Newco Tecregen AG has raised CHF10 million (US$12.5 million) to advance a novel way of regenerating the thymus in order to boost T-cell production and stimulate recovery of the immune system following stem cell transplants and chemo- and radiotherapy. Read More
FGFR1 is a receptor tyrosine kinase that drives multiple intracellular signaling pathways involved in tumor cell proliferation, survival and progression. Because these pathways are frequently hyperactivated in colorectal cancer (CRC), FGFR1 represents a biologically relevant therapeutic target, and its inhibition has the potential to simultaneously suppress several oncogenic mechanisms. Read More
Fannin Partners LLC has been awarded a $300,000 phase I Small Business Innovation Research (SBIR) grant from the National Eye Institute (NEI) to advance a first-in-class, non-VEGF-targeted therapeutic for wet age-related macular degeneration (AMD) in patients not responding to anti-VEGF therapies. Read More
Keiferx LLC has entered into a research collaboration and option agreement with Amneal Pharmaceuticals Inc. to support the pre-IND development of KFRX-06, Keiferx’s brain-penetrant preclinical candidate designed to inhibit leucine-rich repeat kinase 2 (LRRK2), a gene strongly implicated in Parkinson’s disease biology. Read More
The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study. Read More
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Both rare and common genetic variants contribute to the risk of schizophrenia and CALN1 has been proposed as a candidate schizophrenia risk gene. Read More
Enodia Therapeutics SAS has raised €20.7 million ($25 million) in seed financing to advance its work developing novel small-molecule therapies for targeted protein degradation at the point of synthesis. Read More
Jiangsu Deyuan Pharmaceutical Co. Ltd. has described cytochrome P450 11B2, mitochondrial (CYP11B2; aldosterone synthase; ALDOS) inhibitors reported to be useful for the treatment of hypertension. Read More
Adenosine deaminase acting on RNA (ADAR) is the enzyme responsible for adenosine-to-inosine (A-to-I) RNA editing, a process essential for regulating how cells respond to double-stranded RNA. ADAR1 generates two isoforms, p150 and p110, with distinct roles in cancer biology. Researchers from the Wistar Institute of Anatomy & Biology reported the preclinical characterization of ADAR1-i-124, an ADAR1 inhibitor designed for the treatment of cancer. Read More
Hanmi Pharmaceutical Co. Ltd. has disclosed polypeptides acting as triple agonists of glucagon like peptide 1 receptor (GLP-1R), glucagon receptor (GCGR) and glucose-dependent insulinotropic receptor (GDIR; GPR119) reported to be useful for the treatment of dyslipidemia. Read More
Linkcure Therapeutics has synthesized molecular glue degraders acting as zinc finger protein 803 (ZNF803; WIZ) degradation inducers reported to be useful for the treatment of sickle cell anemia and β-thalassemia. Read More
The treatment of Pseudomonas aeruginosa infections is significantly challenged by the pathogen’s diverse resistance mechanisms, with biofilm formation being a key driver of antibiotic tolerance. Furthermore, P. aeruginosa pathogenicity is amplified by virulence factors that both evade host defenses and facilitate biofilm development. Read More
Monash University has identified cyclic peptides acting as melanocortin MC5 receptor (MC5R) agonists reported to be useful for the treatment of diabetes, obesity, cardiomyopathy, heart failure, renal disorders, Rabson Mendenhall syndrome, Donohue syndrome and lipodystrophy. Read More
Triana Biomedicines Inc. has divulged protein/nucleic acid degraders acting as cyclin-dependent kinase 2 (CDK2) degraders reported to be useful for the treatment of cancer. Read More
Disruption of alternative splicing can generate abnormal mRNA variants, producing nonfunctional proteins or triggering nonsense-mediated decay, and is implicated in many splicing-related diseases. Therapeutic approaches that modulate splicing, therefore, hold promise to redirect aberrant transcripts toward normal isoforms and reestablish functional protein expression. Read More
