团队

Michael Rosbash is an American geneticist and chronobiologist, a professor and researcher at Brandeis University and investigator at the Howard Hughes Medical Institute. Michael Rosbash is interested in the RNA processing, genes, and mechanisms that underlie circadian rhythms – the 24-hour pacemaker that is nearly ubiquitous among higher organisms. Rosbash's research group cloned the Drosophila period gene in 1984 and proposed the Transcription Translation Negative Feedback Loop for circadian clocks in 1990. In 1998, they discovered the cycle gene, clock gene, and cryptochrome photoreceptor in Drosophila through the use of forward genetics, by first identifying the phenotype of a mutant and then determining the genetics behind the mutation. Rosbash was elected to the National Academy of Sciences in 2003. Along with Michael W. Young and Jeffrey C. Hall, he was awarded the 2017 Nobel Prize in Physiology or Medicine "for their discoveries of molecular mechanisms controlling the circadian rhythm".

Nahum Sonenberg, OC FRS FRSC is an Israeli Canadian microbiologist and biochemist. He holds theis a Gilman Cheney Chair in Biochemistry and is a James McGill Pprofessor at of biochemistry at McGill University in Montreal, Quebec, Canada. He was an HHMI international research scholar from 1997 to 2011 and is now a senior international research scholar, 2012-2017. Dr. Sonenberg studies the molecular basis of the control of protein synthesis in eukaryotic cells and its importance in diseases such as cancer, obesity, diabetes and neurological diseases. He is best known for his seminal contributions to our understanding of translation, and notable for the discovery of the mRNA 5' cap-binding protein, eIF4E, the rate-limiting component of the eukaryotic translation apparatus. Dr. Sonenberg is the recipient of the Wolf Prize in 2014, the Gairdner Foundation International Award, 2008 and elected Foreign Associate, US National Academy of Sciences in 2015.

Guangping Gao, PhD is the Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems, Penelope Booth Rockwell Professor in Biomedical Research, UMass Chan Medical School. Dr. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, hugely impacting many currently untreatable human diseases. Dr. Gao is an elected fellow of the US National Academy of Inventors (NAI), holding 237 patents with 493 more patent applications pending; some have been licensed to 10+ pharmaceutical companies. Dr. Gao co-founded Voyager Therapeutics and Aspa Therapeutics, focusing on developing rAAV gene therapeutics for treating a variety of devastating CNS disorders.

Gerald (Gerry) Cox, MD, PhD is the founder of Gerald Cox Rare Care Consulting, where he advises biotech companies and provides clinical development solutions for investigational rare disease treatments. Prior to that, he led clinical development programs of CRISPR-editing therapies for genetic diseases as Chief Medical Officer for Editas Medicine. Earlier in his career, Dr. Cox served as Vice President of Rare Disease Clinical Development at Sanofi Genzyme, where he oversaw clinical development programs for several rare disease treatments, including for Aldurazyme^®, Myozyme^®, Elaprase^®, Cerdelga^®, Xenpozyme^®, Hectoral^®, Cerezyme^®, and venglustat. Dr. Cox is a part-time practicing geneticist at Boston Children's Hospital and an instructor in Pediatrics at Harvard Medical School.  He is board-certified in Clinical, Biochemical, and Molecular Genetics.  Dr. Cox is on the board of directors for the National Tay-Sachs and Allied Diseases patient advocacy organization, and he is a member of the scientific and medical advisory committees for the National Organization for Rare Diseases and the CTNNB1 patient advocacy organization.  He is a member of the Data Monitoring Committee for two gene therapy companies. Dr. Cox’s clinical interests include novel approaches to treating genetic diseases, genetic eye diseases, lysosomal storage disorders, pediatric cardiomyopathy, neuromuscular disorders, and Barth Syndrome.