The threat posed by the Ebola outbreak in the Democratic Republic of Congo has intensified, with the confirmation that it is caused by the Bundibugyo species of the virus, for which there are no approved vaccines or antiviral therapies. At the same time, the high positivity rate, with eight laboratory confirmed cases out of 13 samples collected in various areas, and more reports of suspected cases and clusters of deaths, all point to a potentially much larger outbreak than currently is being detected and reported. Read More
Researchers from the University of Copenhagen and collaborating institutions aimed to develop a therapy for chronic neuropathic pain based on gene therapy delivered with adeno-associated viral (AAV) vectors. Read More
Harbour Biomed has released preclinical data for LET-003, a next-generation ACVR2A/2B-targeted monoclonal antibody with potential for the treatment of obesity. Activin receptors ACVR2A and ACVR2B play critical roles in regulating muscle-fat metabolic homeostasis. Read More
Type 2 diabetes is marked by insulin resistance coupled with insufficient insulin secretion due to early β-cell dysfunction and progressive loss of β-cell mass. Pdx1 and MafA, critical for maintaining β-cell function, are progressively reduced under metabolic stress and in patients, driving disease progression. Researchers at the University of Pittsburgh have reported efficacy data demonstrating successful pancreatic delivery of GPX-002, an AAV-Pdx1/MafA construct, in HFD mice. Read More
Neurolambda Therapeutics Inc. has disclosed new non-receptor tyrosine-protein kinase TYK2 inhibitors potentially useful for the treatment of cancer, systemic lupus erythematosus, rheumatoid arthritis and psoriatic arthritis. Read More
The University of Texas MD Anderson Cancer Center has received IND clearance from the FDA to initiate a phase I trial of a novel CAR T-cell therapy, JV-394, for patients with relapsed or refractory CD94-positive T/natural killer (NK) cell lymphomas. Read More
Cell replacement therapy is currently being investigated to restore the retinal pigment epithelium (RPE) layer and treat vision decline due to dry age-related macular degeneration (AMD). The Neural Stem Cell Institute and collaborating institutions developed an approach to RPE replacement using an adult RPE stem cell source derived from the RPE layer of donated cadaver eyes. Read More
Dgenthera Inc. and the University of North Carolina at Chapel Hill have jointly patented new radiolabeled bioactive compounds potentially useful for the diagnosis and treatment of cancer. Read More
Shouyao Holdings (Beijing) Co. Ltd. has reported new proto-oncogene tyrosine-protein kinase receptor Ret (RET; CDHF12; PTC) G810R mutant inhibitors potentially useful for the treatment of cancer. Read More
Ubiquitin carboxyl-terminal hydrolase 1 (USP1) is a synthetic lethality target in cancers with high replication stress, such as tumors with BRCA gene mutations or homologous recombination deficiency (HRD). Targeting USP1 has emerged as a promising approach to overcome resistance observed in BRCA/HRD-driven tumors. Aigen Sciences Inc. developed and presented data for AIG-07025, a potent and selective USP1 inhibitor. Read More
Nxera Pharma UK Ltd. has identified new uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-Like) antagonists potentially useful for the treatment of multiple sclerosis. Read More
Transforming acidic coiled-coil-containing protein 3 (TACC3) is a core member of multiprotein complexes that regulate microtubule- and centrosome-related processes. Its aberrant expression is found in several cancer types with poor prognosis, thus highlighting it as a candidate therapeutic target. Researchers from Beijing Konruns Pharmaceutical Co. Ltd. have presented data for KC-1101, a TACC3 inhibitor for treating aggressive cancers with centrosome amplification. Read More
