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Leucodistrophie-Repositioning: Laquinimod für Zellweger-Syndrom

Ref-Nr: TA-MBM-BioT-1895-UMG


Kurzfassung

Das Zellweger Syndrom, eine seltene Krankheit, ist eine Störung der peroxisomalen Biogenese für die es derzeit keine Heilung gibt. Wir bieten an, das klinisch bekannte Laquinimod (entwickelt für Multiple Sklerose) zur Therapie von Zellweger Syndrom


Hintergrund

Zellweger Syndrome spectrum, an orphan disease, is a peroxisomal biogenesis disorder with no treatment currently available. We offer the use of the clinically known Laquinimod (developed for multiple sclerosis) for the therapy of Zellweger Syndrom, for which we achieved successfully an in vivo proof-of-concept.


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Problemstellung

Leukodystrophies are metabolic and/or hereditary CNS disorders. Among them are peroxisomal disorders, characterized by failure of organelle formation (peroxisome biogenesis disorders) or a defect in a single peroxisomal protein or a distinct peroxisomal pathway. Zellweger Syndrome spectrum (ZSS) is a peroxisomal biogenesis disorder. ZSS can be caused by mutations of the proteins required for peroxiome biogenesis (Pex proteins/peroxins).

Characteristic features: neuropathologically, neuronal migration defects and progressive symmetric white matter abnormalities in the CNS.

No treatment is currently available for ZSS. Severely affected patients die within months.


Lösung

We offer Laquinimod and other quinoline-3-carboxamide derivatives for the therapy of Zellweger Syndrome (ZSS) or similar Leukodystrophies. Laquinimod is a quinoline-3-carboxamide derivative having a strong background in modulating innate immunity, i.e. in animal model of multiple sclerosis (MS) and has been evaluated for the treatment of MS.


Vorteile

  • Successful in vivo proof of concept.
  • Improved walking ability with laquinimod treatment.
  • Laquinimod reduces microglia activation (less inflammation).
  • Laquinimod reduces demyelination and axonal loss.
  • Clinical and histopathological in vivo evidence.
  • Similar pathological findings as in ZSS also occur in other metabolic or hereditary CNS disorders (Leukodystrophies) and could be potentially treated.

Anwendungsbereiche

To be used for the therapy of Zellweger Syndrome or similar Leukodystrophies.


MBM ScienceBridge GmbH

Dr. Stefan Uhle
0551-30724 154
suhle@sciencebridge.de
www.sciencebridge.de
Adresse
Hans-Adolf-Krebs-Weg 1
37077 Göttingen



Entwicklungsstand

Präklinik


Patentsituation

  • EP EP16767186.6 anhängig
  • US US15758098 anhängig

Stichworte

laquinimod, zellweger, leucodistrophy, repositioning, orphan, carboxamide, MBM ScienceBridge GmbH, Technologieangebot/technology offer, Technologietransfer/ technology transfer, Georg-August-Universität Göttingen Universitätsmedizin

Angebot Anbieter-Website


Kontakt | Geschäftsstelle

TechnologieAllianz e. V.
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(Geschäftsstellenleiterin)

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D-45468 Mülheim an der Ruhr