Gene Therapy Sanfilippo B
AMT & Institut Pasteur
Jean-Michel Heard email@example.com
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in human gene therapy, announced that it has entered into an agreement with Institut Pasteur, Paris, France, and a group of French research institutes (together the “Consortium”) to support clinical development of a novel gene therapy to treat Sanfilippo B.
On behalf of the Consortium, Institut Pasteur will lead the development program and will also sponsor the initial Phase I/II clinical study of a gene therapy to replace an enzyme (alpha-N-acetylglucosaminidase) that is missing in brain cells of Sanfilippo B patients. This enzyme is specifically required for the degradation of heparan sulfate glycosaminoglycans (GAGs), essential carbohydrate molecules used to build tissue. The accumulation of incompletely degraded GAG molecules triggers a cascade of pathological events leading to neuronal dysfunction and death.
AMT will manufacture and supply the adeno-associated viral 5 (AAV5) gene therapy product to the Consortium. Thanks to donations collected during the French Telethon, the French Muscular Dystrophy Association (AFM), a Consortium member, will fully fund the development program through to completion of the Phase I/II clinical study, including all AMT manufacturing costs. The overall manufacturing contract entails payments to AMT of EUR1.8 million. If the Consortium successfully demonstrates proof of concept in the Phase I/II study, AMT will have an option to acquire full commercial rights for the program.
Gene Therapy Sanfilippo A
Hôpital Bicêtre – Assistance Publique des Hôpitaux de Paris
Le Kremlin Bicêtre, France, 94275
Hôpital Necker, Assistance Publique des Hôpitaux de Paris
Paris, France, 75015
Michaël HOCQUEMILLER, PhD
Scientific & Clinical Affairs
52 rue la Boétie 75008 PARIS
Alliance Sanfilippo coordinated discussions between researchers and clinicians susceptible of being interested in developing a similar clinical trial for Sanfilippo Syndrome type IIIA. All of them expressed their desire to work together in exchanging expertise, reagents and preclinical data
Gene therapy program for intracerebral administration of an adeno-associated virus vector to replace Sulfamidase enzyme missing in Sanfilippo A patients. The program is structured in phases with synergized management of activities and partnerships:
- Construction and validation of an adeno-associated vector with good brain tropism: a AAV vector (serotype 10) encoding human SGSH and SUMF1 cDNA for treatment of type IIIA Sanfilippo syndrome
- Effectiveness studies carried out on mouse models
- Vector production according to GMP (Good Manufacturing Practice) quality standards
- Regulation toxicological studies
- Preparation of a protocol for open,phase I/II clinical trials to assess the tolerance of intracerebral administration of this adeno-associated vector to treat type IIIA Sanfilippo syndrome
- Management of regulatory affairs in cooperation with the competent health authorities
Study launched under the express condition that proper authorization be granted by health authorities
The phase I/II trial will be start on August 2011. 4 Children, 18 months to 6 years.
Chaperone Therapy Sanfilippo B
CNRS / UMR
Dr. Matthieu Sollogoub firstname.lastname@example.org
Conception and evaluation of new chemical chaperones of the N-acetyglucosaminidase in the context of the development of a treatment for Sanfilippo Syndrome (MPS III B).
This research and development project, organized by the team of Professor Matthieu Sollogoub (CNRS / UMR, Université Pierre and Marie Curie, Paris), consists of developing glycosidic inhibitors of the N-acetyglucosaminidase.
It is expected that these new molecules will be able to correct the structure and intracellular transport of certain mutated forms of the N-acetyglucosaminidase and therefore restore the ability to degrade heparan sulfate.
The Swiss Sanfilippo Foundation create a commercial company, SanOrphan, in February 2011 in order to raise more funds from venture capital in relation to the program of Dr. Sollogoub, the aim is to perform trial as soon as possible. (http://www.sanorphan.com – website under construction)