Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications. This agreement expands on an original non-exclusive license for AAV5 from the NIH. Financial details of the agreement have not been disclosed.
Concurrent with the signing of this exclusive license, AMT has received a notice of allowance from the United States Patent and Trademark Office (USPTO) for its patent application entitled “AAV vectors produced in insect cells”, which covers a core asset of the Company’s proprietary manufacturing technology. This patent comprises a novel and significantly improved method of delivery of therapeutic genes to targets using adeno-associated viral (AAV) derived vectors produced in insect cells. The technology has already been successfully applied to Glybera®, a gene therapy for lipoprotein lipase deficiency (LPLD). AMT believes this intellectual property together with the NIH exclusive license places the Company in a unique position for a number of AAV serotype 5-based programs, including hemophilia, acute intermittent prophyria, GDNF-related diseases and Sanfilippo B.
“This expansion of our existing AAV5 license with NIH is an important addition to our intellectual property position as it supports several of our pipeline products that follow Glybera. The granting of an exclusive license by the NIH is very unusual and we believe acknowledges AMT’s status in the gene therapy sector,” stated Jörn Aldag, CEO of AMT. “As well as continuing to work diligently towards the re-examination of Glybera registration dossier, we also appreciate the significant value and need to patients of the other gene therapy programs.”
About Amsterdam Molecular Therapeutics
AMT is a world leader in the development of human gene based therapies. The company’s lead product Glybera®, a gene therapy for lipoprotein lipase deficiency (LPLD), is currently under review by the European Medicines Agency (EMA). If approved, Glybera will be the first gene therapy product to be marketed in Europe. AMT also has a product pipeline of several gene therapy products in development for hemophilia B, Duchenne muscular dystrophy, acute intermittent porphyria, Parkinson’s disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world’s first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry. AMT was founded in 1998 and is based in Amsterdam. Further information can be found at www.amtbiopharma.com.
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