NEW YORK – Qiagen said this week that Genomics England has selected its Clinical Knowledge Base for the Generation Study, a project that aims to sequence the genomes of 100,000 newborns in England to screen for more than 200 rare, treatable diseases. Qiagen's Clinical Knowledge Base, which contains evidence for genetic variants from more than 40 databases that has been curated for clinical relevance, will be used by the study to help interpret variants and report results.
Health and research company Care Access this week offered free health screenings in Chicago; College Park, Georgia; Aurora, Colorado; and Memphis, Tennessee to identify heart and kidney health risks for patients. The free health screening included testing for lipoprotein(a), a cardiovascular risk factor, as well as tests for other metabolism and kidney risks. The effort is part of the organization's Future of Medicine program, in which it provides free testing and health education and connects participants with clinical research opportunities.
Los Angeles-based Immix Biopharma this week said it will accelerate enrollment in its US clinical trial of NXC-201, an autologous CAR T-cell therapy for relapsed or refractory amyloid light-chain (AL) amyloidosis, having completed a six-patient Phase Ib safety run-in segment at two different dose levels. The company plans to advance into a 34-patient dose expansion segment of the open-label study, dubbed NEXICART-2, in which patients will receive the higher dose level of NXC-201.
Durham, North Carolina-based Atsena Therapeutics this week said it has started Part B of its open-label, dose-escalation, and dose-expansion Phase I/II LIGHTHOUSE trial, in which it is testing ATSN-201 as a gene therapy for X-linked retinoschisis. In Part A, the company evaluated the safety and tolerability of three doses of the gene therapy to establish a dose for the next part of the study. In Part B, nine additional adults will receive the gene therapy at one of two volumes, followed by three children after reviewing preliminary data from the adult cohort.
San Diego-based Aspen Neuroscience this week said it has advanced its plans for automating production of ANPD001, an autologous neuronal cell replacement therapy that it's developing for moderate-to-severe Parkinson's disease and that is currently being evaluated in the Phase I/IIa ASPIRO trial. That includes expanding the capacity for such manufacturing at the company's good manufacturing practice (GMP) facility and entering into a collaboration with London-based Mytos, which automates cell manufacturing, to work on automating the differentiation of dopaminergic neuronal precursor cells from induced pluripotent stem cells that are derived from a sample of a patient's own skin cells.
Adaptive Biotechnologies said this week that the new Medicare Clinical Laboratory Fee Schedule (CLFS) rate for its next-generation sequencing-based ClonoSeq test for minimal residual diseases (MRD) is now in effect. The CLFS rate for ClonoSeq was set at $2,007, consistent with the final gap-fill rate recommendation for the test, the company said.
Managed by the Centers for Medicare and Medicaid Services (CMS), ClonoSeq's CLFS rate, which was finalized after a year of evaluation, may become a benchmark for other payors in the US for establishing their rate schedules for ClonoSeq, according to Adaptive.
Dana-Farber Cancer Institute this week launched the Center for RAS Therapeutics to advance investigation, translational research, and clinical care approaches for patients with RAS-driven cancers. The center will support preclinical studies and clinical trials of RAS-directed cancer therapies and coordinate research across Dana-Farber and its pharma and academic partners to accelerate development of these treatments. The center will be codirected by Alice Shaw, chief of strategic partnerships at Dana-Farber, and Andrew Aguirre, gastrointestinal medical oncologist and physician-scientist at Dana-Farber Cancer Institute.
The US Food and Drug Administration this week granted fast-track designation to Zentalis Pharmaceuticals' WEE1 inhibitor azenosertib for the treatment of patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who are cyclin E1-positive. Azenosertib is currently being studied in this population in a Phase II trial. With fast-track designation, a sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Astellas Pharma this week said Health Canada approved the claudin 18.2 (CLDN18.2)-targeted monoclonal antibody Vyloy (zolbetuximab) with fluoropyrimidine and platinum-containing chemotherapy as a first-line treatment for advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma tumors that test positive for CLDN18.2 by a validated test. Canada's Drug Agency also issued a draft recommendation to reimburse when certain conditions are met including that 75 percent or more of tumor cells have moderate to strong CLDN18.2 immunohistochemical staining. China's National Medical Products Administration also approved Vyloy with chemotherapy for patients with advanced unresectable or metastatic CLDN18.2-positive, HER2-negativ gastric or gastroesophageal junction adenocarcinoma.
The US Food and Drug Administration this week granted fast-track designation to Marengo Therapeutics' dual T-cell agonist, invikafusp alfa (STAR0602), in patients with advanced colorectal cancer with high tumor mutational burden. The agency granted fast-track status, which allows sponsors to meet with the FDA more frequently for advice and submit data on a rolling basis, based on the results of the Phase I/II START-001 trial. Fast-track designation also lets sponsors apply for accelerated approval or priority review.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.