Amsterdam Molecular Therapeutics Provides Status Update on Glybera(R)

By Amsterdam Molecular Therapeutics B.v, PRNE
Monday, May 9, 2011

Company to Present Updated Glybera Biomarker Data at American Society of Gene and Cell Therapy Annual Meeting

AMSTERDAM, May 10, 2011 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, provided today a status update on Glybera
(alipogene tiparvovec), its gene therapy product under development for the
treatment of lipoprotein lipase deficiency (LPLD).

Following the submission of the Day 180 questions at the end
of the first quarter to the European Medicines Agency (EMA) as part of
Glybera's Market Authorisation Application (MAA), AMT continues to work
diligently with the EMA and is confident that a decision on the approval of
Glybera remains on track for mid-2011 as previously guided.

"Our interactions with the EMA continue to be positive and we
are encouraged as we hit each milestone in the path towards a regulatory
decision. If approved, Glybera would be the first gene therapy product to
reach the market in Europe. We will continue to work with the EMA so that the
review of our data remains on track as much as possible from our side and
look forward to a decision in mid-2011," stated Jörn Aldag, CEO of AMT. "Our
data are key to supporting our MAA. Later this month we will present
additional data at ASGCT regarding improved chylomicron handling, which we
believe could be used in the future by physicians as a biomarker for Glybera
in LPLD patients."

AMT scientists and collaborators will present data from
Glybera and several other gene therapy programs in a total of nine posters at
the American Society of Gene and Cell Therapy (ASGCT) 14th Annual Meeting to
be held in Seattle, USA, May 18-21. In particular, the company will provide
an update on previously announced initial results from a long-term clinical
study of Glybera that showed improved chylomicron metabolism could show
utility as a biomarker in LPLD patients.

Several posters will report advances made by AMT in the field
of RNAi delivery including additional data relating to Apoliprotein B
silencing in hypercholesterolemia and delivery of miRNA molecules into the
brain for development in Huntington's disease.

Details and times of the poster presentations can be found

[123] Absence of Integration Hotspots in Mice after
Intramuscular Injection of AAV1-LPLS447X Authors: C. Kaeppel, S. Beattie, A.
Nowrouzi, U. Appelt, R. Kirsten, H. Glimm, J. Snapper, C. von Kalle, H.
Petry, M. Schmidt Date/Time: Thursday, May 19, 2011 - 4:40 pm

[133] Construction of (Multi) Monomeric Duplex
Adeno-Associated Vectors for Human Gene Therapy Authors: Jacek Lubelski, Yvet
, Bas Bosma, Larbi Afia, Harald Petry, Andrew Bakker Date/Time:
Thursday, May 19, 2011 - 4:40 pm

[183] Improved Chylomicron Clearance as a Marker for Long-Term
Efficacy of Alipogene Tiparvovec (AAV1-LPLS447X Gene Therapy) in LPLD
Patients Authors: Jaap Twisk, Frederique Frisch, Stephen Greentree, Harald
, Janneke de Wal, Diane Brisson, Claude Gagné, André C. Carpentier,
Daniel Gaudet Date/Time: Thursday, May 19, 2011 - 4:40 pm

[551] Evaluation of the X-Protein Truncate Expression
Potential from the WPRE Element in Alipogene Tiparvovec, an AAV-Based Gene
Therapy Vector Authors: Jacek Lubelski, Florence Salmon, Betty Au, Larbi
, Andrew Bakker, Harald Petry Date/Time: Friday, May 20, 2011 - 4:40 pm

[562] In Vivo Comparison of the Long-Term Efficacy of AAV
Expressed ShRNA and MiRNA Targeting Apolipoprotein B100 Authors: Piotr
, Annemart Koornneef, Richard van Logtenstein, Florie Borel, Harald
, Sander van Deventer, Pavlina Konstantinova Date/Time: Friday, May 20,
- 4:40 pm

[564] Apolipoprotein B Knockdown by AAV-Delivered ShRNA Lowers
Plasma Cholesterol in Mice Authors: Annemart Koornneef, Piotr Maczuga,
Richard van Logtenstein, Florie Borel, Bas Blits, Tita Ritsema, Sander van
, Harald Petry, Pavlina Konstantinova Date/Time: Friday, May 20, 2011
- 4:40 pm

[566] Multidrug Resistance Transporters and miRNAs Expression:
Changes in Hepatocellular Carcinoma Authors: Florie Borel, Harald Petry,
Peter Jansen, Sander van Deventer, Pavlina Konstan-tinova Date/Time: Friday,
May 20, 2011
- 4:40 pm

[569] RNAi-Mediated Gene Therapy of Human Diseases Authors:
Annemart Koornneef, Piotr Maczuga, Florie Borel, Angelina Huseinovic, Peter
, Harald Petry, Pavlina Konstantinova Date/Time: Friday, May 20, 2011 -
4:40 pm

[847] Apolipoprotein B Knock-Down by AAV-Delivered sh/miRNA
Lowers Plasma Cholesterol in Mice Authors: Piotr Maczuga, Annemart Koornneef,
Richard van Logtenstein, Florie Borel, Bas Blits, Sander van Deventer, Harald
, Pavlina Konstantinova Date/Time: Saturday, May 21, 2011 - 6:15 pm

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based
therapies. The company's lead product Glybera(R), 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, and Parkinson's disease.
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

Certain statements in this press release are "forward-looking
statements" including those that refer to management's plans and expectations
for future operations, prospects and financial condition. Words such as
"strategy," "expects," "plans," "anticipates," "believes," "will,"
"continues," "estimates," "intends," "projects," "goals," "targets" and other
words of similar meaning are intended to identify such forward-looking
statements. Such statements are based on the current expectations of the
management of AMT only. Undue reliance should not be placed on these
statements because, by their nature, they are subject to known and unknown
risks and can be affected by factors that are beyond the control of AMT.
Actual results could differ materially from current expectations due to a
number of factors and uncertainties affecting AMT's business. AMT expressly
disclaims any intent or obligation to update any forward-looking statements
herein except as required by law.


For further enquiries: Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394, j.aldag at ; Mike Sinclair, Partner, Halsin Partners, Tel : +44-20-7318-2955, msinclair at

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