Founding Member, Whitehead Institute for Biomedical Research and Professor of Biology and Professor of Biological Engineering, MIT
Bio: Harvey Lodish has been a biotechnology entrepreneur for over four decades; he was a founder 12 companies including Genzyme, Inc. and Millennium Pharmaceuticals, Inc. In 2014 he started Rubius, a now public company developing genetically modified human red blood cells for multiple therapeutic indications. His lab research has primarily focused on cloning and characterizing proteins and RNAs important for red cell development and function, as well as several genes and proteins involved in insulin resistance and stress responses in adipose cells. Two of his students have won Nobel Prizes and eight have been elected to the US National Academy of Sciences.
Find out more about Harvey & MIT by visiting their website.
Q. This will be CamRARE’s 5th summit. What is the most impactful change you’ve seen happen in rare disease progress in the last 5 years?
Development of many platform technologies in molecular and cellular biology that offer promise for many types of gene therapies and gene editing to treat rare diseases.
Q. How has your work impacted rare disease progress over the last 5 years?
In many ways, which I will describe in my presentation.
Q. What next? What do you want to see happen or change in the next 5 years that will have the most impact on diagnostics, care or treatment for those affected by rare conditions?
Diagnosis of the many rare diseases that are caused by consanguineous marriages in unstudied ethnic groups in many countries throughout the world.