Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them. Read More
Microsatellite instability (MSI), which results from defects in the DNA mismatch repair system, is an important biomarker in colorectal cancer. While the MSI-high (MSI-H) status predicts response to pembrolizumab, a commonly used immune checkpoint inhibitor in metastatic colorectal cancer, a subset of MSI-H patients still does not respond to this treatment. Read More
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome. Read More
Werner syndrome ATP-dependent helicase (WRN) is an enzyme involved in DNA replication and repair and has been identified as a synthetic lethality target in tumors with high microsatellite instability (MSI-H). Read More
Oncolytic viral therapy offers new avenues in anticancer treatment, and two herpes simplex virus (HSV)-1-based virotherapies have recently gained approval for the treatment of advanced melanoma and recurrent glioblastoma. Read More
Starg (Wuhan) Pharmaceutical Technology Co. Ltd. has discovered compounds acting as diacylglycerol kinase ζ (DGK-ζ) inhibitors reported to be useful for the treatment of cancer. Read More
DNA damage repair enzymes are interesting targets in cancer, since genomic instability and DNA repair defects are important cancer cell hallmarks. Poly (ADP-ribose) glycohydrolase (PARG) is the dominant eliminator of PARylation in the cell, the activity of which prevents excessive accumulation of PARylation, and promotes the dissociation of repair proteins, as well as ensuring the smooth completion of DNA repair process. Read More
Gen1e Lifesciences Inc. has disclosed drugs targeting extracellular-regulated kinase (ERK) reported to be useful for the treatment of cancer, inflammatory disorders, autoimmune and lung diseases. Read More
ADC Therapeutics SA has described antibody-drug conjugates comprising antibody targeting sodium-dependent phosphate transport protein 2B (SLC34A2; NaPi-2b) covalently linked to exatecan through a linker reported to be useful for the treatment of ovarian cancer and non-small-cell lung cancer (NSCLC). Read More
Acute myeloid leukemia (AML) harboring KMT2A rearrangements (KMT2A-r) represents a highly aggressive disease subtype, characterized by poor therapeutic response and a high risk of relapse. Read More
Halda Therapeutics LLC has synthesized heterobifunctional compounds able to bind both androgen receptor and bromodomain-containing protein 4 (BRD4; HUNK1) reported to be useful for the treatment of cancer. Read More
Hepatocellular carcinoma (HCC), which accounts for up to 80% of cases of primary liver cancer, is typically diagnosed in an advanced stage, meaning a poor prognosis. Understanding what drives progression may help identify proteins and pathways that can be targeted to slow down the disease. Read More
Pillai Universal LLC has prepared and tested compounds acting as protein kinase inhibitors reported to be useful for the treatment of cancer. Read More
Additional early-stage research and drug discovery news in brief, from: AB Science, Capsida Biotherapeutics, Channel Therapeutics, Excision Biotherapeutics, In8bio, Precision Biosciences. Read More
