Tessera’s gene editing offers hope for sickle cell patients
Posted: 18 December 2024 | Drug Target Review | No comments yet
Find out how Tessera’s latest advancements in gene editing bring promising solutions to treat sickle cell disease.
Tessera Therapeutics has made significant progress in its in vivo gene-editing technology, which was showcased at the 66th American Society of Hematology (ASH) Annual Meeting. The company shared preclinical data on their Gene Writing™ technology, which uses lipid nanoparticles (LNPs) to deliver therapeutic gene edits directly to patients. This method avoids the need for toxic chemotherapy conditioning or lymphodepletion, addressing longstanding challenges in cellular therapies.
Progress in Sickle Cell Disease (SCD) treatments
The early proof-of-concept data showed successful in vivo editing of the HBB gene, which is central to sickle cell disease. In non-human primates (NHPs), a single intravenous dose of the Gene Writer formulation achieved an average of 24 percent gene rewriting in long-term hematopoietic stem cells (LT-HSCs). These levels meet therapeutic thresholds established by clinical research, suggesting the potential to reverse the sickle cell phenotype.
Additionally, the company shared its longest-term proof-of-principle data to date, with rewriting efficiency reaching 76 percent in LT-HSCs of NHPs and stable editing observed over 84 days. The edited stem cells successfully contributed to multi-lineage blood cell production, demonstrating their functionality. Tessera’s delivery system also achieved a 52-fold higher concentration in bone marrow and an 11-fold lower concentration in the liver, enhancing therapeutic precision.
“In our sickle cell disease program, we have shown in vivo editing of HBB in mouse models that surpass that of asymptomatic carriers of the sickle mutation, and for the first time in NHP, editing efficiencies that reach anticipated therapeutic levels,” said Dr Michael Severino, CEO of Tessera Therapeutics.
Innovations in CAR-T cell therapies
Tessera also presented data on in vivo chimeric antigen receptor T-cell (CAR-T) therapies. The company demonstrated that a single dose of its LNP-RNA composition could generate functional CAR T cells in tumour-bearing xenograft mouse models. These CAR T cells expanded within the body and eradicated tumour burdens, all without preconditioning regimens such as lymphodepletion.
Looking ahead
Tessera’s presentations at ASH highlight the versatility of its Gene Writing platform across multiple therapeutic areas. By avoiding the toxic preconditioning typically associated with traditional gene editing and cell therapies, Tessera aims to broaden access to life-changing treatments for patients worldwide.
To learn more about Tessera Therapeutics, visit www.tesseratherapeutics.com
Related topics
Cell Therapy, Clinical Trials, Drug Discovery, Drug Discovery Processes, Gene Therapy, Genome Editing, Immunotherapy, In Vivo
Related conditions
Cancer, Sickle cell disease (SCD)
Related organisations
American Society of Hematology (ASH), Tessera Therapeutics
Related people
Dr Michael Severino
