Date: 2014-07-17
Type of information: Publication of results in a medical journal
phase:
Announcement: publication in Science
Company: aTyr (USA - CA) The Hong Kong University of Science and Technology (China) The Scripps Research Institute (USA - CA) Stanford University (USA - CA)
Product: physiocrines
Action mechanism:
Disease:
Therapeutic area: Rare diseases
Country:
Trial details:
Latest
news: * On July 17, 2014, aTyr Pharma, an innovative rare disease therapeutics enterprise, and its subsidiary in Hong Kong, Pangu Biopharma (aTyr), announced the discovery of a new class of human proteins with therapeutic relevance for a wide range of diseases. The findings unveiled in a publication in Science further supplies the basis for aTyr’s ongoing clinical studies in the areas of rare diseases and represents an entirely new and unmined area of human biology for therapeutics. Scientists from aTyr, The Hong Kong University of Science and Technology (HKUST), The Scripps Research Institute (TSRI) and Stanford University reported for the first time that an entire enzymatic gene family can generate novel splice variants that possess non-enzymatic, extracellular activities. Using deep sequencing, mass spectroscopy, protein purification techniques and parallel cell-based assays, the team found nearly 250 new proteins with previously unidentified activities spanning from stem cell biology to immunology. The vast majority of these novel proteins, called Physiocrines, lacked catalytic domains from the gene family, thereby creating new cellular functions. “We believe this work will enable us to harness the regulating power of these proteins to heal disease states where human physiology is dysregulated,” said John Mendlein, Ph.D., CEO and Executive Chairman of aTyr and co-author of the study. “During evolution this ancient gene family played a critical role in cellular life – catalysis of protein synthesis. Our international effort to understand this enzymatic gene family now leads us to a completely new horizon for all of human physiology – the alternate use of a gene family for non-catalytic regulation of basic physiological processes of humans, such as stem cell biology, immune pathways, vascularization and metabolism. We feel very privileged to have completed the work with our colleagues from HKUST, Stanford University and TSRI and the visionary influence of Dr. Paul Schimmel, Ernest and Jean Hahn Professor of Molecular Biology and Chemistry at The Scripps Research Institute (California and Florida) who also holds an appointment at the Institute for Advance Study (IAS) at The Hong Kong University of Science and Technology.” The paper, “Human tRNA Synthetase Catalytic Nulls with Diverse Functions,” was published in the July 18, 2014 issue of Science, doi: 10.1126/science.1252943.
