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BioWorld - Friday, December 5, 2025
Home » Newsletters » BioWorld Science

BioWorld Science

Sep. 14, 2022

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Lysosome clusters in a mouse model of Alzheimer's disease.

Newly identified lysosomal repair pathway could prevent neurodegeneration

Age-related diseases have been explained as due in part to the excessive generation and accumulation of waste products like the various insoluble protein aggregates observed in nondividing neurons of Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis and Huntington’s disease. Read More

Bioengineered multifunctional exosomes evoke cancer immunity

The design of genetically modified exosomes that combine multiple targets killed cancer cells and conferred immunity against them. Scientists at the University of Southern California (USC) applied bioengineering techniques to introduce up to four antitumor functions in the same type of extracellular vesicles and destroy EGFR-positive triple-negative breast cancer (TNBC) tumor cells. Read More
Antibodies attacking cancer cell

Transcenta cleared to advance gremlin-1-targeting antibody TST-003 into clinic for solid tumors

Transcenta Holding Ltd. has received IND clearance from the FDA for TST-003, its first-in-class, high-affinity, humanized monoclonal antibody targeting gremlin-1. Read More
Lung cancer illustration

BI-4142 and BI-1622 show efficacy against HER2 exon 20 insertion-driven tumors, while preserving EGFR wt signaling

Boehringer Ingelheim Pharma GmbH & Co KG has presented data on the identification of several potent and selective tyrosine kinase inhibitors (TKIs) of HER2 that are highly active against its oncogenic exon 20 YVMA insertion mutant (HER2 YVMA), which is the most prevalent HER2 exon 20 mutation in non-small-cell lung cancer (NSCLC). Read More

NXP-800 results in tumor regression in xenograft model of ARID1a-mutated gastric carcinoma

Nuvectis Pharma Inc. has released promising data on NXP-800 in a preclinical xenograft model of ARID1a-mutated gastric carcinoma. Read More
Test-tube-dropper-research

Akouos receives IND clearance for AK-OTOF gene therapy for OTOF-mediated hearing loss

Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss. Read More

Lift Biosciences' N-Lift destroys tumors in five solid tumor types in PDX tumoroid models

Lift Biosciences Ltd. has shown that its first-in-class cell therapy destroyed on average over 90% of the tumoroid in a PDX (patient-derived xenograft) organoid across five solid tumor types, including bladder cancer, rectal cancer, colorectal cancer, gastric cancer and squamous cell non-small-cell lung cancer (NSCLC). Read More

Coave works with IMN on gene therapies targeting protein degradation in neurodegenerative disorders

Coave Therapeutics SA has entered into a collaboration with the Institute of Neurodegenerative Diseases (IMN), a joint research unit associating the University of Bordeaux and the French National Centre of Scientific Research (CNRS), to develop gene therapy programs targeting protein degradation in neurodegenerative disorders. Read More

Discovery of novel ponatinib analogues with reduced cardiotoxicity

Researchers from Stanford University have published an article on the discovery of novel analogues of ponatinib that retained antitumor efficacy with substantially reduced cardiotoxicity. Read More

Nacuity and Arctic Therapeutics partner on NPI-001 for hereditary cystatin C amyloid angiopathy

Nacuity Pharmaceuticals Inc. and Arctic Therapeutics LLC have entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of NPI-001 (N-acetylcysteine amide, NACA) in Iceland for the treatment of hereditary cystatin C amyloid angiopathy (HCCAA). Read More

Novartis presents new RET inhibitors for lung adenocarcinoma

Proto-oncogene tyrosine-protein kinase receptor Ret is a widely expressed oncogene and chromosomal rearrangements involving RET lead to fusion genes and RET kinase activation, which occur in lung cancer in about in 2% of cases of non-small cell lung cancer. Read More
Science-research-pic.png

Nanoparticle-encapsulated diabetogenic protein cocktail inhibits type 1 diabetes development

Researchers at Northwestern University and COUR Pharmaceutical Development Company Inc. have shown that biodegradable carboxylated poly(D,L-lactic-co-glycolic acid) (PLGA) nanoparticles containing encapsulated Ag (tolerance-inducing microparticles [TIMPs] or COUR nanoparticles [CNPs]) are able to prevent and treat type 1 diabetes (T1D) induced by transfer of monospecific diabetogenic CD4 and CD8 transgenic T cells to NOD.scid mice. Read More

Maze Therapeutics discloses new APOL1 inhibitors

Maze Therapeutics has patented apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of chronic kidney disease, focal segmental glomerulosclerosis, HIV-associated nephropathy, lupus nephritis and sepsis. Read More

Wellstat Therapeutics divulges new codrug of uridine and ketoleucine

Wellstat Therapeutics Corp. has described compounds for co-delivery of uridine and ketoleucine with high bioavailability reported to be useful for the treatment of muscle atrophy, sarcopenia and cachexia. Read More
Microscope and coronavirus illustration

Tikomed reports in vitro findings on ILB against coronavirus infection

Tikomed AB has reported an in vitro study evaluating the antiviral effect of its lead drug candidate ILB in coronavirus infection. Read More

New HDAC8 inhibitors disclosed by Klotho Therapeutics

Klotho Therapeutics Inc. has synthesized new histone deacetylase 8 (HDAC8) inhibitors reported to be useful for the treatment of acute kidney injury and chronic kidney disease. Read More

Biohaven Therapeutics identifies TDP43-targeting compounds for neurologic disorders

Biohaven Therapeutics Ltd. has divulged compounds targeting TAR DNA-binding protein 43 (TDP43) reported to be useful for the treatment of amyotrophic lateral sclerosis, traumatic encephalopathy, sporadic inclusion body myositis, frontotemporal dementia and Alzheimer's disease. Read More
Tuberculosis.png

GSK agent targets M. tuberculosis tryptophan synthase for resistant tuberculosis

GSK plc scientists seeking compounds with the potential to treat resistant tuberculosis infection identified a series of tetrazole agents using phenotypic screening against Mycobacterium tuberculosis. Read More

Concentric Analgesics patents new TRPM8 agonists

Concentric Analgesics Inc. has discovered transient receptor potential cation channel subfamily M member 8 (TRPM8) agonists reported to be useful for the treatment of oropharyngeal dysphagia. Read More

Other news to note for Sept. 14, 2022

Drug R&D updates, including preclinical data readouts and publications, from: Nura Bio, Therapeutic Solutions, Tikomed AB. Read More

Preclinical conference data for Sept. 14, 2022: ESMO

New and updated preclinical data presented at the Annual Congress of the European Society for Medical Oncology in Paris, including: Enlivex, GT Biopharma, Immatics, SQZ Biotechnologies. Read More

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