Previous studies have identified a homozygous CDK9 variant in five different families (four Arabic and one Japanese) with CHARGE syndrome, suggesting that CDK9 could be a causative gene in autosomal recessive CHARGE-like syndrome. Researchers from the University of Kuwait and affiliated organizations thus aimed to further investigate this disease-gene association.

Briacell Therapeutics Corp. has received IND clearance from the FDA to begin clinical studies with Bria-OTS, the first generation of the company’s personalized off-the-shelf cellular immunotherapy for breast cancer.

CSPC Pharmaceutical Group Ltd.’s SYS-6011 has been cleared by China’s National Medical Products Administration (NMPA) to enter clinical trials in China for solid tumors.

It is well known that mutations in the cystic fibrosis transmembrane regulator (CFTR) gene are causative of cystic fibrosis, a lethal autosomal recessive Mendelian disorder. Several studies have also pointed to an association between CFTR mutations and inflammatory bowel disease (IBD).

Polycystic ovary syndrome (PCOS) is a form of anovulatory infertility characterized by hyperandrogenemia, irregular menses and polycystic ovarian morphology. Apart from experiencing reproductive-related symptoms, women with PCOS face an increased risk of obesity, insulin resistance and type 2 diabetes.

Biocryst Pharmaceuticals Inc. has highlighted progress in its pipeline, with five programs set to enter the clinic in the next 24 months.

Scientists at the University of California San Francisco (UCSF) have designed a group of synthetic molecules that could prevent the rejection of allogeneic cell transplants. Their strategy consisted of activating the immune checkpoints of different populations of immune cells from the cell surface, but avoiding the cytotoxicity of natural killer (NK) cells and macrophages that would destroy the transplanted cells.