Precision Biosciences Inc. has received pre-IND regulatory feedback from the FDA and ex-U.S. agencies providing alignment and clarity on Precision's final IND/CTA-enabling preclinical plans and clinical strategy for PBGENE-HBV.
Although there are different methods of nuclear gene editing, there are still no effective treatments against mitochondrial disorders due to genetic alterations. Now, a group of researchers at Precision Biosciences Inc. and the University of Miami (UM) has developed a genetic edition platform that targets mitochondrial DNA (mtDNA) to delete its mutations.
“The ARCUS technology that we use is based on an enzyme found in nature called I-CreI. It is an enzyme that recognizes a 22 base pair DNA sequence within a species of green algae. And when it finds that DNA sequence, it will generate double-strand breaks,” first author Wendy Shoop, a scientist at Precision Biosciences, told BioWorld.
Iecure Inc. has received clearance from the Australian Therapeutic Goods Administration (TGA) of the company’s clinical trial notification (CTN) for ECUR-506 (formerly GTP-506), an investigational therapy in development for the treatment of ornithine transcarbamylase (OTC) deficiency in pediatric (or neonatal) patients.
Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.
Immuno-oncology company Imugene Ltd. has acquired a worldwide exclusive license to Precision Biosciences Inc.’s allogeneic CD19 CAR T-cell therapy program in a deal worth more than $227 million. The off-the-shelf CD19 CAR T-cell therapy, azercabtagene zapreleucel (azer-cel), could potentially be the first allogeneic CAR T to be approved, Imugene CEO Leslie Chong told BioWorld.
Immuno-oncology company Imugene Ltd. has acquired a worldwide exclusive license to Precision Biosciences Inc.’s allogeneic CD19 CAR T-cell therapy program in a deal worth more than $227 million. The off-the-shelf CD19 CAR T-cell therapy, azercabtagene zapreleucel (azer-cel), could potentially be the first allogeneic CAR T to be approved, Imugene CEO Leslie Chong told BioWorld.
Chronic hepatitis B affects around 250 million people in the world and its cure remains elusive. At the 2023 European Association for the Study of the Liver Congress in Vienna, Austria, Emily Harrison of Precision Biosciences Inc. presented the company’s work on using a naturally occurring endonuclease in the development of its ARCUS gene editing approach to eradicating the persistent viral infection.
As fellow gene editing firm Crispr Therapeutics AG hosted an innovation day in which it confirmed plans for regulatory filings by year-end for an ex vivo gene editing therapy in sickle cell disease and beta-thalassemia, Precision Biosciences Inc. announced plans to develop an in vivo gene editing approach through a collaboration with Novartis AG that brings Precision an initial $75 million with up to $1.4 billion in potential milestones.
A collection of CAR T-cell programs originally licensed by Precision Biosciences Inc. to Baxalta Inc. in a $1.6 billion deal are, after handoffs to Shire and Servier SAS, back where they began. Precision said April 15 it would reacquire all development and commercial rights to the programs, including two clinical-stage CD19-targeting allogeneic CAR T candidates, from Servier for $1.25 million in cash, plus the waiver of earned but as-yet unpaid milestones totaling $18.75 million.