distillery

Treating antibiotic-resistant Gram-negative infection with stapled antimicrobial peptides

DISEASE CATEGORY: Infectious disease INDICATION: Gram-negative bacterial infection; sepsis Stapled antimicrobial peptides could treat antibiotic-resistant Gram-negative infections, including Acinetobacter baumanii -induced sepsis. The compounds consisted of stapled peptides derived from the antimicrobial peptide (AMP) magainin...

Agonizing GABA A receptor in peripheral neurons to treat autism

DISEASE CATEGORY: Neurology INDICATION: Autism Increasing signaling through GABA A receptors in peripheral neurons could treat autism. In a genetic mouse model of autism, peripheral sensory nerve expression of GABRB3, an obligatory subunit if GABA...

Beam co-founder describes base editors with reduced RNA editing

TECHNIQUES CATEGORY: Drug platforms TECHNOLOGY: Gene therapy Beam Therapeutics co-founder J. Keith Joung and colleagues described cytosine and adenine base editors that could reduce off-target RNA editing compared with prior versions of the technologies. An...

Perlecan identified as new tumor stroma target

DISEASE CATEGORY: Cancer INDICATION: Pancreatic cancer Depleting perlecan, a protein deposited in the tumor stroma by cancer associated fibroblasts (CAFs), could help treat metastatic pancreatic cancer. An Australian team showed that perlecan secretion was higher...

CDK11B as a new cancer target

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer; pancreatic cancer; breast cancer Inhibiting CDK11B, a kinase involved in cellular processes including transcription and chromosome segregation, could treat cancers including colorectal, pancreatic, lung cancer and melanoma. In colorectal...

ROS1 and pan-NTRK inhibitor for NSCLC resistant to Xalkori

DISEASE CATEGORY: Cancer INDICATION: Non-small cell lung cancer (NSCLC) A dual ROS1 and pan-NTRK inhibitor from Daiichi Sankyo Co. Ltd. could treat Xalkori crizotinib-resistant NSCLC, which has been linked to ROS1 mutations. In vitro high...

Preventing metastasis in TNBC with liposome-delivered TRAIL

DISEASE CATEGORY: Cancer INDICATION: Breast Cancer Liposomes formulated to display the apoptosis inducer TRAIL and the tumor-binding adhesion molecule SELE could treat triple-negative breast cancer (TNBC) and prevent metastases following tumor resection. When administered to...

SMPD1 gene therapy for Niemann-Pick disease type A

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Metabolic SMPD1, a sphingomyelinase, encoded in viral vector could treat Niemann-Pick disease type A. The lysosomal storage disorder is caused by loss-of-function mutations in SMPD1 and is characterized by motor impairments,...

A mAb blocking IL-1 co-receptor for inflammatory, autoimmune diseases

DISEASE CATEGORY: Inflammation; autoimmune disease INDICATION: Inflammation; allergy; psoriasis A mAb against the IL-1 family co-receptor IL1RAP could treat peritonitis, allergic airway inflammation and psoriasis. The anti-IL1RAP mAb, which has an Fc domain mutated to...

Inhibition of G9A and DNMT methyltransferases in bladder cancer

DISEASE CATEGORY: Cancer INDICATION: Bladder cancer Dual inhibition of G9A and DNMT methyltransferases, in combination with cisplatin and anti-programmed cell death 1 ligand 1 (PD-L1), could help to treat bladder cancer. A tool compound that...

Tumor-targeted serum albumin-chemotherapy conjugates for improved delivery to breast and colorectal cancers

DISEASE CATEGORY: Cancer INDICATION: Breast cancer; colorectal cancer Delivering chemotherapies through conjugation to tumor-targeted albumin could treat breast and colorectal cancers. Albumin is fused with collagen-binding domain of vWF to facilitate attachment to the tumor...

CXCL10-CXCR3 axis inhibition to block brain metastasis from melanoma

DISEASE CATEGORY: Cancer INDICATION: Melanoma Inhibiting the chemoattractant CXCL10 or its receptor CXCR3 could prevent melanoma from forming brain metastases. Levels of CXCL10 mRNA were higher in metastases-associated astrocytes isolated from mice with melanoma than...

Peptide-macrocycle conjugates for breast cancer

DISEASE CATEGORY: Cancer INDICATION: Breast cancer Hydroquinone macrocycle-peptide conjugates targeting CD51/CD61 could treat breast cancer by trapping polyamines, which promote cell proliferation and differentiation. The conjugates consist of pillararenes attached to the peptide sequence arginine-glycine-aspartate...

S100A9 could normalize metabolic imbalances from insulin deficiency

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Diabetes Promoting expression of S100A9, a plasma protein induced by leptin, could treat diabetes. Type I and Type II diabetes induce insulin deficiency, which causes metabolic imbalances including hyperglycemia, hyperglucagonemia, hyperketonemia,...

DNM1L identified as pancreatic cancer target

DISEASE CATEGORY: Cancer INDICATION: Pancreatic cancer Inhibiting DNM1L, a GTPase involved in mitochondrial fission, could treat pancreatic cancer. DNM1L knockdown reduced proliferation across four patient-derived pancreatic cancer cell lines with K-Ras (KRAS) mutations. In mice...