AMT has taken steps to bring in non-dilutive financing to cover some or all of the costs associated with its remaining programs, in order to reduce its cash expenditure.
AMT continues to work with St Jude's Children's Hospital in the USA, which is currently financing and conducting a clinical study in US and UK. Initial results are promising, with patients showing stable and persistent expression of the Factor IX clotting protein, and able to reduce or stop their administrations of protein replacement therapy, which is the current standard of care and requires intravenous infusion up to three times per week.
By contrast, the hemophilia B gene therapy requires a single administration to provide lasting benefit - the earliest patient was treated almost 18 months ago and so far has shown no detectable lessening of the benefit from this treatment. This is the second gene therapy program AMT is involved with to show clinical benefit from a single treatment and establishes AMT as the leading gene therapy company worldwide.
Acute Intermittent Porphyria
This program, in collaboration with the University of Navarra and Digna Biotech in Spain, is making encouraging progress. Earlier this year, the consortium won a significant EU grant worth approximately €1 million to AMT, which covers the majority of AMT's expenditure at this time for this program.
In August 2011, the consortium began enrolling patients into a pre-observation study. This initial study will provide baseline data for the subsequent treatment study, which involves administering patients with a one-time gene therapy and is expected to begin in 2012.
Duchenne Muscular Dystrophy
AMT is making good progress in delivery of gene therapy product to the heart. Heart failure is one of the main causes of death in Duchenne patients. The Compan
|SOURCE Amsterdam Molecular Therapeutics B.V|
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