In late March, I participated in several discussions at the 6th Annual Gene Therapy for Rare Disorders (GTRD) Summit about how we continue to further gene therapy development. In 2022 we saw the most gene therapies approved in the U.S. and Europe in one year – five. There are hundreds of active trials for new […]
Author Archives: Kate Marshall
Gene Therapy Remains On Course To Broadly Transform Medicine
When we founded AVROBIO seven years ago, we asked ourselves the question – What if one gene can change your entire world? Every year that question reinvigorates our resolve to develop an answer: a single infusion gene therapy with the potential to alter the downward trajectory of certain genetic diseases. This year, as we review achievements […]
Scaling the slope of enlightenment: Gene therapy progress and the future of healthcare
The gene therapy industry is undergoing a regulatory resurgence. After three years with no new therapies reaching patients in the United States, two approvals in the last two months – of hematopoietic stem cell (HSC) gene therapies for beta thalassemia and cerebral adrenoleukodystrophy – have doubled the number of gene therapies available here. Across the […]
Gene Therapy Is Here To Stay: Should Bigger Companies Build, Buy, Or Partner?
Gene therapy’s tremendous potential to transform the treatment of both rare and common diseases has been understood for decades. But few may appreciate how imminently that revolution is upon us. This summer, approvals on both sides of the Atlantic – for beta thalassemia and cerebral adrenoleukodystrophy in the United States and for severe hemophilia A […]
Combining assays to evaluate genotoxicity risk of integrating vectors
To continue evolving gene therapy, many in our industry are working to characterize and better understand safety concerns. Data on research attempting to do just this, and supported by AVROBIO (my employer), was presented at the European Society of Gene and Cell Therapy (ESGCT) in Scotland on Oct. 14, 2022, by my esteemed colleague Holly […]
Gene Therapy Can Take Us Far If We Seize Its Long-Term Possibilities
Last month marked another historic moment for gene therapy. For the first time in five years, the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee met to consider a new product application. Two, in fact. With a pair of gene therapies to review from the Cambridge, MA company bluebird bio, the […]
