distillery

Peptide inhibitors of a histone chaperone for osteosarcoma and breast cancer

DISEASE CATEGORY: Cancer INDICATION: Bone cancer; breast cancer A team from the French Alternative Energies and Atomic Energy Commission identified two peptide inhibitors of the histone chaperone ASF1A that could treat osteosarcoma and breast cancer....

Dual TLR7 and TLR8 agonism for pancreatic cancer

DISEASE CATEGORY: Cancer INDICATION: Pancreatic cancer Agonizing stromal TLR7 and TLR8 could treat pancreatic ductal adenocarcinoma (PDAC). In mice with PDAC, a TLR7 and TLR8 agonist tool compound reduced tumor growth and hallmarks of cachexia,...

Allosteric inhibitor of PGAM1 identified for NSCLC

DISEASE CATEGORY: Cancer INDICATION: Non-small cell lung cancer (NSCLC) A benzenesulfonamide-based allosteric inhibitor of PGAM1, given alone or in combination with the EGFR inhibitor Tarceva erlotinib, could treat NSCLC. Structure-based optimization of a previously reported...

GCCR inhibition could treat Ewing sarcoma

DISEASE CATEGORY: Cancer INDICATION: Bone cancer A team from the Weizmann Institute showed that GCCR inhibitors could be repurposed to treat Ewing sarcoma. The GCCR inhibitor Mifegyne mifepristone decreased proliferation in cultures of human Ewing...

Human symbiont-derived antimicrobial peptide for Gram-positive infections

DISEASE CATEGORY: Infectious disease INDICATION: Clostridium ; Gram-positive bacterial infection An antimicrobial peptide produced by the human gut microbiome species Ruminococcus gnavus could treat infections caused by Clostridium and other Gram-positive bacteria. In bacterial culture,...

Combined inhibition of G12C-mutant KRAS, mTOR and IGF1R for NSCLC

DISEASE CATEGORY: Cancer INDICATION: Non-small cell lung cancer (NSCLC) Triple therapy with KRAS-G12C, mTOR and IGF1R inhibitors could treat KRAS G12C-mutant NSCLC. Whole genome screening of an shRNA library for targets whose inhibition enhanced the...

Prolactin inhibition could treat MS

DISEASE CATEGORY: Autoimmune disease INDICATION: Multiple sclerosis (MS) Inhibition of PRL or ZBTB20, a transcription factor that promotes PRL expression, could treat MS. In the experimental autoimmune encephalomyelitis (EAE) mouse model of MS, CSF levels...

CD49e/CD61 inhibition for breast cancer and melanoma

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; melanoma Inhibition of CD49e/CD61 alone or in combination with anti-PD-1 therapies could treat breast cancer and melanoma. An siRNA against CD49e/CD61 reduced tumor volume in mice with breast cancer....

Enleofen co-founders identify IL-11 as pulmonary fibrosis target

DISEASE CATEGORY: Pulmonary INDICATION: Pulmonary fibrosis A National Heart Centre Singapore team led by Enleofen Bio Pte. Ltd. co-founders Stuart Cook and Sebastian Schafer showed that inhibiting IL-11 could treat pulmonary fibrosis. Levels of IL-11...

CAR T cell-derived exosomes for cancer

DISEASE CATEGORY: Cancer INDICATION: Lung cancer; breast cancer; ovarian cancer Exosomes derived from CAR T cells could treat solid solid tumors, with greater safety than the cell therapy. Exosomes from CAR Ts targeting EGFR or...

GCase activator reduces α-synuclein in mouse, patient-derived models of PD

DISEASE CATEGORY: Neurology INDICATION: Parkinson's disease (PD) Northwestern University researchers, including Lysosomal Therapeutics Inc. co-founder Dimitri Krainc, developed a small molecule GCase activator that could treat Parkinson’s disease. Mutations in GCase, a lysosomal enzyme, are...

Combined PTP-1B and PTPN2 inhibition for weight loss in obesity

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Obesity Combined inhibition of PTP-1B and PTPN2, which regulate leptin and insulin signaling, could promote weight loss in obese patients. In mice with high fat diet-induced obesity, knockout of both targets...

Engineered hematopoietic stem and progenitor cells for mucopolysaccharidosis type I

DISEASE CATEGORY: Endocrine/Metabolic INDICATION: Mucopolysaccharidosis Hematopoietic stem and progenitor cells (HSPCs) engineered to overexpress IDUA could treat mucopolysaccharidosis I (MPS I; Hurler syndrome), a lysosomal storage disease caused by insufficient IDUA activity. The enzyme deficiency...

Small molecule disruption of Hsp90-CDC37 interaction for colorectal cancer

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer A small molecule inhibiting the interaction between the stress response protein Hsp90 and its co-chaperone CDC37 could treat colorectal cancer. In silico docking studies of a small molecule library...

Boosting YY1 expression to treat diabetic nephropathy

DISEASE CATEGORY: Renal INDICATION: Diabetic nephropathy Promoting the expression of YY1, which represses TGFB1 transcription, could treat diabetic nephropathy. In renal biopsy samples, low levels of YY1 and high levels of TGFB1 were associated with...