Amorfix has begun to explore ways to target disease-causing, misfolded proteins and prevent them from forming aggregates. The best way would be to train the body to recognize the misfolded proteins and remove them just like it does with infectious agents like viruses when the immune system makes an antibody against the virus proteins.
Amorfix has succeeded in producing antibodies which recognize only the misfolded protein present in amyotrophic lateral sclerosis (ALS), in variant CJD and in some types of cancer.
This novel approach has been validated in preclinical models of ALS. Amorfix plans to extend this approach to other neurodegenerative diseases, such as Alzheimer's and Parkinson's diseases, and to cancer to develop new medicines.
The misfolded protein is not recognized by the immune system as abnormal. Amorfix has shown, in preclinical models, it is possible to boost the immune system and assist it to recognize the misfolded proteins and prevent infection with prions. This discovery points to the possibility of a vaccine for ALS or other neurological diseases.
Diagram: Amorfix Therapeutic Strategy
Amorfix's therapeutic platform involves therapeutic antibodies (red), which are designed to differentiate between normally folded proteins and disease-causing misfolded proteins. The antibodies bind specifically to the misfolded proteins and neutralize them.
Amoyotrophic Lateral Sclerosis (ALS) Treatment
Amorfix is developing new therapies for ALS disease by targeting misfolded SOD1 with antibodies and vaccines.
ProMIS™ - Disease Specific Epitope (DSE) targeting
Amorfix uses ProMIS™ to develop antibodies that target proteins likely to be misfolded in cancer and other diseases.