The first cellular human and mouse map focused on muscle fibers and their microenvironment has revealed both the mechanisms of deterioration of this tissue over time and its adaptive capacity for regeneration. “We intended to map the skeletal muscle, isolating all the cell types, and characterizing how they change with age,” first author Veronika Kedlian from the Wellcome Sanger Institute in Cambridge told BioWorld.
The first cellular human and mouse map focused on muscle fibers and their microenvironment has revealed both the mechanisms of deterioration of this tissue over time and its adaptive capacity for regeneration. “We intended to map the skeletal muscle, isolating all the cell types, and characterizing how they change with age,” first author Veronika Kedlian from the Wellcome Sanger Institute in Cambridge told BioWorld.
Proprotein convertase subtilisin/kexin type-9 (PCSK9) is highly expressed in adult hepatocytes. PCSK9 binds to and promotes the degradation of the low-density lipoprotein (LDL) receptor, thereby increasing LDL cholesterol levels. PCSK9 inhibition has emerged as a promising strategy for cardiovascular diseases. However, it is still unclear whether PCSK9 can trigger blood vessel inflammation directly modulating monocytes or endothelial cells independently of LDL receptor.
Saliogen Therapeutics Inc. has nominated a development candidate, SGT-1001, for the treatment of Stargardt disease. SGT-1001 is being developed as a one-time, nonviral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
Derm-Biome Pharmaceuticals Inc. has developed a topical anticancer treatment for patients with or at high risk of developing multiple actinic keratoses.
Researchers from Astrazeneca plc reported preclinical data for AZD-8421, a selective cyclin-dependent kinase 2 (CDK2) inhibitor, currently being evaluated in early-phase clinical trials as a treatment for solid tumors.
Circio Holding ASA has established technical in vivo proof of concept for its proprietary Circvec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression.
Wista Laboratories Ltd. has disclosed thiazole-containing compounds acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of corticobasal syndrome, Alzheimer’s disease, Down syndrome and more.
The leukocyte immunoglobulin-like receptor B2 (LILRB2), also known as ILT4, is an immunoreceptor tyrosine-based inhibitory motif (ITIM)-containing receptor that is widely expressed on immunosuppressive myeloid cells in the tumor microenvironment.
Mutations in GTPase KRAS occur in about 25% of human cancers, with the KRAS G12V mutation being one of the most frequent variants, and lead to activation of the MAPK pathway, thus promoting tumorigenesis.
Nephrotic syndrome is a kidney disorder characterized by abnormal functioning of the glomerular filtration barrier, and is frequently caused by focal segmental glomerulosclerosis (FSGS). At the World Congress of Nephrology meeting this week, researchers presented a case report of a 32-year-old...
Zinc transporter ZIP6, also known as LIV-1, is a transmembrane protein that is an interesting target for antibody-drug conjugate (ADC) therapy because of its higher expression in tumors and almost no expression in normal tissues.
Curevac NV and The University of Texas MD Anderson Cancer Center have entered into a co-development and licensing agreement to develop novel mRNA-based cancer vaccines.
Spikimm SAS has signed an exclusive collaboration and license option agreement with SATT Conectus Alsace SAS for monoclonal antibodies targeting the BK virus. Reactivation of the BK virus (BKV) in patients receiving kidney transplant, bone marrow or stem cell grafts has potential serious consequences.
The FDA has granted orphan drug designation to the active ingredient in Soligenix Inc.’s Marvax, a heat stable subunit protein vaccine of recombinantly expressed Marburg marburgvirus (MARV) glycoprotein, for the prevention and post-exposure prophylaxis against MARV infection.
Vaccination with infectious Plasmodium falciparum sporozoites (PfSPZ) administered with antimalarial drugs (PfSPZ-CVac) is more effective than vaccination with replication-deficient, radiation-attenuated PfSPZ. However, the requirement for drug administration is a significant limitation of the...
Trace amine-associated receptor 1 (TAAR1) is a member of the G protein-coupled receptor (GPCR) TAAR family, which has been shown to be enriched in the central nervous system and periphery. TAAR1 can couple to diverse G protein subtypes, including Gs, Gq and Gi. Shandong University investigators...
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
Shandong Ruzhi Biomedical Technology Co. Ltd. has synthesized compounds reported to be useful for the treatment of neurodegeneration, cardiovascular disorders and cerebrovascular disorders.
Researchers from Hefei Industrial Pharmaceutical Institute Co. Ltd. published data detailing the design and biological evaluation of novel NMDAR-GluN2B antagonists as potential candidates for the treatment of ischemic stroke.
Bioage Labs Inc. has synthesized NLRP3 inflammasome inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease and traumatic brain injury.
Jnana Therapeutics Inc. has synthesized sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of amino acid metabolism disorders, amino acid transport disorders and metabolic and nutritional genetic disorders, among others.
The autophagy process, a critical regulator of T-cell function, has been shown to control acute HIV-1 infection and play a crucial role also in HIV-1 disease pathogenesis.
Triple-negative breast cancer (TNBC) is an aggressive type of breast cancer with poor prognosis and limited therapeutic options, and it accounts for 15%-20% of female invasive breast cancers.
Palisade Bio Inc. has completed its analysis evaluating ex vivo bioactivation of PALI-2108, an orally administered, locally acting colon-specific phosphodiesterase-4 (PDE4) inhibitor prodrug in development for ulcerative colitis.
Scientists from Shanghai Jiao Tong University and affiliated organizations have discovered novel GPR183 antagonists as potential therapeutic candidates for the treatment of inflammatory bowel disease (IBD). With the aim of improving the...
Parvus Therapeutics Inc. has entered into an exclusive worldwide collaboration and option agreement with Abbvie Inc. for the development and commercialization of novel treatments for inflammatory bowel disease (IBD), utilizing Parvus’ Navacim...
Entera Bio Ltd. has announced promising pharmacokinetic (PK) results from its collaborative research combining a proprietary long-acting GLP-2 agonist developed by Opko Health Inc. with Entera’s proprietary N-Tab technology.
Nuclear factor erythroid 2-related factor 2 (NRF2) is a master regulator of cytoprotective mechanisms, and the cellular levels of this transcription factor are maintained low under normal conditions via actions of a CUL3-based E3 ligase,...
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.