The US Food and Drug Administration (FDA) has given an orphan drug design for a new gene therapy for the Ameotrophic Lateral Sclerosis (ALS) developed in Universitate Autonem de Barcelona and US company Clotho Neurosis, Inc.
The drug uses a viral vector of AAV (Adeno-Juda Virus) type that expresses neuroregensing, antioxidants and anti-inflammatory properties with clotho (S-KL) secreted isoforms of proteins. To reach neuromuscular junctions affected by ALS disease, the vector acts under the control of a DNA sequence that controls the expression of protein, especially in muscles (a muscle-specific promoter), so that therapeutic activity is directed towards neuromuscular junctions. This innovative approach has shown very promising results in the most widely used mouse model for pre -pregnant studies of ALS, delaying the onset of the disease, preserving a neuromuscular function and expanding existence.
The technological development was led by the UAB researchers, with the participation of Ciber, ICREA and VAL D’Hibron Research Institute, the co-owner of intellectual wealth related to the use of cloths and the co-owner of the use of protein and the Clotho neurocains-e-up was licensed on the basis of knowledge generated in the UAB and a license was formed to the 2023 (Nasdak. Technology was developed by research groups of assumpció bosch and miquel chillón, both UAB Department of Biochemistry and Molecular Biology and UAB Institute De Neurocaines (Inc-UAB), Professor Xavier Narvaro and Celller Vivor Sciences, Body Sciences and Celller in Research Project. The cooperation of the group was also included in collaboration with the researcher of the UAB department of immunology and a specialist of neuroregation and motor neuron diseases.
The therapy for which we have developed an orphan drug design accepts the relevance of the muscles and neuromuscular junctions target as a strategy for ALS. ,
Assumpció bosch, head investigator of study
“To date, we are able to display Efficacy In a major animal model for this pathology. Now we are testing it in other ALS models to confirm that this medical solution can be applied to the wide possible number of patients “, the postdoctoral researchers of the research team say Serji Vardes.
Getting an orphan drug design by the FDA underlines the ability of treatment for rare and severely disabled disease ALS, which affects about 65,000 people in Europe and there is no effective treatment for which. This recognition provides benefits such as seven years of specificity for drugs in the US market, fee discount and tax incentive for clinical trials.
Clotho neurocaines will now start the construction of the vector, followed by meetings with the FDA and European Medicine Agency (EMA) in the near future to the first clinical tests in patients.
