Two new polio vaccine candidates designed to prevent the emergence of vaccine-derived virulent polioviruses have been shown to induce immune responses in mice, raising the possibility of eradicating the virus. For that to happen, the transmission of all poliovirus serotypes must be blocked. However, the vaccine used to control polio prevents disease but does not stop transmission, enabling the virus to mutate and regain virulence. Read More
New research shows base and prime editing can correct some forms of phenylketonuria (PKU) in mice and human cell lines, raising the prospect that this gene-editing approach could allow children born with the inherited metabolic disorder to have a treatment that would avoid the need for dietary restrictions and medication. Read More
In recent work, researchers from Mount Sinai School of Medicine applied a drug repurposing approach to identify candidates that could target inflammation in atherosclerosis. Read More
Researchers from F. Hoffmann-La Roche Ltd., Hoffmann-La Roche Inc. and Icagen Inc. have disclosed heteroaryl-urea compounds acting as potassium voltage-gated channel subfamily KQT member 2 (KCNQ2; Kv7.2) activators reported to be useful for the treatment of autism spectrum... Read More
Bruton tyrosine kinase (BTK) is involved in cell proliferation and survival in B-cell malignancies. Both covalent and noncovalent BTK inhibitors are in place, but their clinical benefit may be limited due to acquired BTK mutations. Read More
Alentis Therapeutics AG has received FDA clearance of the company’s IND application for ALE.C04, a first-in-class monoclonal antibody developed to specifically target exposed Claudin-1 (CLDN1) on cancer cells. Read More
Researchers at Hitgen Ltd. and Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. have identified proteolysis targeting chimeras (PROTACs) comprising cereblon (CRBN) ligands coupled to a Bcl-2-like protein 1 (Bcl-xl; Bcl-X; BCL2L1) targeting moiety via linker. Read More
Along with preliminary first-in-human data, investigators from F-star Therapeutics Inc. recently presented preclinical results for the tetravalent bispecific antibody FS-222, which targets PD-L1 and CD137 and is being developed for the treatment of advanced treatment-refractory solid tumors. Read More
Janssen Pharmaceutica NV has divulged macrocyclic compounds acting as induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reported to be useful for the treatment of cancer. Read More
Primary lymphedema (PL) is a chronic condition that results from abnormal development or functioning of the lymphatic system caused by gene mutations. Researchers from St George's University of London and colleagues reported the identification of novel variants in the ERG gene during their study performed through whole genome analysis of PL cases included in the 100,000 Genomes Project. Read More
Researchers from the University of Lausanne and affiliated organizations recently presented data from studies that aimed to identify novel genes associated with neurodevelopmental disease (NDD). Read More
Researchers from INSERM, Samsara Sciences Inc., Sorbonne University and Université Paris Cité have described autophagy inducers reported to be useful for the treatment of cancer, age-related disorders and viral infections. Read More