Weekly Quick Hits (Research Triangle Park) – Week of April 17, 2023

By Alex Keown, Mark Terry, and Sarah Ellinwood
April 21, 2023

In the Clinic

Renovion, Inc. Enrolls First Patient in Phase III Lung Transplant Trial

Chapel Hill-based Renovion, Inc. enrolled the first patient in a Phase III study evaluating ARINA-1, an investigational nebulized therapy to prevent the progression of bronchiolitis obliterans syndrome (BOS) progression in individuals with a bilateral lung transplant. The study is expected to enroll approximately 100 participants across 15 sites in the U.S. There are currently no FDA-approved therapies to treat patients with this condition. BOS is the most common form of chronic lung allograft dysfunction following a lung transplant. It is the leading cause of graft failure and mortality in lung transplant patients. 

Chimerix Presents Multiple Imipridone Datasets at AACR

Chimerix shared multiple datasets related to its imipridone platform’s potential that included preclinical data supporting ONC201’s mechanism of action as an anti-cancer agent, as well as in-human clinical data that demonstrate its clinical efficacy. Data demonstrated that H3 K27M-mutant glioma patients treated with ONC201 experienced a statistically significant reversal of the H3 K27 trimethyl-loss epigenetic signature throughout their tumor compared to patients who did not receive ONC201. Concordant findings were also reported in preclinical models that were mechanistically linked to specific metabolic changes induced in tumors by ONC201. The ongoing ACTION trial, a global Phase III trial of ONC201 in newly diagnosed H3 K27M-mutant diffuse glioma, was also highlighted. 

New Products

FirstHealth of the Carolinas to Offer GRAIL’s Early Cancer Detection Blood Test

FirstHealth of the Carolinas partnered with GRAIL, LLC to offer Galleri, a multi-cancer early detection screening test. FirstHealth will be the first health system in North Carolina to offer the Galleri MCED test to those at an elevated risk of cancer, including those aged 50 or older, as part of its comprehensive cancer screening and care. In a clinical study, the Galleri test demonstrated the ability to detect a shared cancer signal across more than 50 types of cancer, as defined by the American Joint Committee on Cancer Staging Manual.

New Patents

Precision BioSciences Receives Patent Allowances for PCSK9 ARCUS Nuclease

Precision BioSciences, Inc. received a Notice of Allowance from the U.S. Patent and Trademark Office for a patent titled “Engineered meganucleases specific for recognition sequences in the PCSK9 gene.”  The allowed composition of matter claims encompasses a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has been used preclinically for both gene deletion approaches for cardiovascular diseases such as familial hypercholesterolemia and a viable and safe site to insert a functional copy of a specific gene to restore function addressing other diseases requiring gene addition.

Research Roundup

Humacyte Shares Preclinical Human Acellular Vessel Data

Durham-based Humacyte, Inc. published data from a preclinical study in the Journal of Trauma and Acute Care Surgery comparing the use of its Human Acellular Vessel (HAV) to expanded polytetrafluorethylene (ePTFE) grafts for vascular repair following arterial trauma. In the preclinical study the HAV performed better than ePTFE on multiple indices, the data showed. The data in this preclinical study indicate that the HAV performed better than ePTFE on multiple indices, including recovery of limb function after six hours of ischemia and conduit patency. In addition, the HAV showed no incidence of infection, degradation, aneurysm or mechanical failure. Host recellularization of the HAV conduits was observed to be greater than that of ePTFE grafts.

UNC Researchers boost new neurons, restore memory in Alzheimer’s disease model

New research conducted by a team from the University of North Carolina School of Medicine revealed that stimulating a region of the brain called Supramammilary nucleus (SuM), which is located in the hypothalamus, enhanced adult-born neurons in Alzheimers impaired brains of mice. The stimulation generated more adult-born neurons which led to the restoration of cognitive and affective deficits in AD mouse models. The data was published in the journal Cell Stem Cell.   

Precision BioSciences to Share Preclinical In Vivo Gene Editing Research Data

Precision BioSciences, Inc. will share data from its BGENE-DMD program for the potential treatment of Duchenne muscular dystrophy at the American Society of Gene & Cell Therapy meeting in May. Precision is developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV in order to make a variant of the dystrophin protein that is functionally competent, said Jeff Smith, chief research officer at Precision BioSciences. 

Additionally, Precision BioSciences intends to present preclinical data from its PBGENE-HBV program at the Global Hepatitis Summit 2023 in Paris. A gene therapy, BGENE-HBV is designed to potentially eliminate hepatitis B virus (HBV) by targeting viral covalently closed circular DNA (cccDNA) in vivo.

People on the Move

Premier Research Fortifies Cell and Gene Therapy Leadership Team

Morrisville-based Premier Research expanded its cell and gene therapy leadership team with the addition of Kenneth Ndugga-Kabuye, vice president of cell & gene therapy. Ndugga-Kabuye is focused on gene therapies, RNA therapies, synthetic biotics and enzyme replacement therapies. He is an ABMGG board-certified clinical geneticist and holds medical licenses in California and Washington. Previously, he was Medical Director, Metabolic Diseases at a biotech focused on the development of novel biotherapeutics.

Science 37 Adds New Head of Quality, Irena Lambridis

Science 37 Holdings announced the appointment of Irena Lambridis as its Global Head of Quality Assurance & Compliance. She will be responsible for overseeing quality and compliance across all functions, including the company’s newly announced global Centers of Excellence in India, Pakistan, and Slovakia. Lambridis previously held senior leadership positions at KCR and Novella Clinical, now known as IQVIA Biotech. 

Parexel Appoints Scott Smith as New Head of Hematology

Clinical Research Organization Parexel named Scott Smith as Senior Vice President, Global Therapeutic Area Head – Hematology. In this role, Smith will lead Parexel’s Hematology therapeutic area and global team in the development of new treatments for malignant hematology diseases as well as in the development of new therapies to treat non-malignant hematology diseases. Prior to joining Parexel, Scott held several positions of increasing responsibility at Loyola Medicine’s Cardinal Bernardin Cancer Center, including Director, Cancer Clinical Trials Office; Director, Bone Marrow Transplant Program; Section Chief of Hematology; and Associate Director, Clinical Research.

Areteia Therapeutics Adds Elyse Stock to Board of Directors

Chapel Hill-based Areteia Therapeutics appointed Elyse Stock to the company board of directors. She will serve as a member of the compensation, nominating and corporate governance committee of the board.Stock spent the last six years at Biohaven Pharmaceuticals where she was the CMO responsible for the development of numerous investigational agents including Nurtec. Prior to Biohaven, she spent 19 years at Bristol-Myers Squibb developing numerous experimental agents across multiple therapeutic areas including neuroscience, oncology, immunology and cardiovascular.