Recently, Modis Therapeutics raised a $30 million round to advance the development of a new treatment for a mitochondrial disease, called MT1621. This therapy was developed by Dr. Ramon Martí, head of the Neuromuscular and Mitochondrial Pathology Research Group of the Vall d'Hebron Research Institute, and Dr. Michio Hirano at Columbia University, and in 2016 it was licensed to the company Modis Therapeutics, specifically created to bring patients this therapy.In 2015, the laboratories of Dr. Michio Hirano and Dr. Ramon Martí discovered and protected a new treatment for a mitochondrial disease caused by the deficiency of TK2 (TK2d). The TK2 gene encodes the enzyme thymidine kinase, a key protein for good cellular functioning. This innovative treatment, a combination of nucleosides, has been successfully tested in animal models with a deficiency in TK2.A year later, in 2016, VHIR and Columbia University licensed this treatment to the company Modis Therapeutics, which was created "ad hoc" in the United States to assess this therapy. They have not only licensed the patent family but the orphan drug designations of the European and American regulatory agencies (EMA and FDA), as well as the drug designation for the rare pediatric disease by the FDA."This is a unique opportunity to develop orphan drugs with a very high therapeutic impact on a very serious illness that affects both children and adults. We are fortunate to have solid clinical data, the collaboration of committed doctors and scientists and two recently published studies on TK2d to help interpret the effect of the treatment", remarks Joshua Grass, CEO of Modis Therapeutics."The increasing use of advanced DNA sequencing technology to diagnose patients with mitochondrial diseases at the genetic level creates new opportunities to direct therapeutics to specific causes of the disease, which will result in great benefits in the treatment. TK2 deficiency may be the first of several mitochondrial DNA depletion disorders that can be addressed with this strategy or other methods that are being investigated by Modis Therapeutics and its collaborators", concludes Grass.