Liver damage arrests growth mediated by the somatotroph axis, which prevents liver cell death and inflammation, but increases fibrosis in nonalcoholic fatty liver disease (NAFLD). The explanation for this effect could lie in the relationship between the activating transcription factor 3 (ATF-3) and insulin-like growth factor 1 (IGF-1), according to a study from the University of California at Berkeley. Read More

Unlike amphibians, mammals do not regenerate appendages. Except when they do. “If you amputate one of the branches off of the antler [of a reindeer], it will also regenerate,” Jeff Biernaskie told BioWorld. Even without amputation, the antlers of both male and female reindeer regenerate annually, including their skin. That regeneration is “the only large mammal model of true skin regeneration,” he said. Read More

Treatment with anti-CD19 bispecific T-cell engager and CAR T therapies can lead to T-cell exhaustion and treatment failure. Novartis AG’s first-in-class anti-CD19, anti-CD3 and anti-CD2 IgG-like trispecific antibody PIT-565, which engages CD19+ on tumor cells, and CD3 (TCR signaling component) and CD2 (a costimulatory receptor) on T cells simultaneously to redirect T-cell cytotoxicity toward CD19-positive malignant B cells, has been designed to avoid T-cell exhaustion. Read More

Vega Therapeutics Inc. has reported promising preclinical data on VGA-039, a first-in-class monoclonal antibody directed against human protein S (ProS) that inhibits ProS cofactor activity for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), thus enhancing thrombin generation by acting on both the initiation (TFPIα) and propagation (aPC) phases of coagulation for potential activity against various bleeding disorders. Read More

Arthex Biotech SA has achieved key regulatory milestones in its program to develop ATX-01 in myotonic dystrophy type 1 (DM1). Having held a pre-IND meeting with the FDA last year and received scientific advice from the EMA, Arthex plans to file an IND application in the U.S. and a clinical trial application (CTA) in Europe next year. Read More

Erasca Inc. has received FDA clearance of its IND application for ERAS-3490, an orally available small-molecule KRAS G12C inhibitor designed to have high central nervous system (CNS) penetration for the treatment of KRAS G12C-mutated solid tumors, including non-small-cell lung cancer (NSCLC). Read More

Researchers from Salarius Pharmaceuticals Inc. have presented preclinical data on SP-3164, a novel cereblon (CRBN)-binding protein degrader intended for the treatment of lymphoma. SP-3164 bound to cereblon and consequently induced the degradation of hematological transcription factors Ikaros and Aiolos. The aim of their studies was to investigate the drug’s antitumor efficacy in preclinical models of diffuse large B-cell lymphoma (DLBCL). Read More

Vincerx Pharma Inc. has received FDA clearance of its IND application for VIP-236, a small molecule-drug conjugate (SMDC) for the treatment of advanced solid tumors. A first-in-human study for advanced or metastatic solid tumors is expected to start in the first quarter of next year. Read More

Researchers from Tubulis GmbH presented preclinical data for a CD30-targeting antibody-drug conjugate (ADC), TUB-010, being developed as a potential anticancer immunotherapy candidate. Read More

Jing Medicine Technology (Shanghai) Ltd. has patented proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to ALK tyrosine kinase receptor- and/or epidermal growth factor receptor (EGFR)- and/or proto-oncogene tyrosine-protein kinase ROS (ROS1)-targeting moiety reported to be useful for the treatment of cancer. Read More

Clearmind Medicine Inc. has completed IND-enabling studies with its 5-methoxy-2-aminoindane (MEAI)-based lead compound, CMND-100, aimed at treating alcohol use disorder (AUD). The company is preparing for an IND submission early next year to the FDA and the Israeli Ministry of Health to start first-in-human phase I/IIa studies in the U.S. and Israel. Read More

Aurigene Discovery Technologies Ltd. has divulged fused isoxazolyl compounds acting as histone acetyltransferase KAT6A (monocytic leukemia zinc finger protein; MOZ; MYST-3) inhibitors reported to be useful for the treatment of cancer. Read More

Chengdu Baiyu Pharmaceutical Co. Ltd. has identified piperazine derivatives acting as protein mono-ADP-ribosyltransferase TIPARP inhibitors reported to be useful for the treatment of cancer. Read More

Programmable genome insertion of long DNA sequences, useful for both gene therapy and basic research, commonly relies on cellular responses to double-strand breaks (DSBs) using programmable nucleases, such as CRISPR-Cas9, for induction of repair pathways such as non-homologous end joining (NHEJ). To overcome the current limitations of gene integration approaches, scientists from the Massachusetts Institute of Technology and colleagues developed a new strategy based on advances in programmable CRISPR-based gene editing, such as prime editing, together with the application of precise site-specific integrases. Read More

Drugs for Neglected Diseases Initiative has described pyrrolopirimidine derivatives reported to be useful for the treatment of leishmaniasis. Read More

Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has presented solute carrier family 22 member 12 (SLC22A12) inhibitors reported to be useful for the treatment of gout and hyperuricemia. Read More

The California Institute for Regenerative Medicine (CIRM) has awarded Calidi Biotherapeutics Inc. a US$3.1 million grant to support continued development of the company's Supernova-1 (SNV-1) preclinical program through IND application. The grant was awarded to Calidi to support IND-enabling studies, finalize manufacturing and the completion of Calidi's IND application for the SNV-1 program. Read More

Wave Life Sciences Ltd. and GSK plc have entered into a strategic collaboration to advance oligonucleotide therapeutics, including Wave's preclinical RNA editing program targeting α1-antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial 4-year research term. The first part of the arrangement is a discovery collaboration that enables GSK to advance up to eight programs and Wave to advance up to three programs, leveraging Wave's PRISM oligonucleotide platform and GSK's expertise in genetics and genomics. Read More

New and updated preclinical data presented at the American Society of Hematology Annual Meeting in New Orleans, by: Affimed, Asher Biotherapeutics, Disc Medicine, Keros Therapeutics, Kymera Therapeutics, Neoleukin Therapeutics, NGM Biopharmaceuticals, Oric Pharmaceuticals, Salarius Pharmaceuticals, Vincerx Pharma. Read More