Investigators from Biomarin Pharmaceutical Inc. have presented the first preclinical data for BMN-293, a novel adeno-associated virus (AAV) gene transfer vector carrying the MYBPC3 gene. MYBPC3 mutations can cause hypertrophic cardiomyopathy (HCM), a heart medical condition characterized by an abnormally thick myocardium, which makes it more difficult for the heart to pump blood.

Heterozygous familial hypercholesterolemia (HeFH) is caused by mutations in the LDL receptor gene, resulting in unusually high levels of low-density lipoprotein (LDL-C) in serum. Researchers from Epigenic Therapeutics Co. Ltd. presented the discovery of an epigenetic modulation therapeutic, EPI-001, for HeFH.

During the IDWeek conference held in Boston earlier this month, presentations on Climate Change were spread throughout the program. Some talks were on the direct effects of weather on infectious agents. Others discussed what healthcare workers could do to mitigate the effects of climate change, from antibiotic stewardship to decarbonization of day to day operations.

There is a need for new agents targeting PD-1/PD-L1 because many patients either do not respond or develop resistance to treatment.

Despite its promising therapeutic efficacy in patients with narcolepsy type 1, the previously reported orally available orexin OX2 receptor (OX2R) agonist TAK-994 has also demonstrated off-target liver toxicity. Now, researchers from Takeda Pharmaceutical Co. Ltd. have reported the discovery and early evaluation of a new OX2R agonist, TAK-861, being developed for the treatment of narcolepsy and other hypersomnia disorders.

Researchers from Boehringer Ingelheim Pharma GmbH & Co. KG presented the discovery and preclinical characterization of BI-3706674, a potent and orally available small-molecule inhibitor of the KRAS oncogene, targeting both KRAS-mutant and KRAS wild-type amplified (WTamp) cancers.