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Amsterdam Molecular Therapeutics BV receives global rights to develop and
commercialize AMT-020 for acute intermittent porphyria
Rights from University of Navarra, Pamplona, Spain strengthen AMT’s portfolio of
gene therapy products
Amsterdam, the Netherlands, February 28 2007 – Amsterdam Molecular
Therapeutics BV (AMT), a leader in the field of human gene therapy, announces
that it has obtained exclusive worldwide rights to develop and commercialise
AMT-020 as a therapeutic product from UTE CIMA, Proyecto de Biomedicina CIMA,
S.L. and Digna Biotech (all parties part of the University of Navarra, Pamplona,
Spain).
The licence allows AMT to initiate the clinical development of AMT-020 to treat
acute intermittent porphyria.
This follows successful collaboration between CIMA and AMT, demonstrating the
safety and preclinical efficacy of the product, AMT-020. AMT-020 is an AAV
vector gene therapy containing the porphobilinogen deaminase gene which encodes
for the enzyme that is defective in acute intermittent porphyria. This disease
is associated with recurrent attacks of abdominal pain, gastrointestinal
dysfunction, and neurologic disturbances.
AMT-020 builds the Company’s gene therapy portfolio. AMT’s lead product is
AMT-011, in phase II trials for the treatment of genetic lipoprotein lipase
(LPL) deficiency type I.
-Ends-
About AMT
Amsterdam Molecular Therapeutics BV (AMT) is a gene therapy company founded by
scientists of the University of Amsterdam Medical Center (AMC) in 1998. AMT
focuses on the development of gene-based therapies for orphan metabolic and
ocular diseases. AMT’s long-term gene expression technology is based on specific
delivery of therapeutic genes into target organs or tissues. Production of AAV-based
gene therapy vectors has been optimized and AMT has developed and validated a
unique, stable and scalable GMP production platform. Its lead product, AMT-011,
is in phase II for the first indication: treatment of lipoprotein lipase
deficiency type I.
The company's Management, Supervisory and Scientific Advisory Board bring
together an extensive know-how from both gene science as well as the biotech and
pharmaceutical worlds. For further information, go to
www.amtbv.com .
About Acute Intermittent Porphyria
Acute intermittent porphyria is an inherited disease characterised by attacks of
acute abdominal pain, muscular weakness and a complex array of neurovisceral and
psychiatric symptoms that may be life threatening if treated incorrectly. There
is also an increased risk of primary liver cancer and renal failure. The attacks
are normally triggered when the heme synthesis is induced by infections, stress,
alcohol, hormones, or specific drugs. It is estimated that prevalence of the
disease can be as high as 60-100 cases per 100,000 population in northern
Sweden. Women are more commonly affected than men.
Acute intermittent porphyria is a metabolic error in heme biosynthesis in which
the porphobilinogen deaminase (PBGD) is partially deficient by inactivating
mutations in one allele. The condition is inherited in an autosomal dominant
pattern with incomplete penetrance. The prevalence of symptomatic disease is 1–2
per 20,000 individuals, and women are more frequently affected than men.
For further information, please contact:
Media contact:
Northbank Communications
Adam Michael, Account Director
Phone: +44 20 7268 3002
Email: a.michael@northbankcommunications.com
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