Spur Therapeutics Ltd. has selected SPR-301 as lead development candidate from its gene therapy program for a genetically defined subset of Parkinson’s disease characterized by mutations in the GBA1 gene. The mutations cause a deficiency in the enzyme glucocerebrosidase (GCase), leading to the accumulation of α-synuclein and subsequent death of neuronal cells that are hallmarks of Parkinson’s disease.
Switch Therapeutics Inc. has announced its first development candidate, a liver-sparing APOE (apolipoprotein E) RNAi therapy for treatment of Alzheimer’s disease in APOE4 carriers. Switch’s conditionally activated siRNA (CASi)-APOE program is designed to knock down APOE in the CNS without affecting APOE in the liver, where it plays a vital role in systemic lipid homeostasis.
Kinnate Biopharma Inc. and affiliated organizations recently reported preclinical data for the novel brain-penetrant combination therapy using the MEK inhibitor KIN-7136 and the RAF inhibitor KIN-8391, as a novel therapeutic strategy for the treatment of melanoma brain metastasis.
In breast cancer, neoadjuvant chemotherapy reduces the tumor before surgery. However, the response to this treatment does not depend solely on the subtype of malignancies. Other factors could play a key role in its effectiveness, as shown in a study that described how the estrous cycle phases contribute to this variation. The researchers propose adjusting the approach to the most suitable moment for patients.
Amgen Inc. has reported new 5,6- and 6,6-fused bicyclic alcohols and ethers acting as 15-hydroxyprostaglandin dehydrogenase (15-PGDH) inhibitors. They are reported to be useful for the treatment of gastrointestinal disorders.