Date: 2015-10-13
Type of information: Licensing agreement
Compound: NAV vector technology
Company: RegenXBio (USA - MD) Dimension Therapeutics (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases - Metabolic diseases
Type agreement: licensing collaboration
Action mechanism: gene therapy
Disease: hemophilia, ornithine transcarbamylase deficiency (OTC), glycogen storage disease type 1a (GSD1a)
Details: * On October 31, 2013, Fidelity Biosciences and RegenXBio announced the formation of Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Dimension will focus on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for AAV therapeutics. Dimension has completed an undisclosed Series A financing led by Fidelity Biosciences. In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with RegenXBio. RegenXBio holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV® vector technology that includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Through its license and collaboration with RegenXBio, Dimension has acquired preferred access to NAV vector technology and rights in RegenXBio product programs in multiple rare disease indications. Dimension has assembled a team of leaders in the areas of rare disease and gene therapy as well as industry veterans and experienced entrepreneurs. The company has appointed Dr. Beck as interim chief executive officer and Sam Wadsworth, Ph.D. as chief scientific officer. Dr. Wadsworth was previously head of gene therapy research and development at Genzyme, where he led preclinical development for multiple rare disease and gene therapy programs. The company’s scientific advisors are leading experts in the field of gene therapy and rare disease. Dr. James Wilson, director of the gene therapy program at the University of Pennsylvania and the scientific founder of RegenXBio, will chair the company’s Scientific and Technical Advisory Board. NAV vector technology was discovered in the laboratory of Dr. Wilson at the University of Pennsylvania. Other advisors to Dimension include Emil D. Kakkis, M.D., Ph.D., president and chief executive officer of Ultragenyx, a leading rare disease company, and former chief medical officer of Biomarin. Ben Auspitz, partner at Fidelity Biosciences, has been appointed chairman of the Board of Dimension, and will be joined by directors Allan M. Fox, founding and managing partner of FOXKISER, the entrepreneurial force behind RegenXBio; Donald J. Hayden, an experienced pharmaceutical executive and the chairman of RegenXBio; and Dr. Beck.
Financial terms: * On April 23, 2015, RegenXBio, a leading biotechnology company in gene therapy, announced that it has added the indications of ornithine transcarbamylase deficiency (OTC) and Glycogen Storage Disease Type 1a (GSD1a) to its existing license agreement with Dimension Therapeutics. This will enable Dimension to develop and commercialize products to treat OTC and GSD1a – as well as hemophilia A and B, which Dimension licensed from RegenXBio in 2013 – using RegenXBio’s proprietary NAV® Technology. “The addition of OTC and GSD1a to our license agreement with Dimension is a natural extension of the founding intent of the company, as it enables the development of innovative liver-directed treatments using NAV Technology for patients suffering from these important, rare metabolic diseases,” said Ken Mills, President and CEO of RegenXBio. “In addition to our own programs, we have now granted 16 commercial licenses and remain committed to the development and commercialization of our NAV Technology in disease areas where patients need better treatment options.”
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