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Amsterdam Molecular Therapeutics Reports Half-Year Results 2011
Date:8/24/2011

y has also won support from the Duchenne Parents Association and continues to work with them to progress this program through pre-clinical evaluation, in addition to the ongoing support from Agentschap NL (formerly SenterNovem).

GDNF

AMT is conducting pre-clinical research and has successfully completed a proof of concept study in a disease model of Parkinson's disease in collaboration with the University of Lund, Sweden. Data generated for AMT by the University of Wisconsin (USA) in a further pilot study using large animals also showed effective delivery, distribution and expression at levels that are expected to correlate with clinical efficacy; overcoming these challenges is one of the major challenges to clinical development. Taken together, these positive data encourage us to continue with the development of GDNF gene therapy and to extend it to other neurodegenerative indications such as multiple system atrophy (MSA) and Huntington's disease.

Sanfilippo B

Under an agreement signed at the beginning of this year, AMT is collaborating with a consortium led by Institut Pasteur in the clinical development of a novel gene therapy to treat Sanfilippo B. This rare genetic disease affecting new-born children leads to progressive neuronal degeneration and death. There is no approved therapy currently available.

On behalf of the Consortium, Institut Pasteur will lead the development program and will also sponsor the initial Phase I/II clinical study. AMT will manufacture and supply the adeno-associated virus, serotype 5 (AAV5) gene therapy product to the Consortium. The overall manufacturing contract entails payments to AMT of € 1.8 million. 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 in 2012.

Other Research and Development

AMT has demonstrated the ad
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