On the heels of a $4.6 million series A round in December 2023, cell therapy company Rxcell Inc. is planning to raise another $15 million in 2024 to take its iPSC-derived photoreceptors to the clinic for retinitis pigmentosa and other degenerative diseases of the retina.
On the heels of a $4.6 million series A round in December 2023, cell therapy company Rxcell Inc. is planning to raise another $15 million in 2024 to take its iPSC-derived photoreceptors to the clinic for retinitis pigmentosa and other degenerative diseases of the retina.
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 pharmacokinetic (PK) and safety profile of previously reported oxysterol receptor GPR183 antagonists, structural optimization of the reference antagonist NIBR-189 was conducted.
Inmed Pharmaceuticals Inc. has released new preclinical data on INM-089 further demonstrating promising pharmacological effects targeting dry age-related macular degeneration (AMD). INM-089 is a proprietary small-molecule compound acting as a preferential signaling ligand of cannabinoid CB1 and CB2 receptors.
Geneventiv Therapeutics Inc. has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2.5 million from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH) to support development of a universal gene therapy for hemophilia A or B with or without inhibitors.
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 female patient with nephrotic syndrome caused by FSGS diagnosed when she was 27.
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.
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.
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.
With the aim of overcoming drug resistance and reducing the toxicities associated with pan-fibroblast growth factor receptor (FGFR) inhibitors, scientists from 3H Pharmaceuticals Co. Ltd. developed a highly selective and potent small-molecule inhibitor of FGFR2, 3HP-2827, to be developed for the...
Multiple myeloma (MM) stands as the second most common hematologic malignancy. Proteasome inhibitors are effective in MM, but many patients develop resistance, which is thought to be caused by mutations in the PSMB5 gene.
Researchers from Abbvie Inc. recently presented preclinical data for ABBV-706, an SEZ6-targeted topoisomerase 1 (TOP1) inhibitor antibody-drug conjugate (ADC), being developed for the treatment of small-cell lung cancer (SCLC) and other SEZ6-expressing cancers.
A research team at University of California, Los Angeles (UCLA), funded by Cancervax Inc., has created a promising new bispecific antibody vaccine for treating recurrent Ewing sarcoma.
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...
An enzyme that activates cell death could be targeted to avoid the inflammation and lung lesions caused by influenza A virus (IAV). A collaborative study demonstrated that an inhibitor of receptor-interacting serine/threonine-protein kinase 3 (RIPK3) blocked necroptosis in infected alveolar...
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.
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.
Harmony Biosciences Holdings Inc. has announced an exclusive licensing agreement with Bioprojet Pharma SAS to develop, manufacture and commercialize TPM-1116, a highly potent and selective oral orexin OX2 receptor agonist that will be evaluated for the treatment of narcolepsy and other sleep/wake...
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.
Bladder cancer is among the top 10 malignant tumors, still with a high mortality rate. There is a need for reliable biomarkers for early diagnosis of the disease. Proximity extension analysis (PEA) is a method that uses a quantitative PCR readout to analyze protein levels in plasma in longitudinal...
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,...
Asialoglycoprotein receptor 1 (ASGR1) is a transmembrane protein specifically expressed in hepatocytes that plays a key role in maintaining circulating glycoprotein homeostasis.
In a recent study published in PNAS, researchers from the University of Texas Southwestern Medical Center investigated cholesterol-mimetic compounds specifically binding and inhibiting Scap. Their final goal was to understand cholesterol...
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.