Gene Therapy Worldwide

Geographical Distribution of Gene Therapy Trials
		Gene Therapy Clinical Trials
	AMT				Number	%
				America	950	64.5
				Europe	427	29.0
				Asia	52	3.5
				Austalasia	29	2.0
				Africa	1	0.1
				Multi-country	13	0.9
					1472	100

 

Indications Addressed by Gene Therapy Trials
		Gene Therapy Clinical Trials
	AMT				Number	%
				Cancer diseases	960	65.2
				Cardiovascular	137	9.3
				Gene marking	50	3.4
				Healthy volunteers	32	2.2
				Infectious diseases	112	7.6
				Monogenic diseases	120	8.2
				Neurological diseases	17	1.2
				Ocular diseases	12	0.8
				Others	32	2.2
					1472	100

 

Vectors used in Gene Therapy Trials
		Vectors used in Gene Therapy Trials
	AMT				Number	%
				Adenovirus	367	24.5
				Retrovirus	320	21.3
				Naked/Plasmid DNA	270	18.0
				Vaccinia virus	120	8.0
				Lipofection	105	7.0
				Poxvirus	90	6.0
				Adeno-associated virus	60	4.0
				Herpes simplex virus	47	3.1
				RNA transfer	21	1.4
				Other categories	60	4.0
				Unknown	41	2.7
					1472	100

 

Phases of Gene Therapy Trials
		Gene Therapy Clinical Trials
	AMT				Number	%
				Phase I	888	60.3
				Phase I/II	280	19.0
				Phase II	244	16.6
				Phase II/III	13	0.9
				Phase III	47	3.2
					1472	100

Web Links

Genes, Diseases and Gene Therapy

MEDLINEplus: Genes and Gene Therapy
Access news, information from the U.S. National Institutes of Health, clinical trials information, research, and more.

Orphanet
All kinds of information on rare diseases.

Fundamentals of Gene Therapy
Diagrams and basic description of gene therapy from the U.S. Food and Drug Administration (FDA).

Recombinant DNA and Gene Transfer
National Institutes of Health Guidelines.

Gene Therapy and Children
From KidsHealth for Parents.

Gene Transfer
An overview of gene therapy science issues, ethical concerns, and regulation and policy from the Genetics & Public Policy Center, John Hopkins University.

Gene Therapy Clinical Trials

Gene Therapy Clinical Trials in the United States
Gene therapy in the U.S. National Institutes of Health database of clinical trials.

Gene Therapy Ethics

Gene Therapy
Ethical Issues

Regulatory Agencies

European Medicines Agency (EMA)
Details about and links to all EMA information.

U.S. Food and Drug Administration (FDA)
Details about and links to all FDA cellular and gene therapy product information.

Patient Groups Europe - General

European Organisation for Rare Diseases (EURORDIS)

European Genetic Alliances' Network (EGAN)

Patient Groups Europe – Disease Specific

European Parkinson's Disease Association (EPDA)

Patient Groups United States - General

Genetic Alliance
National Organization for Rare Disorders (NORD)

Patient Groups United States – Disease Specific

CureDuchenne
Muscular Dystrophy Association
American Parkinson Disease Association (APDA)
The Michael J. Fox Foundation for Parkinson's Research

Professional Associations

American Society of Gene & Cell Therapy

European Society of Gene and Cell Therapy (ESGCT)

Gene Therapy Journals

www.genetherapynet.com

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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.

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