Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later.
Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later.
Researchers from Merck & Co have presented the discovery of inhibitors of the YAP/TAZ-TEAD complex as potential anticancer agents. YAP and its paralogue TAZ act as terminal effectors of the Hippo signaling pathway by regulating the transcriptional activity of the different TEAD isoforms.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Scientists from the Australian National University have discovered the gene mutation responsible for causing psoriasis, and the findings could lead to improved diagnosis and treatment for patients with psoriasis and psoriatic arthritis, a chronic inflammatory skin disease. “We were able to identify the gene that could be important in enabling this progression from a skin-only condition to a skin-and-joint condition,” lead study author Chelisa Cardinez told BioWorld.
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
Defence Therapeutics Inc. has announced the successful testing of a second-generation anticancer vaccine, ARM-002, using its lead anticancer molecule Accutox. When tested as a therapeutic vaccine in a melanoma cancer model, ARM-002 led to an 80% complete response when combined with an anti-PD-1 immune-checkpoint inhibitor.
Ubiquigent Ltd. has signed an agreement with Debiopharm International SA to support the development of Debiopharm’s preclinical ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitor program, Debio-0432.
Cinfina Pharma Inc. has obtained FDA clearance of its IND application for CIN-110, a PYY3-36 analogue, allowing it to initiate a first in-human study in otherwise healthy subjects with obesity. CIN-110 is a stable and long-acting analogue of PYY3-36 being developed both as a monotherapy and for co-administration for obesity.
Previous research has shown that adenosine impairs antitumor immunity, and high levels of adenosine and CD73 have been associated with poor prognosis in cancer.
Researchers from Foghorn Therapeutics Inc. recently presented preclinical data for FHD-609, a heterobifunctional degrader of BRD9 designed for the treatment of sarcoma. Synthesis and optimization of a series of first-generation compounds led to the identification of FHD-609 as a rapid, selective...
One of the building blocks for newly launched Clasp Therapeutics Corp. is making the right patient choices for treatment. If those who receive the company’s therapy are correctly identified, CEO Robert Ross told BioWorld, it will have a profound effect on outcomes. The missing link in cancer...
While there has been progress in the development and availability of treatments and vaccines for Ebola virus (EBOV), there are still no treatments for other filoviruses such as Sudan virus (SUDV, species Sudan ebolavirus), which caused the 2022-23 outbreak in Uganda.
Scientists at Fujifilm Corp. and Osaka University have described piperazine derivatives acting as drug efflux pump inhibitors reported to be useful for the treatment of bacterial infections.
Researchers from Viiv Healthcare Ltd. presented preclinical data for the next-generation maturation inhibitor (MI) VH-3739937 (VH-937, zegruvirimat), currently in clinical testing for the treatment of HIV.
Nurexone Biologic Inc. has signed a strategic service agreement with Vivox Ltd. for large-scale animal testing of Exopten therapy for spinal cord injury (SCI).
To address the need for novel therapeutic candidates against Duchenne muscular dystrophy (DMD), investigators at Mitorx Therapeutics Ltd. developed a library of novel small-molecule mitochondriotropic agents.
Galectin-3 (Gal-3) is used as a marker of activated microglia, and thus a protein that drives inflammation. Patients with Alzheimer’s disease (AD) have shown increased levels of Gal-3 in their cerebrospinal fluid (CSF) compared to healthy controls, as well as increased expression in their brain...
Vaccines that target α-synuclein have a lot of potential for the treatment of Parkinson’s disease (PD) and other synucleinopathies. Tridem Bioscience GmbH & Co. KG has developed the proprietary WISIT vaccine technology.
Leucine-rich repeat kinase 2 (LRRK2), a key drug target, plays a major role in Parkinson’s disease (PD). Mutations in LRRK2, specifically LRRK2 G2019S which increases LRRK2 kinase activity, have been observed in a majority of PD patients.
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which there is a 10% rate of familial cases, with the rest being sporadic cases. Both genetic and environmental factors contribute to the etiology of ALS, and more than 120 genes have been reported to be tied to the...
Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of...
Hidradenitis suppurativa (HS) is an inflammatory skin disease with significant diagnostic delay. Type XXII collagen is a fibrillar collagen located in the skin epidermis. Reliable biomarkers to aid in the diagnosis of HS and monitor the severity of disease are needed.
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...
Everzom SAS, a CNRS/Université Paris Cité spin-off, has signed a second exclusive license agreement with Erganeo SAS for the development of Evergel, an exosome drug candidate for fistulas and fibrosis of the digestive tract.
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.