Michael Severino on In Vivo Gene Editing With RNA Gene Writers

“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