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BioWorld - Wednesday, April 29, 2026
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Neurons
Genetic/congenital

Single-gene therapy for LSDs with modified lysosomal enzyme shows preclinical efficacy

April 27, 2026
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In previous work, researchers from Kawasaki Medical School and collaborating institutions engineered a modified HEXB construct, modHexB, to improve GM2 ganglioside (GM2) recognition and GM2-activating protein (GM2A) interaction. The team has now combined these previous advancements to develop a new gene therapy strategy for Sandhoff disease.
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Digital cancer cells illustration
Cancer

Insilico Medicine nominates ISM-0387 as development candidate

April 27, 2026
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Insilico Medicine Cayman Topco has nominated ISM-0387, an MTA-cooperative protein arginine methyltransferase 5 (PRMT5) inhibitor, as a preclinical developmental candidate for glioblastoma. The discovery and optimization process for ISM-0387 was powered by Chemistry42, Insilico’s generative chemistry platform integrated with over 40 generative AI models.
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Immuno-oncology

Anti-LAG-3 IL-2 fusion protein enhances ICI and CAR T response in solid tumors

April 27, 2026
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Although CAR T-cell therapy has been highly effective in hematological cancers, its activity in solid tumors is still constrained by inhibitory checkpoints such as PD-1, LAG-3 and TIM-3, together with the lack of locally available cytokines such as IL-2 needed to sustain T-cell function. Anwita Biosciences Inc. and collaborators aimed to overcome these challenges by developing a strategy integrating an immune checkpoint inhibitor (ICI) with a T-cell stimulatory cytokine.
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Silhouette of head, brain
Neurology/psychiatric

Plasticity, properly parsed, provides psychiatric platform

April 27, 2026
By Anette Breindl
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If Benjamin Braddock, of The Graduate fame, were a young neuroscientist in the 21st century instead of a liberal arts graduate in 1967, the advice he received from his parents’ neighbor might not have been “One word: plastics!” but “One word: plasticity!” Plasticity is a hot concept in neuropsychiatric disorders. New and old treatment modalities, these days, are said to work as psychoplastogens or neuroplastogens.
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Glowing neural network inside a transparent capsule surrounded by a large language model
Drug design, drug delivery & technologies

Pharma industry faces long haul to get return on investment from AI

April 24, 2026
By Nuala Moran
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Artificial intelligence tools are springing up at multiple points along drug discovery and development, but despite the hype, as yet there is minimal return on investment (ROI). “I would say a lot of companies sort of get this big excitement about AI, but then when you look at how much ROI they get, it’s actually very little. And that’s because the workflow and the process, end-to-end, isn’t mapped to really understand where AI can truly make an impact,” said Laura Matz, chief science and technology officer at Merck KGaA.
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Respiratory

Jiangsu Carephar Pharmaceutical describes new AT2 receptor agonists

April 24, 2026
Jiangsu Carephar Pharmaceutical Co. Ltd. has patented angiotensin AT2 receptor (AGTR2) agonists reported to be useful for the treatment of idiopathic pulmonary fibrosis.
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Cancer

Shanghai Meiyue Biotech Development presents new molecular glue degraders

April 24, 2026
Shanghai Meiyue Biotech Development Co. Ltd. has divulged molecular glue degraders comprising an E3 ubiquitin-protein ligase/proto-oncogene Vav (VAV1) interaction inducer and VAV1 degradation inducer. They are designed for potential use in the treatment of cancer, autoimmune diseases, cardiovascular, renal, metabolic, inflammatory and neurological disorders.
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Neurology/psychiatric

Sichuan Kelun Pharmaceutical Research Institute discovers GPR6 inverse agonists

April 24, 2026
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. has identified G protein-coupled receptor 6 (GPR6) inverse agonists reported to be useful for the treatment of depression, bipolar disorder, substance abuse and dependence, eating disorders, schizophrenia, cognitive disorders, Parkinson’s disease and Huntington's disease, among others.
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Endocrine/metabolic

Suzhou Spring-Sea Bio-Pharmaceuticals discloses new GLP-1R/GCGR/GIPR agonists

April 24, 2026
Suzhou Spring-Sea Bio-Pharmaceuticals Co. Ltd. has synthesized glucagon-like peptide 1 receptor (GLP-1R), glucagon receptor (GCGR) and gastric inhibitory polypeptide receptor (GIPR) agonists that are potentially useful for the treatment of dyslipidemia, hepatic steatosis, metabolic syndrome, obesity and type 2 diabetes.
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Cancer

Chinese researchers identify new antibody-drug conjugates

April 24, 2026
Scientists from Jiangsu Hengrui Pharmaceuticals Co. Ltd., Shanghai Senhui Pharmaceutical Co. Ltd. and Shanghai Shengdi Pharmaceutical Co. Ltd. have disclosed antibody-drug conjugates comprising antibodies covalently linked camptothecin derivatives through a linker reported to be useful for the treatment of cancer.
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