A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease. Read More

There are real world demonstrations of how autonomous artificial intelligence agents are poised to disrupt biomedical research, according to two papers published May 19 in Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data. Read More

Mekanistic Therapeutics Inc. has obtained IND clearance from the FDA for MTX-531, the company’s lead oncology candidate. A phase I study will be conducted in patients with advanced solid tumors characterized by dysregulated EGFR and/or PI3K signaling, including head and neck and endometrial cancers. Dosing is expected to begin in the third quarter. Read More

Re-Aim Therapeutics Ltd. has launched with £7 million (US$9.4 million) in seed investment backing and a focus on developing functional cures for T-cell-mediated autoimmune diseases. Read More

Genescience Pharmaceutical Co. Ltd. has presented data on a new STAT6 PROTAC degrader – GenSciP166 – which selectively targets STAT6 for proteasomal degradation. GenSciP166 was assayed in vitro as well as in vivo in the MC903 atopic dermatitis murine model. Read More

Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients. Read More

Zhejiang Normal University has patented new inhibitors of proto-oncogene tyrosine-protein kinase receptor Ret (RET; CDHF12; PTC) and its mutants potentially useful for the treatment of cancer. Read More

Matter Bio has submitted its first IND application to the FDA for Lm-LLO-TT, the company’s lead therapeutic candidate, seeking to initiate a first-in-human phase I/IIa trial in patients with pancreatic ductal adenocarcinoma (PDAC). Read More

F. Hoffmann-la Roche Ltd. and Hoffmann-La Roche Inc. have identified new triggering receptor expressed on myeloid cells 2 (TREM2) agonists potentially useful for the treatment of rheumatoid arthritis, amyotrophic lateral sclerosis, frontotemporal dementia, multiple sclerosis, prion disease, stroke, Parkinson’s and Alzheimer’s disease. Read More

Inxmed (Nanjing) Co. Ltd. has discovered new focal adhesion kinase (FAK) and focal adhesion kinase 2 (FADK2; PTK2B; PYK2) dual inhibitors potentially useful for the treatment of cancer. Read More

Etherna Immunotherapies NV has reported a milestone in its ongoing partnership with Dropshot Therapeutics Inc. Based on Etherna’s nucleic acid and lipid nanoparticle (LNP) capabilities, Dropshot has elected to advance one of its selected targets in renal disease to the clinical stage. Read More

Beijing Tide Pharmaceutical Co. Ltd. has divulged new salts of known angiotensin AT2 recpetor antagonists potentially useful for the treatment of allergy, autoimmune and demyelinating diseases, cerebrovascular, respiratory, cognitive, gastrointestinal and inflammatory disorders, among others. Read More

Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Yantai New Drug Creation Shandong Laboratory have reported new degrader-antibody-drug conjugates (DACs) potentially useful for the treatment of cancer which are comprised of an antibody covalently bound to a molecular glue degrader moiety. Read More

Maxwell Biosciences Inc. has reported findings from a study showing that its broad-spectrum small molecules, named Claromers, are able to destroy Epstein-Barr virus. Claromers destroy pathogenic bacteria, viruses, fungi and biofilms, without harming healthy cells or the microbiome. Read More