Warning: in_array() expects parameter 2 to be array, null given in /home/pharmalicensing/public_html/detail.php on line 234

Warning: in_array() expects parameter 2 to be array, null given in /home/pharmalicensing/public_html/inc_stats.php on line 82

Warning: array_push() expects parameter 1 to be array, null given in /home/pharmalicensing/public_html/inc_stats.php on line 85
Pharmalicensing | Life Science's Global Technology Marketplace
Save this technology
close
Save to Existing Project
Save to a New Project
RIP3 as a Novel Therapeutic Target for Gaucher's Disease
Yeda R&D Co. Ltd Israel flag Israel
Abstract ID: 1698
GD is an inherited metabolic disorder, affecting about 1 in 20,000 births. GD is divided into three clinical subtypes: type 1 is the most common and is characterized...
Contact
Participants
You
Email me a copy of this message
Introduction/Background

GD is an inherited metabolic disorder, affecting about 1 in 20,000 births. GD is divided into three clinical subtypes: type 1 is the most common and is characterized by bruising, fatigue, anaemia, low blood platelets, and enlargement of the liver and spleen. Types 2 and 3, also called neuronopathic GD (nGD), affect 4% of GD patients and additionally include neurological symptoms. Type 1 patients can have a normal life expectancy if treated whereas type 2/3 patients do not survive to reach adulthood. Moreover, GD carriers, approximately 1% of the population, are in a major risk of developing Parkinson's disease. Current therapies suffer from severe drawbacks in the treatment of type 1 GD and no therapy exists that effectively treat nGD.

Aims/Hypothesis

There is a need for novel therapies for type 1 GD and nGD.

Results

The proposed technology is based on the discovery that RIP3 is a key player in the manifestation of GD and that inhibiting RIP3 activity is effectively ameliorating the symptoms of GD not only in the less severe type 1 but also in the neuropathic form of the disease, types 2 and 3. nGD is associated with a massive neuronal loss and elevated RIP3 levels. Inhibition of RIP3 in a mouse model of nGD resulted in a dramatic attenuation of disease signs: drastic extension of life span, no weight loss, improvements in motor coordination, reduced neuroinflammation and improved liver and spleen injuries.

Conclusion

The present technology offers a novel therapeutic target for the treatment of Gaucher's disease (GD) which addresses also the neurological symptoms.

Relevance/Opportunity

Please enquire regarding licensing or codevelopment partnerships quoting reference no. 1698.
FEATURED
Last Updated May 2015
Technology Type MECHANISM
Phase of Development PRECLINICAL
CORPORATION