Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is an important factor from a complex downstream of Bruton tyrosine kinase (BTK) in the B-cell receptor (BCR) signaling pathway.
CAR T-cell therapy for B-cell malignancies has still to be improved regarding durability, manufacturing complexity or toxicity, among others. Precigen Inc. has presented data regarding the preclinical development and efficacy of PRGN-3008, a PD-1-expression inhibitor cell therapy targeting CD19 that was built in a next-generation ultra CAR T platform.
Minerva Biotechnologies Corp. recently released details on their work to develop a treatment for HER2-positive patients who have acquired resistance to Herceptin (trastuzumab) or Enhertu (trastuzumab-deruxtecan).
At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.
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.
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.
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).
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.
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