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BioWorld Science

Fri, May 23, 2025
Breaking News: 100 days of uncertaintyBreaking News: 100 days of uncertaintyBreaking News: 100 days of uncertaintyBreaking News: 100 days of uncertainty
  • Silhouette of head and brain with DNA double helixes

    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

  • Avenzo gains IND clearance for CDK4 inhibitor

  • 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.
  • Novel peptide-drug conjugate achieves durable tumor retention with favorable safety profile

    Art concept for drug research
  • Chinese researchers divulge new GSPT1 degradation inducers

  • Bayer and Dana Farber describe new EGFR and/or HER2 Ex20Ins mutant inhibitors

  • PERK inhibition reverses asbestos-induced pulmonary fibrosis in mice

    Lungs
  • New Nav1.7 channel blockers for cough disclosed in Chiesi patent

  • Chengdu Easton Biopharmaceuticals discovers new MEN1/MLL interaction inhibitors

  • Dual knockout of DGK-α and -ζ enhances TAG-72 CAR T-cell antitumor efficacy in ovarian cancer

    Ovarian cancer illustration
  • BBIT-20 inhibits DNA repair to overcome resistance in pancreatic cancer treatment

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Conferences

  • Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.

    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.
  • DNA double helix under a magnifying glass

    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...
  • Photomicrograph of diffuse large B-cell lymphoma

    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.
More in Conferences

Today's news in brief

  • Respiratory

  • Dermatologic

  • Cardiovascular

  • Hematologic

  • Nephrology

Patents

  • Esperion Therapeutics discovers new ATP citrate lyase inhibitors

  • New PKCθ inhibitors disclosed in Evommune patent

  • Merck Sharp & Dohme describes new RIPK1 inhibitors

  • CSIC divulges new antiviral compounds for coronavirus acute respiratory syndrome

  • C4 Therapeutics patents new B-Raf V600E mutant degradation inducers

  • Chengdu Zeling Biomedical Technology discovers new PARP-1 inhibitors

  • New FABP inhibitors disclosed in patent

  • Roche describes new GTPase KRAS mutant inhibitors

  • Neurobiogen divulges new MAO-B inhibitors for Parkinson’s disease

  • Bristol Myers Squibb patents new BCL-6 degradation inducers

Cancer

  • Conceptual image for brain cancer treatment

    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.
  • ALX Oncology presents preclinical data, plans for ALX-2004

    Antibody-drug conjugate
  • New oncolytic virus expressing FLT3L enhances antitumor immunity by activating dendritic cells

    Immuno-oncology
  • PRGN-3008 exerts potent antitumoral activity and surpasses conventional CAR T

    Conferences
  • Rationalizing the design of styrylquinolinium derivatives to bind c-Myc G-quadruplexes in breast cancer

More in Cancer

Infection

  • Illustration of measles virus particles

    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.
  • Arrepath develops new LpxH (bacterial) inhibitors

    Patents
  • Two cores better than one: combining carboline and triazole into small molecules against malaria

  • New HIV integrase inhibitors disclosed in Gilead patent

    HIV/AIDS
  • Immunogenicity and efficacy of a bivalent self-amplifying RNA vaccine against yellow fever and Zika

    Immune
More in Infection

Neurology/psychiatric

  • Illustration of magnifying glass inspecting brain

    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.
  • Illustration of brain in head highlighting the blood-brain barier.

    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.

  • Art concept for gene therapy research

    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.
  • Illustration of human brain and dna

    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.
More in Neurology/psychiatric

Immune

  • Shenzhen Zhongge Biotechnology presents IRAK-4 degraders

  • Hitgen identifies IL17RA antagonists

  • Ensoma gains IND clearance for in vivo HSC-directed therapy for X-linked chronic granulomatous disease

  • Lipid uptake drives immune evasion in high-grade serous ovarian cancer

  • Kymera unveils new oral IRF5 degrader for immuno-inflammatory diseases

  • Liver is also immune organ, influenced by microbiome

  • Ahead Therapeutics advances myasthenia gravis program toward toxicology studies

  • WTX-921: An IL-10 Indukine designed to minimize systemic toxicity

  • Seasonal flu vaccine found to trigger a broadly protective antibody targeting neuraminidase

  • Key genes and educated natural killer cells keep HIV in remission

Endocrine/metabolic

  • High-density lipoprotein particles and red blood cells

    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).
  • TAOK1 identified as a potential therapeutic target in cancer cachexia-associated muscle atrophy

  • Validation of a fatty acid receptor agonist in a fly model of metabolic syndrome

  • Preclinical data for Wave’s INHIBE GalNAc-siRNA in obesity

    Conferences
  • Abcellera gains Canadian clearance for phase I study of ABCL-635 for vasomotor symptoms in menopause

    Regulatory
More in Endocrine/metabolic

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.
  • PSMD14: a protector protein that jeopardizes bladder cancer patients

    Cancer
  • The GTPase GIMAP8 implicated in lung adenocarcinoma

    Cancer
  • Identifying NNMT as a therapeutic target in an autoimmune eye disorder

    Ocular
  • SLC25A19 predicts prognosis in hepatocellular carcinoma

    Cancer
More in Biomarkers

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.
More in Gastrointestinal

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podcast microphone, sound waves on purple backgroundBreakthrough 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.


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  • The first half of 2024 was strong as M&As and financings dominate
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