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Mar 10, 2014

ReGenX, Esteve to Develop Sanfilippo Syndrome Treatments

  • ReGenX Biosciences is teaming up with Laboratorios Dr. Esteve to develop and commercialize treatments for the rare lysosomal storage disease mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome Type A) using ReGenX' NAV rAAV9 vectors for treatment of MPS IIIA in humans.

    ReGenX has given Esteve a nonexclusive worldwide license to the vectors per the agreement, along with rights to sublicense. In return, ReGenX will receive an up-front payment, milestone fees, and royalties on net sales of any product incorporating NAV rAAV9.

    NAV is, according to ReGenX, a gene delivery technology that includes recombinant adeno-associated viral (rAAV) vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. NAV rAAVrh10 was the focus of a similar agreement back in December between ReGenX and gene therapy firm Lysogene, also to develop and commercialize treatments for MPS IIIA. NAV rAAV9, along with NAV rAAV8, was also the subject of a July 2013 deal with Audentes Therapeutics to develop X-linked myotubular myopathy and Pompe disease treatments.

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Scientifically Studying Ecstasy

MDMA (commonly known as the empathogen “ecstasy”) is classified as a Schedule 1 drug, which is reserved for compounds with no accepted medical use and a high abuse potential. Two researchers from Stanford, however, call for a rigorous scientific exploration of MDMA's effects to identify precisely how the drug works, the data from which could be used to develop therapeutic compounds.

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