New biomarker may improve RSV drug screening
Respiratory syncytial virus (RSV) infection is responsible for 3.2 million hospital admissions and about 118,000 deaths in children under 5 years of age. The increased risk of RSV infection (and other infectious diseases) in young children is broadly attributed to their immature immune system. But researchers from University College London have published work suggesting that this is not the only reason.

ALN-5288 lowers tau levels in preclinical models
Pathological tau accumulation is a key driver of neurodegeneration in Alzheimer’s disease (AD) and other tauopathies such as progressive supranuclear palsy. Researchers at Alnylam Pharmaceuticals and Regeneron Pharmaceuticals Inc. presented preclinical efficacy data on ALN-5288 in tauopathy models.

New biomarker may improve RSV drug screening
Respiratory syncytial virus (RSV) infection is responsible for 3.2 million hospital admissions and about 118,000 deaths in children under 5 years of age. The increased risk of RSV infection (and other infectious diseases) in young children is broadly attributed to their immature immune system. But researchers from University College London have published work suggesting that this is not the only reason.
ALN-5288 lowers tau levels in preclinical models
Pathological tau accumulation is a key driver of neurodegeneration in Alzheimer’s disease (AD) and other tauopathies such as progressive supranuclear palsy. Researchers at Alnylam Pharmaceuticals and Regeneron Pharmaceuticals Inc. presented preclinical efficacy data on ALN-5288 in tauopathy models.
Valerio to collaborate with Abivax, outlines pipeline progress
Valerio Therapeutics SA has entered into a binding term-sheet with Abivax SA setting out the principal terms of an exclusive worldwide research and development collaboration to identify, design and evaluate next-generation therapeutic candidates for immune-mediated inflammatory diseases.
Mirador licenses Kira’s long-acting anti-C5a antibody KP-301
Mirador Therapeutics Inc. has entered into an exclusive worldwide license agreement with Kira Pharmaceuticals for KP-301, a long-acting anti-C5a antibody with potential to treat ANCA-associated vasculitis.
Inxmed discloses new FAK inhibitors
Inxmed (Nanjing) Co. Ltd. has divulged focal adhesion kinase (FAK) inhibitors that are potentially useful for the treatment of cancer.
New indole-based hybrids emerge as resistance-resilient antimalarial leads
Indole-based compounds have been explored as antimalarial agents because they can disrupt the Plasmodium cell cycle. In a recent study, researchers from the University of São Paulo and the Federal University of São Carlos reported the evaluation of scaffolds selected from three classes of indole hybrids against Plasmodium falciparum.

Conferences

Dual FAAH/MAGL inhibition targets Alzheimer’s disease hyperexcitability
Neurology/psychiatric
Reduced endocannabinoid signaling has been linked to neuronal hyperexcitability in Alzheimer’s disease (AD), suggesting that dual inhibition of fatty acid amide hydrolase (FAAH) and monoacylglycerol lipase (MAGL) may restore endocannabinoid tone and alleviate hyperexcitability-driven symptoms,...
PSB-229 reduces plaque burden in Alzheimer’s models
Neurology/psychiatric
Pyroglutamate-modified amyloid-β (pE3-Aβ) is a highly pathogenic Aβ species that accumulates within amyloid plaques and contributes to aggregation, neuroinflammation and neuronal dysfunction in Alzheimer’s disease (AD). Researchers from Sound Biologics (Qilu Puget Sound Biotherapeutic Corp.)...
HMB-003 outperforms tranexamic acid in fibrinolysis models
International Society on Thrombosis and Haemostasis
Excessive fibrinolysis is a major driver of bleeding across multiple clinical settings, including heavy menstrual bleeding and other hemorrhagic disorders. Researchers from Hemab ApS presented preclinical data on HMB-003, a long-acting plasmin inhibitor that directly targets the enzyme’s active...
New FTO inhibitor shows neuroprotective effects
Neurology/psychiatric
FTO is an α-ketoglutarate-dependent RNA demethylase that regulates N6-methyladenosine (m6A), one of the most abundant epitranscriptomic modifications in mammalian mRNA. Emerging evidence suggests that aberrant m6A signaling may contribute to neuronal dysfunction and neurodegeneration. Researchers...

Cancer

Tempest advances next-generation in vivo CAR T pipeline
CAR T
Tempest Therapeutics Inc. has announced details of its pipeline of in vivo CAR T product candidates differentiated by a CD7-targeted mRNA/LNP delivery approach, with application in the fields of oncology and immunology. Tempest’s in vivo CAR T platform, CD7-targeted mRNA lipid nanoparticle...

Infection

New research identifies EBV antigen targets for MS therapies
Science
New research has teased out specific aspects of how Epstein-Barr virus (EBV) sparks the immune response that leads on to the development of multiple sclerosis (MS), opening the way to the rational design of vaccines and antivirals that address the root cause of the disease.

CNRS and collaborators discover new Mycobacterium ATPase inhibitors
Patents
Researchers from the Centre National de la Recherche Scientifique, the Institut National de la Santé et de la Recherche Médicale, Nanyang Technological University and the University of Montpellier have patented new Mycobacterium sp. ATP synthase (F0F1 ATPase) inhibitors potentially useful for the...
Preclinical data support Evaxion’s platform behind CMV vaccine candidate
Conferences
Human cytomegalovirus (CMV) is a clinical challenge in infants and immunocompromised individuals, since no licensed vaccine exists. Vaccine development is complex due to several factors, including viral latency, genomic complexity and immune evasion, among others. Evaxion A/S recently presented...

Neurology/psychiatric

Voyager’s gene therapy reduces tau levels in AD models
Conferences
At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD).
New model explores peripheral α-Syn’s impact on brain
Conferences
Aggregated α-synuclein (α-Syn) is encoded by the SNCA gene and is one of the key pathological proteins linked to Parkinson’s disease. However, it is still not clear whether or not α-Syn originating in the periphery has a role in the pathology in the brain.
Novel analgesics targeting adenosine A3A receptor show promise
Despite multiple analgesics on the market, current therapeutics often fail at achieving complete pain relief and are usually tied to undesired adverse effects. Recent studies have pointed to adenosine A3 receptor (A3AR) as a promising target for pain management.
GDF5 overexpression targets MuSCs, emerging as potential DMD therapy with AAV-microdystrophin
Genetic/congenital
Duchenne muscular dystrophy (DMD) is a progressive, genetic (X-linked recessive) neuromuscular disorder caused by mutations to the DMD gene, resulting in the dysfunction or absence of the dystrophin protein. In DMD, muscle regeneration initially depends on the proliferation and differentiation of...
Niagen’s NB-4168 designated orphan drug in EU
Regulatory
Niagen Bioscience Inc.’s proprietary lead small-molecule drug candidate, NB-4168, has been awarded European orphan drug designation and U.S. rare pediatric disease designation for the treatment of ataxia telangiectasia.

Endocrine/metabolic

Fosun cleared to conduct clinical trials with FXR-0906
Regulatory
Fosun Pharma subsidiary Fosun Pharmaceutical Industrial Development (Shenzhen) Co. Ltd. has received approval from China’s National Medical Products Administration (NMPA) to commence clinical trials for FXR-0906 for the treatment of hypertriglyceridemia.

Biomarkers

Blood-based circRNAs for early detection and monitoring of Alzheimer’s
Diagnostics
In a recent study, researchers from Washington University School of Medicine and collaborators analyzed cross-sectional blood samples to identify circRNAs associated with Alzheimer’s disease (AD) clinical status, amyloid-β and tau stages, and progression to symptomatic AD.

Case report links APOLD1 variant to vascular-type bleeding disorder
Conferences
Vascular-type bleeding disorder (BDVAS) is a rare, autosomal dominant disorder mainly caused by impaired vascular integrity.
KANSL2 is oncogene and actionable biomarker in multiple myeloma
Cancer
Due to the availability of drugs targeting histone acetylation and associated reader proteins, the equilibrium of histone acetylation and deacetylation has attracted attention in multiple myeloma as a potential therapeutic target. Therefore, the identification of novel predictive biomarkers for...

Gastrointestinal

U&S Bio identifies new H+/K+-ATPase inhibitors
Patents
U&S Bio Co. Ltd. has divulged H+/K+-ATPase inhibitors with improved stability found to be potentially useful for the treatment of gastrointestinal disorders, cancer, Helicobacter pylori infection, sialorrhea, asthma and airways obstruction, among...
Ribocure and Madrigal nominate siRNA drug candidate for MASH
Endocrine/metabolic
Ribocure Pharmaceuticals AB, a subsidiary of Suzhou Ribo Life Science Co. Ltd., and Madrigal Pharmaceuticals Inc. have achieved the first drug candidate nomination within their siRNA partnership.
Trex Bio discloses TRB-071 as CD30 agonist antibody
Monoclonal antibody
Trex Bio Inc. has disclosed that its development candidate TRB-071, currently in IND-enabling studies, is a CD30 agonist antibody. The approach could have therapeutic potential in inflammatory bowel disease and other autoimmune and inflammatory...
Pharmgen Science characterizes new H+/K+-ATPase inhibitors
Patents
Pharmgen Science Inc. has patented H+/K+-ATPase inhibitors that are potentially useful for the treatment of asthma, esophagitis, Helicobacter pylori infection, gastroesophageal reflux disease, gastritis, peptic ulcer and more.
New RIPK1 inhibitor protects from hepatic fibrosis
New compound
Receptor-interacting protein kinase 1 (RIPK1) acts as a central signaling node regulating apoptosis, necroptosis and inflammatory pathways. Researchers from China Pharmaceutical University reported the discovery and preclinical characterization of...
Zealand Pharma reports IL-23R antagonists
Patents
Zealand Pharma A/S has identified new interleukin-23 receptor (IL-23R) antagonists potentially useful for the treatment of Crohn’s disease, ulcerative colitis, psoriasis and psoriatic arthritis.

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