Viralgen Partners with Broad Institute to Develop Gene Therapy for Prion Disease

• Viralgen has partnered with labs at the Broad Institute of MIT and Harvard University to develop gene therapy for prion disease.

• The collaboration aims to create an AAV-based treatment, targeting genetic neurodegenerative disorders with no current cure.

Viralgen, a leader in AAV gene therapy manufacturing, has joined forces with two labs from the prestigious Broad Institute of MIT and Harvard University to develop an innovative treatment for prion disease. This partnership brings together expertise in gene therapy manufacturing and cutting-edge research on neurodegenerative diseases.

Prion disease is a fatal condition caused by protein misfolding, leading to terminal neurodegeneration. Currently, there is no treatment or cure. By developing an AAV gene therapy, the teams aim to delay or prevent the onset of this devastating disease. Viralgen will contribute its expertise in AAV manufacturing, using the advanced Pro10 platform cell line to produce viral capsids—key to delivering the therapeutic genes to the brain.

Sonia Vallabh, co-leader of the prion disease initiative at the Broad Institute, shared the personal motivation behind the project. After losing her mother to the disease, Vallabh discovered she had inherited the same genetic mutation, placing her at risk. She and her husband Eric Minikel transitioned from law and consulting to becoming scientists focused on combating prion disease.

“We’re incredibly lucky to be doing this work at this exact moment in time,” Sonia reflects. “Because prion disease is a whole brain disease, we’ve faced two major challenges from the beginning: finding a molecular machine that can target our gene and delivering that machine to enough neurons in the adult human brain to make a difference. It is just now becoming imaginable that, with the right team, we could piece together answers to these two big questions in our lifetime. We’re so fortunate to have the chance to try.”

“We are honored to collaborate with the Broad Institute on this investigational initiative,” commented Jimmy Vanhove, CEO of Viralgen. “By combining our expertise in gene therapy manufacturing with the Broad Institute’s scientific proficiency, we aim to progressing significantly in the development of advance treatments for prion disease patients.”