Precision BioSciences, Inc. is a clinical-stage biotechnology firm based in the United States, specializing in the creation of both in vivo gene editing solutions and ex vivo allogeneic CAR T-cell therapies. At its core is ARCUS, a proprietary genome editing platform designed to address and potentially cure genetic diseases. The company's portfolio also encompasses Ex vivo Allogeneic CAR T Immunotherapy, an innovative approach where specific immune cells, known as T-cells, are genetically modified outside the body to precisely identify and eliminate cancer cells. Among its prominent therapeutic candidates are: PBCAR0191, currently undergoing Phase 1/2a clinical trials for adult patients battling relapsed/refractory (R/R) non-Hodgkin lymphoma or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). PBCAR19B, an anti-CD19 CAR T candidate leveraging a "stealth cell" platform through a single-step gene edit, engineered to reduce the likelihood of chromosomal abnormalities. PBCAR269A, an investigational allogeneic CAR T immunotherapy formulated to target BCMA, intended for the treatment of R/R multiple myeloma. Precision BioSciences has strategically formed several collaborative alliances. These include an agreement with Les Laboratoires Servier for the development and commercialization of allogeneic chimeric antigen receptor T-cell therapies, aimed at various antigen targets, including hematological cancers beyond CD19, and solid tumors. The company is also partnering with Tiziana Life Sciences to assess foralumab, a fully human anti-CD3 monoclonal antibody, for its utility as a lymphodepleting agent in potential cancer treatments. Furthermore, a collaboration with iECURE, Inc. focuses on advancing ARCUS-based gene editing therapies. Established in 2006, Precision BioSciences, Inc. maintains its corporate headquarters in Durham, North Carolina.
iECURE to Present Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal-Onset Ornithine Transcarbamylase Deficiency at SSIEM Annual Symposium
iECURE, a licensee of Precision BioSciences' ARCUS technology, announced that clinical data from the ongoing OTC-HOPE trial of ECUR-506 will be presented at the SSIEM Annual Symposium in late August.
The oral presentation will include updated safety observations and genome editing data on metabolic control measures from the trial's low-dose cohort.
ECUR-506 is a first-in-human gene insertion therapy designed to treat neonatal-onset ornithine transcarbamylase (OTC) deficiency by inserting a functional OTC gene.