The director of the Mass General Brigham Gene and Cell Therapy Institute discussed the history and future of gene therapy in cardiology at the American Heart Association’s Scientific Sessions 2023.
“We've seen in [noncardiac gene therapy] trials that there's been a lot of adverse events based on the massive doses that have to be given with adeno-associated virus vectors. With the emergence of novel, more cardiotropic vectors and minimally-invasive techniques to deliver locally, we are now able to deliver much lower amounts of vector—100 times less. That will basically allow us to have a safer vector when we deliver these next generation capsids.”
Despite the great strides that have been made in recent years with small-molecule drugs and medical devices, significant unmet need remains in the field of cardiology, and cases of cardiac disease continue to rise both in the United States and in the European Union. In order to address this concern, some investigators, companies, and research institutions are now turning increased attention towards gene therapy as a promising new modality for treating cardiac diseases.
With this in mind, Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, gave a talk on the importance of gene therapy in cardiology at the American Heart Association’s (AHA) Scientific Sessions 2023, held November 10-13 in Philadelphia, Pennsylvania. The talk, entitled “The Quest to Overcome the Challenges in Cardiac Gene Therapy” covered the surprisingly long history of gene therapy clinical trials for cardiovascular indications and the future directions efforts to bring this modality to patients are likely to take.
In an interview with CGTLive™ at the conference, Hajjar gave an overview of the key points he discussed during his talk. He noted that the history of gene therapy trials in cardiology stretches back over 2 decades, with initial efforts having used plasmid vectors in an attempt to induce angiogenesis for the treatment of ischemic heart disease. Hajjar also spoke on more recent gene therapy efforts that have used adeno-associated virus vectors and the need to continue to pioneer new vector types and delivery methods in order to improve specificity and safety.