Michael Severino on In Vivo Gene Editing With RNA Gene Writers

Commentary
Video

The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.

“Our programs are based on a platform that we call our RNA gene writers. So, our RNA gene writers modify DNA, but they do it using all RNA constructs. So, the protein that does the work is delivered as an mRNA, and the template which contains the instructions is an RNA, and that enables a number of advantages.”

Tessera Therapeutics is aiming to develop effective and safe in vivo, nonviral gene and cell therapies, which could eliminate issues inherent with current modes of approved gene therapies such as ex vivo chimeric antigen receptor (CAR) T-cell therapies and adeno-associated virus (AAV) therapies. The company presented on a number of these programs at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.

CGTLive spoke with Michael Severino, chief executive officer, Tessera, while at ASGCT to learn more about the company’s RNA gene writing platform and the promising advantages it might offer for a variety of different fields and indications. He overviewed research Tessera is conducting into using its platform for sickle cell disease and for in vivo generation of CAR T-cell therapies for oncologic indications. He also shared validation data on the platform in mouse models that were presented at the meeting.

REFERENCES
1. Palchaudhuri R, Gupta M, Monte MT, et al. In Vivo RNA Delivery to T Cells and Hematopoietic Stem Cells in Humanized Mice and Non-Human Primates Using Targeting Lipid Nanoparticles. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #132
2. Stein KT, Rodriguez J, Johnson L, et al. A Same-Day Manufacturing Platform Leveraging an RNA-Based Lipid Nanoparticle (LNP) Gene Writer System to Generate Chimeric Antigen Receptor (CAR) T Cells. ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #379
3. Schiroli G, Heshmati Y, Cao Y, et al. In Vivo HSC Gene Editing for Correction of Sickle Cell Mutation Using RNA Gene Writers. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #280
Recent Videos
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
David Barrett, JD, the chief executive officer of ASGCT
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
David Barrett, JD, the chief executive officer of ASGCT
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia
Related Content
© 2024 MJH Life Sciences

All rights reserved.