University of California

Fondazione Telethon Ets

Other

Massachusetts Institute of Technology

The University of Texas System

The United States Government as Represented by the Department of Veterans Affairs

Marine Biological Laboratory

Universitat Duisburg-Essen

Universitat Ulm

Gal Bitan

Thomas Schrader

Antonio Monaco

Alessandro Fraldi

Sharmistha Sinha

Alberto Auricchio

Luitgard Nagel-Steger

Sally A Frautschy

David J Busch

Irene Sambri

Julia März-Berberich

Katrin Hochdörffer

Frank Bastkowski

Jolanta Polkowska

Peter Talbiersky

Frank Gerrit Klarner

Aleksey Lomakin

Akila Shanmugam

Jennifer R Morgan

Andrea Sowislok

Annika Röcker

Ravinder Malik

Christian Heid

Danielle Bouquio

Frank Bastowski

Sally Frautschy

In various embodiments methods for the treatment and/or prophylaxis of lipofuscin-related disorders are provided. In certain embodiments the methods involve administration of an effective amount of a molecular tweezers to a subject in need thereof.

Inhibition of lipofuscin aggregation by molecular tweezers

In various embodiments methods are provided for the treatment or prophylaxis of liposomal storage diseases. In certain embodiments the methods involve administering to a subject in need thereof one or more molecular tweezers that inhibit protein aggregation.

Treatment of lysosomal storage disorders

Methods for the treatment of spinal cord injury or traumatic brain injury are provided. In certain embodiments, the methods include the use of a molecular tweezers and/or nucleobase oligomer capable of inhibiting the accumulation or aggregation of one or more amyloidogenic proteins and/or synuclein proteins. Examples of treatments that may inhibit accumulation or aggregation of one or more amyloidogenic proteins and/or synuclein proteins include treatment with a synuclein antisense nucleobase oligomer or treatment with the molecular tweezers CLR01. These treatments may improve outcomes of spinal cord surgery or traumatic brain injury, including, without limitation, neuronal survival, neuronal regeneration, and recovery of neuronal functions.

Treatment of spinal cord injury or traumatic brain injury by inhibition of synuclein protein aggregation

This invention provides novel compositions to inhibit the aggregation of amyloid proteins. In various embodiments the compositions comprise a molecular tweezers that binds lysine and/or arginine and thereby inhibits the aggregation of amyloidogenic proteins. 

Molecular tweezers for the treatment of amyloid-related diseases

The present invention relates to the field of protein misfolding diseases and thus to diseases which are associated with or induced by abnormal or pathogenic three-dimensional folding of proteins and/or peptides or which are linked to pathogenic conformational changes of proteins and/or peptides, such as Alzheimer's disease. Particularly, the present invention provides novel trimeric pyrazole compounds, which exhibit a therapeutic effectiveness in regard to the aforementioned protein misfolding diseases, and refers to their use for the treatment of such protein misfolding diseases, especially neurodegenerative diseases as well as to medicaments or pharmaceutical compositions comprising these compounds.

Compounds for the treatment of diseases related to protein misfolding

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Los Angeles, CA, United States of America

Gal Bitan