Date: 2018-11-26

Type of information: Licensing agreement

Compound: NAV AAV9 vector and two additional NAV AAV vectors

Company: RegenXBio (USA - MD) Rocket Pharmaceuticals (USA - NY)

Therapeutic area: Rare diseases - Genetic diseases - Cardiovascular diseases - Neuromuscular diseases

Type agreement: licensing

Action mechanism:

  • gene therapy. 

Disease: Danon disease


  • • On November 26, 2018, Regenxbio and Rocket Pharmaceuticals announced an exclusive, worldwide license agreement for Rocket to develop and commercialize gene therapy treatments for Danon disease using Regenxbio's NAV AAV9 vector, as well as exclusive options for two additional NAV AAV vectors for the treatment of Danon disease.
  • Under the terms of the agreement, Regenxbio has granted Rocket an exclusive, worldwide license, with rights to sublicense, to Regenxbio's NAV AAV9 vector for the development and commercialization of gene therapy treatments for Danon disease. Regenxbio has also granted Rocket exclusive options to two additional undisclosed NAV AAV vectors for the treatment of Danon disease for up to four years.
  • Danon disease is a rare neuromuscular and cardiovascular disease characterized by profound cardiomyopathy, skeletal myopathies, and mild cognitive impairment. The disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. The LAMP-2 protein has three distinct variants generated by alternative splicing: LAMP-2A, LAMP 2B and LAMP-2C. Mutations resulting in LAMP-2B dysfunction are associated with severe cardiac disease features. The disease is often fatal in patients in the second or third decade of life due to progressive heart failure unless treated with a cardiac transplantation, which is nonetheless not considered curative. There are no specific therapies available for the treatment of Danon disease.

Financial terms:

  • In return for these rights, Regenxbio will receive a $7 million upfront payment, ongoing fees, milestone payments and high-single to low-double digit royalties on net sales of products incorporating the licensed intellectual property.

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Is general: Yes