NEW YORK – ReCode Therapeutics this week said it has received $15 million from the Cystic Fibrosis Foundation, which it will use to develop and commercialize new gene correcting therapies for cystic fibrosis. Specifically, ReCode will put the funds toward advancing a gene correction research program with Intellia Therapeutics, in which the companies are aiming to develop treatments that correct the underlying genetic cause of this disease by using Intellia's CRISPR-based gene-editing platform and ReCode's selective organ-targeting lipid nanoparticle delivery platform. ReCode will also use the funds to advance up to three gene correction therapies, initially focusing on treatments targeting cystic fibrosis caused by CFTR mutations that are not the focus of existing treatments. Finally, the funds will also support the development of RCT2100, ReCode's investigational inhaled mRNA cystic fibrosis therapy, currently undergoing testing in a Phase Ib trial that recently began dosing patients. The CF Foundation last year invested $15 million as part of a Series B financing round, in which ReCode raised $210 million in support of the mRNA therapy development program.
Anixa Biosciences this week said it dosed the first patient of a third cohort of study participants in a Phase I trial of its FSHR-mediated CAR T-cell therapy for recurrent ovarian cancer who have progressed on at least two prior therapies. Patients in this cohort will receive a dose 10 times higher than the starting dose. Based on results from the first cohort, in which one patient showed mild improvement for over a year after the first infusion, Anixa has submitted an amendment to the trial protocol to allow a second dose in patients who show signs that they may benefit.
The German Research Foundation (DFG) is funding a new research training group at the University of Cologne that is focusing on tumor heterogeneity and genomic instability in lung carcinoma with €5.3 million ($5.6 million) over five years. Funding will start April 1, 2025, and support doctoral students in a four-year research program. The research training group announced this week will rely on expertise from the Center for Molecular Medicine at the University of Cologne (ZMMK), the Molecular Pathology Department of Cologne University Hospital, and the Samsung Advanced Institute for Health Sciences & Technology (SAIHST) at Sungkyunkwan University in Seoul, South Korea. The researchers plan to focus on resistance mechanisms, developing clinically relevant models for predicting resistance and clonal evolution and developing preclinical models for testing combination therapies in lung tumors with high-risk genomes.
Eisai said the FGFR selective tyrosine kinase inhibitor Tasfygo (tasurgratinib) was published in Japan's National Health Insurance drug price list this week after receiving manufacturing and marketing approval for treatment of unresectable biliary tract cancer with FGFR2 gene fusions or rearrangements in September. Japan's Pharmaceuticals and Medical Devices Agency also approved a companion diagnostic test to detect FGFR2 gene fusions or rearrangements for use with Tasfygo in biliary tract cancer in August.
The European Medicines Association earlier this month granted orphan drug designation to KYV-101, an autologous CAR T-cell therapy developed by Emeryville, California-based Kyverna Therapeutics for treatment of myasthenia gravis. Kyverna in the third quarter initiated dosing in its Phase II KYSA-6 trial evaluating the cell therapy in this indication. The company is studying KYV-101 in multiple other autoimmune conditions, as well, including multiple sclerosis, systemic sclerosis, and lupus nephritis.
Mindera Health this week announced a partnership with patient advocacy organization National Psoriasis Foundation to raise awareness of personalized medicine within psoriasis and accelerate adoption of its transcriptomics test, Mind.Px. The test is designed to predict which class of biologic a patient is likely to respond to, based on an analysis of mRNA. The National Psoriasis Foundation provides patients with resources, tools, and educational information to manage psoriasis.
Nouscom said this week that it has completed enrollment in a colorectal cancer cohort of its ongoing Phase II trial of its neoantigen-targeted cancer vaccine, NOUS-209. The 69-patient cohort includes patients with microsatellite instability (MSI)-high, DNA mismatch repair deficient (dMMR) metastatic or unresectable colorectal cancer patients. Nouscom is evaluating NOUS-209 combined with Merck's checkpoint inhibitor Keytruda (pembrolizumab).
Biotech firm NeuroKaire said this week that it has received Clinical Laboratory Improvement Amendments certification from the US Centers for Medicare and Medicaid Services to perform high-complexity clinical testing. The company said that its first offering is a pharmacogenetic assay for 95 variants in 24 genes to guide treatment with 132 drugs for various psychiatric and neurological conditions.
The Parker Institute for Cancer Immunotherapy said this week that it has awarded a total of $525,000 in grants to scholars whose work involves developing cancer immunotherapies, including CAR T-cell therapies, investigating immunotherapy resistance tumor microenvironments, and advancing T-cell engineering. The seven scholars, who comprise the institute's inaugural class of Representation In SciencE (RISE) recipients, will each receive $75,000 for their projects.
Precision for Medicine this week announced a strategic partnership with Agena Bioscience. Under the collaboration, the two organizations have profiled challenging lung tumor samples provided by Precision for Medicine using Agena's Iplex HS Lung Panel on the MassArray platform. They presented the results at the Association for Molecular Pathology annual meeting in Vancouver, Canada, this week.
China's National Medical Products Administration this week approved Merck's HIF-2α inhibitor Welireg (belzutifan) for patients with von-Hippel-Lindau (VHL)-associated renal cell carcinoma, pancreatic neuroendocrine tumors, or central nervous system hemangioblastomas. The US Food and Drug Association approved Welireg for this patient population in 2021.
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.