NEW YORK – Tempus said this week that the US Food and Drug Administration has granted the firm's HLA-LOH assay breakthrough device designation as a companion diagnostic test. Leveraging a machine learning model, the test analyzes sequencing data produced by Tempus' xT CDx assay. The HLA-LOH assay is for identifying cancer patients with solid tumors who may benefit from treatment with specific targeted therapies when the tumor has allele-specific loss of heterozygosity for specific human leukocyte antigen Class I alleles, the Chicago-based company said.
Solve GNE this week said it will work with Columbus, Ohio-based biotechnology firm Genosera to develop a novel adeno-associated virus-based gene therapy for GNE myopathy, a muscle weakness disorder that manifests in early adulthood. The rare genetic disease, which can lead to all loss of mobility, occurs in 10 percent to 15 percent of Iranians of Jewish descent who carry at least one copy of the M743T mutation.
Genosera is developing bicistronic AAV treatments that target both genetic loci involved in the pathophysiology of muscle diseases. Solve GNE, a Los Angeles-based nonprofit focused on advancing a cure for GNE myopathy, will fund Genosera's efforts to develop a gene therapy for this condition based on the research of one of the biotech's founders, Paul Martin at the Nationwide Children's Hospital in Ohio. The companies didn't disclose the financial details of the sponsored research agreement.
Bluebird Bio this week said it has received notification from the US Food and Drug Administration that it will not convene an advisory committee meeting for lovotibeglogene autotemcel, a gene therapy for sickle cell disease that the firm is seeking market approval for. Bluebird submitted a biologics license application in April seeking approval for lovo-cel as a one-time treatment for patients at least 12 years old who have sickle cell disease and a history of vaso-occlusive events. The application contains data from a Phase I/II trial, in which 96 percent of 36 patients on lovo-cel experienced resolution of severe vaso-occlusive events, and data on two patients from a Phase III trial, in which both also benefited from the treatment. The FDA accepted the application in June under priority review and is slated to issue a decision by Dec. 20.
The University of Montana Foundation this week said it has received an $8 million grant from the Alsam Foundation, which will fund the academic institution's L.S. Skaggs Institute for Health Innovation (SIHI). The institute was founded three years ago as a place to advance health education, research, and outreach within the state. SIHI aims to use the university's pharmacy and rural health expertise to address health disparities in Montana and expand access to the latest medical innovations. The institute also has research programs in precision medicine and pharmacogenomics, including training opportunities for pharmacy and graduate students in these disciplines.
2seventy Bio said this week that the US Food and Drug Administration has placed a clinical hold on the firm's Phase I CAR T-cell therapy trial, PLAT-08. The study, in which 2seventy was evaluating its autologous cell therapy SC-DARIC33, was voluntarily paused in June after a patient died on treatment at Seattle Children's hospital. 2seventy says it is working with Seattle Children's to investigate the root cause of the patient death and aims to amend the trial protocol so as to resume the study as soon as possible.
Avidity Biosciences this week said the US Food and Drug Administration has granted orphan drug designation to AOC 1044, an antibody oligonucleotide conjugate that it is developing for patients with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). Drugs for rare diseases with orphan designation have market exclusivity upon regulatory approval, and the sponsor is exempt from FDA application fees and eligible for tax credits for qualified clinical trials.
Avidity is studying AOC 1044 in the Phase I/II EXPLORE44 trial. The San Diego-based firm expects to share results from the healthy volunteer portion of the study in Q4 2023 and is currently enrolling patients living with DMD44 into the study. The FDA in April also granted AOC 1044 fast-track designation.
Nuvectis Pharma this week said the US Food and Drug Administration has granted orphan drug designation to NXP800 as a potential cholangiocarcinoma treatment. In preclinical studies, the HSF1 inhibitor has shown activity in ovarian, endometrial, gastric, and bile duct cancer models bearing ARID1a mutations. The Fort Lee, New Jersey-based firm is already conducting a Phase Ib trial of the drug in patients with platinum-resistant ARID1a-mutated ovarian cancer and a Phase I trial in patients with a range of ARID1a-mutated advanced solid tumors, including ovarian clear cell and endometrial cancers.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.