Newco Outrun Therapeutics Ltd. has raised $10 million in a seed round to develop small-molecule E3 ubiquitin ligase inhibitors that prevent programmed protein degradation. The lead program targeting an E3 ligase that tags a tumor suppressor protein for destruction will now advance to preclinical development.
Newco Outrun Therapeutics Ltd. has raised $10 million in a seed round to develop small-molecule E3 ubiquitin ligase inhibitors that prevent programmed protein degradation. The lead program targeting an E3 ligase that tags a tumor suppressor protein for destruction will now advance to preclinical development.
Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.
Ipsen SA and Skyhawk Therapeutics Inc. have entered an exclusive worldwide collaboration to discover and develop small molecules that modulate RNA for rare neurological diseases. Skyhawk has a unique platform that accelerates building RNA-targeting small molecules across several therapeutic areas.
Researchers at CNCCS Scarl (Collezione Nazionale Dei Composti Chimici E Centro Screening) and IRBM SpA have disclosed tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP2) inhibitors reported to be useful for the treatment of cancer, Noonan and Leopard syndrome.
The generation of pathogenic autoantibodies is a crucial event in the development of inflammation and complement activation, leading to immune cell responses.
In tumors with amino acid deprivation, eIF-2α kinase GCN2 is activated and triggers a signaling response to promote cell survival and proliferation. This is important in high metabolically active hematological cancers, such as acute myeloid leukemia (AML).
AR-V7 is the most clinically relevant androgen receptor (AR) splice variant associated with endocrine resistance and poor prognosis in patients with prostate cancer.
Steroid-resistant nephrotic syndrome (SRNS) is a disease characterized by hypoalbuminemia, proteinuria, edema and hyperlipidemia, and a cause of chronic kidney disease in the pediatric population.
Researchers from Zhuhai Grit Biotechnology Co. Ltd. recently presented preclinical characterization of a new genetically modified tumor-infiltrating lymphocyte (TIL) product, GT-216, being developed for the treatment of solid tumors.
Researchers from Biocytogen Pharmaceuticals (Beijing) Co. Ltd. presented the development of a humanized VEGFA model (B-hVEGFA mice) as a new tool for in vivo testing of VEGFA-targeting therapeutics.
Cross-talk between macrophages and tumor cells could modulate cachexia in pancreatic cancer patients. A group of scientists from the University of Oklahoma has discovered a new pathway that promoted muscle wasting after the recruitment of this immune cell in the tumor microenvironment, activating...
YS Biopharma Co. Ltd. has received clinical trial approval by the Philippine Food and Drug Administration to begin a phase I trial of its YS-HBV-002 immunotherapeutic vaccine, designed to treat chronic hepatitis B virus (HBV) infection. The trial will begin in the Philippines in June.
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.
Biointervene Inc. has described adenosine analogues acting as adenosine A3 receptor (ADORA3) agonists reported to be useful for the treatment of mild cognitive impairment, diabetic neuropathy, irritable bowel syndrome, neurodegeneration, ototoxicity, neuropathic pain, chemotherapy-induced...
Neurocrine Biosciences Inc. has synthesized vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of hyperkinesia and psychosis.
University of California Oakland has described potassium channel subfamily K member 2 (TREK-1; KCNK2) and/or potassium channel subfamily K member 10 (TREK2; KCNK10) activators reported to be useful for the treatment of Caisson disease (decompression sickness), depression, headache, pulmonary...
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.
Unknown etiology is commonly encountered in the kidney pre-transplant routine program. A screening program was performed to detect patients and study recipients that meet the following features: hypertension with no clear etiology and biopsies that do not match with clinical features of classical...
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...
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.
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...
Metabolic dysfunction-associated steatotic liver disease (MASLD), previously known as nonalcoholic fatty liver disease, shows different occurrence between sexes, being less prevalent in premenopausal women than in men or postmenopausal women.
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.
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.