Date: 2010-01-06
Type of information: Development agreement
Compound: adeno-associated viral 5 (AAV5) gene therapy
Company: Amsterdam Molecular Therapeutics (The Netherlands) - consortium led by Institut Pasteur (France)
Therapeutic area: Rare diseases
Type agreement: Development
Manufacturing
Production
Action mechanism:
Disease: Sanfilippo B (rare autosomal recessive lysosomal storage disease, which manifests in young children)
Details: Amsterdam Molecular Therapeutics and Institut Pasteur have entered into an agreement support clinical development of a novel gene therapy to treat Sanfilippo B. Institut Pasteur will lead the development program and will also sponsor the initial Phase I/II clinical study of a gene therapy to replace an enzyme (alpha-N-acetylglucosaminidase) that is missing in brain cells of SanfilippoB patients. AMT will manufacture and supply the adeno-associated viral 5 (AAV5) gene therapy product to the Consortium. If the Consortium successfully demonstrates proof of concept in the Phase I/II study, AMT will have an option to acquire full commercial rights for the program. The Phase I/II clinical study is scheduled to begin before 2013.
Financial terms: Donations collected during the French Telethon, the French Muscular Dystrophy Association (AFM), a Consortium member, will fully fund the development program through to completion of the Phase I/II clinical study, including all AMT manufacturing costs. The overall manufacturing contract entails payments to AMT of €1.8 million.
Latest news: In December 2010, Consortium researchers published preclinical data on the Sanfilippo B gene therapy in Molecular Therapy, the official publication of the American Society of Gene and Cell Therapy (Mol Ther. 2011 February; 19(2): 251–259. Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes.N Matthew Ellingwood et al http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3034858/?tool=pubmed).
This data demonstrated safety and efficient spreading of the AAV5 gene vector particles throughout the brain in models of the disease. The gene vectors also exhibited long-term viability within the cells and an improvement of histological and biochemical markers.
