Gene Therapy News

An innovative program project grant that will fund new approaches to gene therapy for alpha-1 antitrypsin deficiency has been awarded to a multidisciplinary team of researchers and clinicians at UMass Medical School by the National Heart, Lung and Blood Institute. The grant, totaling more than $11 million over five years, will be used to investigate three different genetically based approaches to treat the disease.

Graduate School of Biomedical Sciences Class of 2016 speaker Aditya Venkatesh will reflect on his path to science when he takes the stage at the 43rdUMass Medical School Commencement on Sunday, June 5. As a child growing up in India, he said he was fascinated by biology and dreamed of becoming a doctor.

Miguel Sena-Esteves, PhD, associate professor of neurology and a member of the Horae Gene Therapy Center, talked to the Washington Post about the progress he and others have made in developing a gene therapy treatment for the disease.

Scientists at UMass Medical School will share their latest research at an inKNOWvation panel discussion, “The Return of Gene Therapy,” on Wednesday, May 11, at the Massachusetts Biotechnology Council in Cambridge.

Researchers in the lab of Guangping Gao, PhD, have developed a novel gene therapy that cures the rare, inherited neurodegenerative disorder Canavan disease in mice and is on track to be brought to clinical trial

The Gene Therapy Center, a global leader in adeno-associated virus (AAV) gene therapy, has received a $2 million endowment from the Horae Oriental Shenzhen Investment Company of Guangdong, China. In recognition of the gift, the Gene Therapy Center has been named the Horae (红瑞) Gene Therapy Center.

Terence R. Flotte, MD, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost and dean of the School of Medicine, is the new editor-in-chief of the journal Human Gene Therapy and its associated periodicalsHuman Gene Therapy Methods and Human Gene Therapy Clinical Development, effective July 1. Dr. Flotte is currently an associate editor of the journal.

LYSOGENE today announced that it has entered into a strategic collaboration with the University of Massachusetts Medical School (UMMS) in Worcester, Massachusetts, and Auburn University (AU) in Auburn, Alabama. Through the collaboration, LYSOGENE, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus (AAV) gene therapy technology.

UMass Medical School celebrated the opening of Voyager Therapeutics’ new headquarters in Cambridge yesterday. Voyager is a gene therapy company founded by four world leaders in the fields of AAV (adeno-associated virus) gene therapy, RNA biology and neuroscience to develop life-changing treatments for fatal and debilitating diseases of the central nervous system.

UMass Medical School recognizes the launch today of Voyager Therapeutics, a gene therapy company founded by four world leaders in the fields of AAV gene therapy, RNA biology and neuroscience, to develop life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS).