Research & Development


AAVAA now collaborates with Horae Gene Therapy Center and RNAi Therapeutics Institute and gains full access to clinical trial experience of UMMS.
∙ UMMS was founded in 1962.

∙ Federal and private research grants and contracts of approximately $250 million in fiscal year 2018.

∙ In 2006 the Nobel Prize in Physiology or Medicine was awarded to UMMS professor Craig C. Mello, PhD, for their discoveries related to RNA interference (RNAi), a cellular process that offers astounding potential for understanding and, ultimately treating, human disease.

∙ UMMS research programs are central to the
Massachusetts Life Sciences Initiative, with major funding from the $1 billion since 2008.

University of Massachusetts Medical School, Worcester, MA
∙ The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited disease for which there is no cure.

∙We utilize state of the art technologies to either edit mutated genes that produce disease-causing proteins or introduce a healthy copy of a gene if the mutation results in a non-functional protein.

∙The University of Massachusetts conducts clinical trial on site and some of these trials are conducted by the investigators at the Gene Therapy center.

∙The RTI was founded in 2009 and became an academic department in 2016, chaired by Phillip D. Zamore, Ph.D.

∙The RTI is dedicated to leveraging the strong RNA biology and clinical research communities at UMMS to develop novel therapies for which RNA is the therapeutic target or modality.

∙The RNA Therapeutics Institute faculty are recognized as scientific trailblazers, and include a Nobel Laureate, a Gairdner Prize recipient, the 2018 RNA Society early and mid-career award recipients, and two Howard Hughes Medical Institute Investigators.

Research & Development

Severance Hospital