See-Tx applications for Protein Misfolding diseases


We have initially applied See-TX to selective protein misfolding diseases and have targeted lysosomal enzyme, to address the unmet medical needs of rare and neurodegenerative diseases.

There are about 50 to 70 different lysosomal storage disorders and only for a portion, there are treatments but no cure exists. This represents a significant addressable market with a very high demand which can meet the medical needs of a large pediatric population.

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At the same time, a malfunctioning protein leads to the molecular onset of some neurodegenerative diseases. Neurodegenerative diseases are large, and there is a growing population with no disease-modifying therapies on the market. But only symptom relief approaches exist currently.

Gain is therefore working towards addressing those aspects for some of those disorders. There are other targets and disorders we are currently working on,  such as mitochondrial and metabolic disorders. As previously described by Professor Barril and Dr. Broder, there are several targets and molecular aspects of cancer metabolism that warrant the development of new drugs.

Thus we have started entering into oncology research as well. 

Thanks to See-TX being an agnostic broadcasting technology, we are currently working on four different therapeutic areas addressing the needs of nine different disease indications.

Later today I will be presenting our program targeting GBA in Parkinson’s disease. Here, we are currently performing new clinical regulatory studies on our candidate drugs for Parkinson’s. Additionally to Gaucher, our current programs in the Lysosomal story Disorder therapeutic field are GM1 gangliosidosis, mucopolysaccharidosis, type 1 Krabbe disease, and metachromatic leukodystrophy. In the metabolic therapeutic areas, we have identified a target to address the needs of a rare liver and lung condition in Oncology.

We have identified a relevant Oncology target and are currently working on it as mentioned by Dr. Broder as well. We are also in the process of selecting a new cancer target later this year.

The current status of our GM1 program is that we have entered a lead optimization with molecules showing interesting data supportive of beta-galactosidase stabilization. Toxic software depletion with molecules being orally bioavailable, brain and bone penetrant.

In our Mucopolysaccharidosis type 1 program, we have identified a tool compound targeting IDUA which can stabilize exogenous recombinant IDUA while ameliorating its biodistribution also towards tissue that current medication cannot penetrate.

We have also started lead series identification to target GALC for Krabbe disease and we are happy to report that some early molecules are already showing a positive toxic software depletion supportive of the continuation of this program.

A new program to the Gain pipeline is targeting metachromatic leukodystrophy and where we have already identified a unique allosteric pocket on ARSA.

Another new program is targeting a rare liver and lung disorder where we have selected a target on which a unique allosteric site has been identified thanks to See-TX and where the hit-to-lead series has been initiated.

I would like to introduce to you our new targets in oncology where a unique allostatic site has been identified on a relevant cancer target. We are currently not disclosing, but where we are performing experimental validation, which is ongoing. Additionally, we are in the process of selecting a new target in oncology as well. I am sure you will appreciate this during this R&D Day. See-TX has been generating data validating its approach which has allowed us to broaden its application to other relevant therapeutic areas.