-sulfoglucosamine sulfohydrolase (SGSH) (HNS)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Adeno-associated virus (AAV)
Latest Stage of DevelopmentPhase I/II
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome)
Regulatory Designation U.S. - Fast Track (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome));
U.S. - Orphan Drug (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome));
EU - Orphan Drug (Treat mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome))
Partner

 Product Details
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Total Number of DealsTotal Deal ValueTotal Upfront CashTotal Milestone Payments

1

$40.9M

0

$9.0M


 Deals Details
DateCompanies InvolvedDeal HeadlineTotal Deal ValueTotal Upfront CashTotal Milestone

05/18/2015

$40.9M

0

$9.0M

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