“I love the idea of ‘micropublications’ (preparing one now),” the neurobiologist Oded Rechavi commented on social media in July. The term clearly suggests a short article, and although the publishing model has been around for more than a decade, not everyone is familiar with this type of scientific communication. What are they? What’s their impact? Rechavi, a professor at the School of Biochemistry, Neurobiology and Biophysics at Tel Aviv University, was pointing to an emerging discussion among scientists, the search for alternative formats for their work. Read More

Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, which is then converted by ENPP1 into AMP and pyrophosphate (PPi). Because PPi is a potent inhibitor of calcification, reduced systemic PPi production is a key driver of PXE. University of Pennsylvania investigators and collaborators proposed applying liver-targeted variant correction via genome editing as a single-intervention therapeutic approach for PXE, leading to subsequent restoration of systemic PPi. Read More

Nuage Therapeutics SL has secured €2.7 million (US$3.1 million) in funding from the Spanish State Research Agency (AEI) of the Ministry of Science, Innovation and Universities that will support a research project focused on developing a new drug against the most common subtype of small-cell lung cancer (SCLC-A), in collaboration with the Spanish National Cancer Research Centre (CNIO), the European Molecular Biology Laboratory (EMBL), and the Vall d’Hebron Institute of Oncology (VHIO). Read More

Treatment in glioblastoma usually fails due to tumor heterogeneity and persistence of glioblastoma multiforme stem-like cells (GSCs) within the tumor margin. Researchers from Trogenix Ltd. engineered TGX-007, an AAV1-mediated therapeutic that delivers both cytotoxic and immunomodulatory genetic payloads to the tumor. Read More

Apichope Pharmaceutical Co. Ltd., Guangzhou Lianrui Pharmaceutical Co. Ltd. and Guangzhou Runlin Pharmaceutical Technology Co. Ltd. have disclosed solute carrier family 22 member 12 (SLC22A12; URAT1) inhibitors reported to be useful for the treatment of hyperuricemia, nephropathy and gout. Read More

IL-2 is clinically validated as an immunotherapeutic, but its use is limited by toxicity issues. AZD-6750 is an approach from Astrazeneca plc that applies cis-guiding to deliver a modified IL-2 mutein to CD8+ T cells. Read More

Akeso Biopharma Inc. has developed an anti-IL-1RAP therapeutic, AK-135, for the potential treatment of chemotherapy-induced peripheral neuropathy. Read More

G1 to S phase transition 1 (GSPT1) is a protein involved in cell cycle progression, translation termination, and protein homeostasis, and its overexpression has been implicated in various cancers. Although GSPT1 is considered a promising therapeutic target, the lack of conventional ligand-binding pockets has historically rendered it undruggable. Read More

Abbisko Therapeutics Co. Ltd. has synthesized probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) inhibitors reported to be useful for the treatment of cancer. Read More

Chengdu Chipscreen Pharmaceutical Ltd. has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer. Read More

T cell-engaging bispecific antibodies (TCEs) are engineered molecules designed to bring cytotoxic T cells into proximity with tumor cells, triggering a targeted, antigen-dependent immune attack. However, TCE may activate T cells in healthy tissues, leading to off-tumor toxicity. Read More

Adlai Nortye Biopharma Co. Ltd. and Adlai Nortye Pte Ltd. have divulged cyclin-dependent kinase 2 (CDK2) and/or CDK4 and/or CDK6 inhibitors reported to be useful for the treatment of inflammatory disorders, immunological disorders and cancer. Read More

Alebund Pharmaceuticals (Hong Kong) Ltd. has described complement factor B (CFB) inhibitors reported to be useful for the treatment of glomerular disorders and rheumatoid arthritis. Read More

Triple-negative breast cancer (TNBC) is a highly aggressive subtype affecting 15%-20% of breast cancer patients. TNBC patients harboring breast cancer susceptibility gene 1/2 (BRCA1/2) mutations have shown improved therapeutic response to poly(ADP-ribose) polymerase (PARP) inhibitors (PARPi). Read More