The US Food and Drug Administration (FDA) has given an orphan drug to a new gene therapy for Amyotrophic Slippers (ALS) developed at Universitat Autònoma de Barcelona and a license to the US company Klotho Neurosciences, Inc.
The drug uses an AAV viral carrier (ADeno associated with the virus) that expresses the secretive isomorphic of the Klotho protein (S-KL), with neuroeacheric, antioxidant and anti-inflammatory properties. In order to reach the neuromuscular connections affected by ALS’s disease, the carrier acts under the control of a DNA sequence that regulates protein expression especially in the muscle. This innovative approach has shown very promising results in the most widely used mouse model for the preclinical study of ALS, delaying the onset of the disease, maintaining neuromuscular function and expansion of survival.
Technological Development was guided by UAB researchers, with the participation of the Ciber, ICREA and Vall D’Hebron Research Institute of Research Nasdaq in 2023 (Nasda: Kltoq: Klto)-. Technology was developed by the research teams of Assumpció Bosch and Miquel Chillón, both by the Department of Biochemistry and the Department of Biochemistry and Molecular Biology and the UAB Institut De Neurociències (INC-UAB). The research program also included the collaboration of the group led by Professor Xavier Navarro, researcher at the Institut de Neurociències and the UAB’s Cell Biology, Physiology and Immunology Department of Neurosacial and Mobile Neurons.
The orphan characterization of medications for the treatment we have developed recognizes the relevance of therapies aimed at muscles and neuromuscular intersections as a strategy for ALS “.
Assumpció bosch, main researcher of the study
“To date, we have been able to demonstrate efficiency in a top animal model for this pathology. We are now trying it on other models als to confirm that this therapeutic solution can be applied to the widest possible number of patients,” adds Sergi Verdés, a postdoctoral researcher to the research team.
FDA’s receipt of the orphan drug highlights the ability to treat the rare and severely disabled AlS disease, which affects about 65,000 people in Europe and for which there is no effective treatment. This recognition offers advantages such as seven years of exclusivity for the drug in the US market, remuneration exemptions and tax incentives for clinical trials.
Klotho Neurosciences will now launch the construction of the body, followed by meetings with the FDA and the European Medicines Agency (EMA) in the near future in order to move to the first clinical trials in patients.
