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BioWorld - Friday, April 24, 2026
Home » Newsletters » BioWorld Science

BioWorld Science

Sep. 5, 2023

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Metastatic rhabdomyosarcoma in sample aspirate from inguinal lymph node

Cell differentiation insight could help develop rhabdomyosarcoma treatment

Investigators at Cold Spring Harbor Laboratory have discovered a method of switching cancerous rhabdomyosarcoma cells back into normal muscle cells that could lead to the development of a treatment for this rare aggressive childhood cancer. Read More

Acuitas tackles mRNA drug delivery challenges with lipid nanoparticles

Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own. Read More
Multiple sclerosis

ASH-41020, a dendranib nanomedicine, reduces disease severity in EAE preclinical models

Researchers from Ashvattha Therapeutics LLC recently reported on ASH-41020, a new dendranib compound acting as a CSF-1R inhibitor with anti-inflammatory and immunomodulatory effects. Read More
3D-head-brain-cancer

Beactica selects BEA-17 as preclinical candidate from LSD1 program

Beactica Therapeutics AB has selected BEA-17 as a preclinical candidate from its lysine demethylase 1 (LSD1) program targeting aggressive brain tumors and other cancers, and IND-enabling toxicology studies will now be initiated. Read More

Vertex Pharmaceuticals patents new APOL1 inhibitors

Vertex Pharmaceuticals Inc. has disclosed apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of focal segmental glomerulosclerosis and pancreatic cancer. Read More
Epileptic brain and abnormal EEG wave discharges

Uniqure’s AMT-260 gene therapy cleared by FDA to enter clinic for mesial temporal lobe epilepsy

Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE). Read More

Enliven Therapeutics divulges new HER2 inhibitors for cancer

Enliven Therapeutics Inc. has synthesized HER2 (erbB2) inhibitors, including Exon 20 insertion (Ex20Ins) mutant inhibitors, reported to be useful for the treatment of cancer. Read More
Test tubes, dropper and capsules

Krystal Biotech’s KB-408 awarded US orphan drug designation for AATD

The FDA has awarded orphan drug designation to Krystal Biotech Inc.’s KB-408 for the treatment of α1-antitrypsin deficiency (AATD). Read More

Gasherbrum Bio describes new GLP-1R agonists

Gasherbrum Bio Inc. has identified glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others. Read More

Madrigal Pharmaceuticals presents new THR-β agonists

Madrigal Pharmaceuticals Inc. has divulged thyroid hormone receptor β (THR-β) agonists reported to be useful for the treatment of familial hypercholesterolemia, hyperlipidemia, hypertriglyceridemia and nonalcoholic steatohepatitis. Read More
pill over molecule structures

Novartis discloses new molecular glue degrader against NEK7

Molecular glue degraders are compounds intended to prompt the degradation of E3 ubiquitin ligases by engaging and modifying their surface. Molecular glue-induced protein degradation is considered an emerging strategy in drug discovery. Read More

Novel TLR7 agonist demonstrates synergistic antitumor activity with anti-PD-1 antibodies

Researchers from Bristol Myers Squibb Co. recently discussed the discovery and preclinical characterization of a series of Toll-like receptor 7 (TLR7) agonists with potential as anticancer drug candidates. Read More

Biogen discovers new EBP inhibitors for multiple sclerosis

Biogen Inc. has described 3-β-hydroxysteroid-δ(8),δ(7)-isomerase (EBP) inhibitors reported to be useful for the treatment of multiple sclerosis. Read More
3D representation of tumor

Discovery of selective CDK2 inhibitors with potent antitumor activity

Researchers from Genentech Inc. have reported the discovery of new cyclin-dependent kinase 2 (CDK2) inhibitors as potential anticancer agents. Read More

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