Platform

Using adeno-associated viral (AAV) derived vectors as the vehicle of choice for delivery of therapeutic genes, AMT 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. Following clinical testing in more than 60 trials, AAV vectors are generally considered to be safe.

Technologies:

AMT has developed a broad technology platform specifically designed to overcome major challenges associated with gene therapy. The following key technologies and capacities clearly differentiate our approach from other gene delivery systems:

• Platform vector technology
• Platform manufacturing technology
• Clinical development and regulatory expertise
• Modular platform focused approach

 

Platform vector technology

One of the key challenges in gene therapy is to identify a delivery vehicle (vector) that can effectively and safely carry a gene into the target cells and its nucleus. AMT’s AAV platform vectors offer the potential for safe and effective gene delivery. AAV vectors are generally considered safe and have been tested in over 60 clinical trials to date.

‘Wild type’, or naturally occurring AAVs, are non-pathogenic and do not in themselves cause disease in humans. Before use as vectors, these naturally occurring AAVs are ‘stripped’ so that they cannot replicate. The genes in wild type, and hence in our modified AAV vectors, are not able to integrate into the genome of the patient, unlike many other gene vectors. Instead a special version of a gene (extra-chromosomal) is created in each nucleus that guarantees long-term, persistent activity in the target cells. Non-integration is an important factor in determining the safety of our vector technology as it essentially eliminates the risk of inducing cancer, a risk seen with some other, non-AAV vectors.

Efficacy in patients requires lasting therapeutic gene expression in the target tissue. The extra-chromosomal version created after administration of the AAV-based gene therapy induces persistent therapeutic gene activity. Also, AAV can be used to target both dividing and non-dividing cells. Other gene therapy platforms cannot deliver genes into non-dividing cells. Targeting non-dividing cells has the added advantage of limiting the loss of activity of the gene therapy. Our experience with Glybera® for lipoprotein lipase deficiency (LPLD) has demonstrated that our technology is able to provide multi-year, tissue-targeted expression of the therapeutic protein following one-time administration.

Platform manufacturing technology

AMT has succeeded in developing a proprietary platform manufacturing technology that allows safe, effective, cGMP compliant, economically feasible and commercially scalable manufacturing of our products. In the past, the production of gene therapy products, especially AAV-based vectors, has been hampered by the challenges of scaling-up to commercial production (traditionally carried out in mammalian cells) in an economic way.

AMT’s novel approach is based on the use of a combination of baculoviruses and insect cells. It is a highly flexible process that can be easily and quickly adapted to produce a wide variety of products based on our vector technology, thereby significantly reducing the time needed for development.

Clinical development and regulatory expertise

AMT believes it has all the necessary skills to advance a product through every stage of clinical development and regulatory filing. We have established close links to the regulatory bodies and seek early discussions to ensure that our trials are in conformity with legal requirements. In developing and submitting Glybera® for approval, we have managed the entire process.

Modular platform focused approach

Using unique manufacturing technology, AMT is able to package a wide range of therapeutic genes into the relevant vector in a modular way. Therefore, we expect to be able to address a large number of disease indications. Based on the modular concept of our technology – the same vector type may carry different genes for different diseases – there is the possibility of faster development times, lower cost of development and lower sales infrastructure.

 

 

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DISCLAIMER – IMPORTANT

THE MATERIALS YOU ARE SEEKING TO ACCESS ARE BEING MADE AVAILABLE ON THIS WEBPAGE BY AMSTERDAM MOLECULAR THERAPEUTICS (AMT) HOLDING N.V. IN GOOD FAITH AND FOR INFORMATION PURPOSES ONLY. THE MATERIALS CONTAINED HEREIN ARE NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, DIRECTLY OR INDIRECTLY, IN WHOLE OR IN PART, OR DIRECTED AT OR ACCESSIBLE BY PERSONS LOCATED IN THE UNITED STATES OR ANY OTHER JURISDICTION WHERE TO DO SO WOULD CONSTITUTE A VIOLATION OF THE RELEVANT LAWS OR REGULATIONS OF SUCH JURISDICTION.

Please read this notice carefully - it applies to all persons who view this webpage. Please note that the disclaimer set out below may be altered or updated. You should read it in full each time you visit the site.

Non-Dutch persons

Viewing the materials made available on this website may not be lawful in certain jurisdictions. In other jurisdictions, only certain categories of person may be allowed to access such materials. Any person resident outside the Netherlands who wishes to access these materials must first satisfy themselves that they are not subject to any local requirements that prohibit or restrict them from doing so.

The materials made available on this website do not contain, constitute or form part of, and should not be construed as, an offer, or the invitation or solicitation to subscribe for or purchase any securities ("Securities") mentioned in the materials and nothing contained herein shall form the basis of, or be relied on in connection with, any offer or commitment whatsoever.

The Securities have not been (nor will be) registered under the US Securities Act of 1933, as amended (the "US Securities Act"), or the securities laws of any state or other jurisdiction of the United States, and may not be offered, sold, resold, taken up, exercised, renounced, transferred or delivered, directly or indirectly except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the US Securities Act.

No Securities will be offered or allotted directly or indirectly into, or by use of the mails of, or by any means or instrumentality of interstate or foreign commerce of, or any facilities of a national securities exchange of, the United States, unless an exemption from registration under the US Securities Act, is available. This includes, but is not limited to, facsimile transmission, electronic mail, telephone and the internet. Accordingly, copies of materials made available on this website and any related documents will not and must not be mailed or otherwise transmitted, distributed or forwarded in or into the United States.

The materials contained herein have not been submitted to or reviewed by the US Securities and Exchange Commission (the "SEC") or any state securities commission of any other jurisdiction, and neither the SEC nor any such state securities commission of any other jurisdiction has (a) approved or disapproved, (b) passed upon the merits or fairness of, or (c) passed upon the adequacy or accuracy of the disclosure of any materials contained herein. Any representation to the contrary is a criminal offence in the United States.

If you are not permitted to view materials on this webpage or are in any doubt as to whether you are permitted to view these materials, please exit this webpage.

Basis of access

Access to these materials is being made available on this webpage by Amsterdam Molecular Therapeutics (AMT) Holding N.V. in good faith and for information purposes only. Any person seeking access to this webpage represents and warrants to Amsterdam Molecular Therapeutics (AMT) Holding N.V. that they are doing so for information purposes only.

Neither any press announcement nor any other document made available on this website constitutes an offer to sell, an invitation to induce an offer or the solicitation of an offer to acquire, Securities in Amsterdam Molecular Therapeutics (AMT) Holding N.V. or Kairos Therapeutics B.V. and cannot be relied upon for any investment contract or decision. Furthermore, such documents do not constitute a recommendation by Amsterdam Molecular Therapeutics (AMT) Holding N.V. or any other party to sell or buy Securities.

Unless otherwise determined by Amsterdam Molecular Therapeutics (AMT) Holding N.V. and permitted by applicable law and regulation, copies of the contents of the following pages (including documents posted thereon) are not being, and must not be, released or otherwise forwarded, distributed or sent in or into the United States or any other jurisdiction where their extension or availability (and/or any transaction contemplated thereby) would breach any applicable law, and persons receiving such documents (including custodians, nominees and trustees) must not distribute or send them in, into the United States or any other jurisdiction where to do so would breach any applicable law.

Confirmation of understanding and acceptance of disclaimer

I certify that I am not located in the United States or any other jurisdiction where the release, publication or distribution of these materials would be unlawful.

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I confirm that I am permitted under applicable law and regulation to proceed to the electronic versions of these materials and receive information of the kind contained in this website.