Armamentarium, the new genetic weapon to study brain disorders
A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.

Armamentarium, the new genetic weapon to study brain disorders
A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory.
KT-621: a first-in-class STAT6 degrader for Th2-driven diseases
STAT6 plays a central role in regulating Th2-driven immune responses. Recent studies have identified gain-of-function mutations in the STAT6 gene that are associated with early-onset, severe allergic diseases. As a result, STAT6 has emerged as a promising therapeutic target in conditions such as asthma, eosinophilic inflammation, food allergies and atopic dermatitis, particularly in cases that are refractory to standard therapies.
Avenzo gains IND clearance for CDK4 inhibitor
Avenzo Therapeutics Inc. has gained IND clearance from the FDA for AVZO-023 (formerly ARTS-023), a CDK4-selective inhibitor. The company also announced it has exercised its exclusive option for AVZO-023 from Allorion Therapeutics Inc., securing global (excluding Greater China) development, manufacturing and commercialization rights.
Astrazeneca’s AZD-6793 arises as a promising therapeutic for COPD
Chronic obstructive pulmonary disease (COPD) is among the most leading causes of death around the world and there are insufficient treatment options that prevent exacerbations or alter the progression of the disease. COPD is a complex disease with multiple factors driving inflammation, emphysema or small airway remodeling, among others, where interleukin-1 receptor-associated kinase 4 (IRAK-4) plays a crucial role in the pathogenesis of the disease.
Aurigene Oncology patents new SMARCA2 and SMARCA4 degradation inducers
Aurigene Oncology has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase protein binding moiety covalently linked to probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) and/or transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β) binding moieties through a linker.
Ascletis Pharma’s oral IL-17 inhibitor cleared to enter clinic for plaque psoriasis
Ascletis Pharma Inc. has announced IND clearance by the FDA for a phase I trial of ASC-50 for the treatment of mild to moderate plaque psoriasis. Dosing is expected to start in the third quarter of this year.

Conferences

Huidagene’s HG-303 improves neuromuscular function in preclinical ALS
Neurology/psychiatric
Huidagene Therapeutics Co. Ltd. has presented data for HG-303, a new CRISPR-hfCas12Max-based therapeutic approach that knocks down ATXN2 expression for the treatment of amyotrophic lateral sclerosis (ALS).
Zymeworks’ bispecific antibody relieves airway inflammation in asthma and COPD models
Respiratory
Chronic obstructive pulmonary disease (COPD) is a prevalent and heterogeneous respiratory disorder with limited effective treatments. IL-33 and IL-4Rα are key mediators of airway inflammation in COPD and hence represent potential therapeutic targets.
Base and prime editions to achieve genetic cure
Drug design, drug delivery and technologies
Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene...
MALT1 inhibitor SY-12696 proves efficient for treating BTKi-resistant DLBCL
New compound
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is an important factor from a complex downstream of Bruton tyrosine kinase (BTK) in the B-cell receptor (BCR) signaling pathway.

Cancer

miR-1290 predicts seizure susceptibility in glioblastoma patients
Biomarkers
Glioblastoma (GBM) is an aggressive cancer from the CNS usually characterized by a very bad prognosis. It is known that around 30%-35% of patients with GBM develop epilepsy as a comorbidity of the disease.

Infection

Invivyd initiates measles monoclonal antibody discovery program
Immune
Invivyd Inc. has initiated a discovery program for a measles monoclonal antibody (mAb).

ABI-4334 outperforms vebicorvir in HBV suppression
Conferences
Current therapies for chronic hepatitis B virus (HBV) infection can effectively suppress viral replication but do not achieve a functional or complete cure. Capsid assembly modulators inhibit the assembly of viral capsids, prevent the encapsidation of pregenomic RNA, and interfere with both the...
US researchers patent new H+/K+-ATPase inhibitors
Patents
The Research Foundation of State University of New York and the U.S. Department of Health and Human Services have jointly developed prazole-based compounds acting as H+/K+-ATPase inhibitors and thus reported to be useful for the treatment of viral infections.

Neurology/psychiatric

Toward in vivo imaging of α-synuclein aggregates using azocoumarin derivatives
Diagnostics
Being able to detect and monitor the aggregation of α-synuclein in situ could lead to more objective, earlier diagnosis of Parkinson’s disease as well as allow real-time monitoring of whether patients are responding to treatment.
App- or Rab5-targeting ASOs reverse Down syndrome-linked Alzheimer’s disease effects in mouse model
Antisense
Down syndrome (DS) is the most prevalent genetic cause of Alzheimer’s disease (AD). Previous evidence suggests that increased dosage of the amyloid precursor protein (APP) gene plays a crucial role in AD in individuals with Down syndrome (DS-AD), making APP expression a crucial therapeutic target.
An adeno-associated virus to deliver therapeutic genes efficiently to the brain
Drug design, drug delivery and technologies
It is far easier and safer to inject drugs into veins than directly into the brain, yet it is extremely difficult for systemically delivered drugs to cross the blood-brain barrier and achieve therapeutic concentrations there.
Caspida’s CAP-004 exhibits potential as a best-in-class therapy for Friedreich’s ataxia
Conferences
Capsida Biotherapeutics Inc. has reported development of a systematically administered capsid, CAP-004.
Quralis signs agreements to further fragile X syndrome research
Collaboration
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.

Endocrine/metabolic

Repair Biotechnologies’ REP-0003 designated orphan drug for homozygous familial hypercholesterolemia
Regulatory
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).

Biomarkers

Targeting B4GALT5 to boost tumor antigen display in pancreatic ductal adenocarcinoma
Cancer
Pancreatic ductal adenocarcinoma (PDAC) accounts for more than 90% of cases of pancreatic cancer, and prognosis for PDAC remains poor despite treatment advances. One reason is that PDAC downregulates the display of antigens on the surface of tumor cells, helping it evade the patient’s immune system...

RGS14: An endogenous inhibitor of G protein-coupled receptors that promotes hepatocellular carcinoma
Cancer
Hepatocellular carcinoma (HCC), which accounts for up to 80% of cases of primary liver cancer, is typically diagnosed in an advanced stage, meaning a poor prognosis. Understanding what drives progression may help identify proteins and pathways that can be targeted to slow down the disease.
GPR171 is a prognostic marker in cervical cancer
Conferences
Recent evidence has shown that G protein-coupled receptor 171 (GPR171) may be a key T-cell checkpoint in tumor immunity.

Gastrointestinal

Identification of a deuterated TNF-α inhibitor with potential for treating ulcerative colitis
Tumor necrosis factor-α (TNF-α) is a key cytokine involved in the pathogenesis of ulcerative colitis (UC), a chronic inflammatory bowel disease, and has emerged as a promising therapeutic target.
N4 Pharma completes in vivo study of siRNA and mRNA-loaded oral Nuvec formulations
Drug design, drug delivery and technologies
N4 Pharma plc has successfully completed the first in vivo study of orally delivered Nuvec using its lead program N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD).
ASGCT 2025: Gene and cell therapies transform metabolic diseases
Conferences
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments...
GLP-1/GDF15 dual agonist reduces inflammation and fibrosis in MASH
Conferences
Beijing QL Biopharmaceutical Co. Ltd. has presented preclinical data on their GLP-1/GDF15 dual agonist QL-1005 for the potential treatment of inflammation and fibrosis in murine models of metabolic dysfunction-associated steatohepatitis (MASH) and...
Korean researchers patent new autotaxin inhibitors
Patents
Scientists at Arontier Co. Ltd. and Korea Research Institute of Chemical Technology have disclosed ectonucleotide pyrophosphatase/phosphodiesterase 2 (ENPP2; autotaxin) inhibitors reported to be useful for the treatment of hepatic steatosis,...
Plant compound PEITC may mitigate both oxidative stress and inflammation in ulcerative colitis
Ulcerative colitis, one type of chronic inflammatory bowel disease, is driven by a form of programmed cell death called pyroptosis.

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