Alltrna advances tRNA-based strategy for stop codon diseases
Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU).

Alltrna advances tRNA-based strategy for stop codon diseases
Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU).
LTX-002: a pathway-focused ASO therapy for ALS
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.
Peptidream reveals milestone in Alnylam collaboration
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
Taigen in-licenses Insilico Medicine’s PHD inhibitor ISM-4808
Taigen Biotechnology Co. Ltd. and its Taigen Biopharmaceuticals subsidiary have entered into an exclusive in-licensing agreement with Insilico Medicine Inc. for ISM-4808, a prolyl hydroxylase domain (PHD) inhibitor.
Shenzhen Targetrx patents new Bcr-Abl1 degradation inducers
Shenzhen Targetrx Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a Bcr-Abl (Bcr-Abl1) kinase and its mutant targeting moiety through a linker reported to be useful for the treatment of cancer and immunological disorders.
Johnson & Johnson’s JNJ-5322 shows improved efficacy in MM
Antibodies targeting CD269 and GPRC5D have shown unprecedented clinical efficacy in the treatment of multiple myeloma (MM), but many patients still develop progressive disease. It was hypothesized that dual-targeting T-cell immunotherapies might improve the efficacy by addressing the difficulty of heterogenous target expression and preventing resistance development due to antigen escape.

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Conferences

Apollo’s APL-4098 shows potent antileukemic effects
American Society of Hematology
Apollo Therapeutics Ltd. has developed APL-4098, a small-molecule general control nonderepressible 2 (GCN2) inhibitor for the potential treatment of AML.
Astrazeneca reports data on AZD-4512 for B-cell malignancies
American Society of Hematology
Astrazeneca plc has provided data for their CD22-targeting antibody-drug conjugate (ADC) AZD-4512 under development for the treatment of B-cell malignancies, which still have significant rates of disease resistance and relapse, as well as treatment-related toxicities.
NMT.001 exerts antiepileptic effects in mice
Neurology/psychiatric
Researchers from Neumirna Therapeutics ApS have presented an anti-miR-134 ASO approach named NMT.001 for the potential treatment of drug-resistant epilepsy.
Amphista’s AMX-883 shows synergy with venetoclax in AML
American Society of Hematology
Amphista Therapeutics Ltd. has developed and presented data for AMX-883, a novel orally bioavailable bromodomain-containing protein 9 (BRD9) degradation inducer for acute myeloid leukemia (AML) treatment.

Cancer

Disco closes €36M seed round to advance surfaceome technology
Financings
Disco Pharmaceuticals GmbH announced the final close of its seed round at €36 million (US$42 million), as it shapes up novel targets it has discovered with its surfaceome platform technology for formal preclinical development.

Infection

Lipopeptides against orthoflavirus protease
Orthoflaviruses such as dengue, West Nile and Zika viruses are a threat to public health for which no specific treatments exist. Their protease NS2B-NS3, also called orthoflavivirin, is an attractive drug target because it is essential for virus maturation. Targeting viral proteases has already...

Research unpicks molecular mechanism of vaccine-induced cardiac inflammation
Science
The cardiomyositis that is a rare adverse effect of mRNA-based COVID vaccines is due to immune cell activity as a result of increased levels of the chemokines CXCL10 and interferon-γ (IFN-γ). Blocking CXCL10 and IFN-γ could prevent muscle cell damage in cell culture, and cardiomyositis in animal...
Peptidomimetic against drug-resistant HIV-1
HIV/AIDS
Many cases of human immunodeficiency virus (HIV)-1 infection can be effectively treated with existing drugs, but they can lose efficacy over time because of the emergence of resistance. In an effort to generate next-generation drugs, Chinese researchers at the Chinese Academy of Medical Sciences &...

Neurology/psychiatric

Astrocyte-to-neuron conversion rescues motor deficits in spinal cord injury
Researchers from Wuhan Institute of Biomedical Sciences, Jianghan University and collaborators recently showed that activating the endogenous transcription factors Ngn2 and Isl1 via CRISPRa can directly reprogram astrocytes into functional motor neurons in the mouse spinal cord.
Epilepsygtx's $33M advances focal epilepsy gene therapy
Science
Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.
ABS-1230 controls seizures in KCNT1-driven severe epilepsy
Conferences
Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically...
Dimerizing peptide ligands of the nociceptin receptor to strengthen agonism
Peptide
In an alternative to small molecules, researchers at the University of Ferrara and Università degli Studi di Padova sought to create an NOP agonist based on peptides, specifically dimers of peptides derived from native nociceptin.
Research grants support early clinical trials of ALS treatments
Financings
The ALS Association has announced the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate critical data to accelerate the development of new amyotrophic lateral sclerosis (ALS) treatments.

Endocrine/metabolic

Zealand Pharma and OTR Therapeutics partner in metabolic diseases
Collaboration
Zealand Pharma A/S and OTR Therapeutics (Shanghai) Co. Ltd. have signed a multi-program strategic collaboration and license agreement to discover and develop novel therapeutics for metabolic diseases.

Biomarkers

Mesothelin is biomarker, potential target in arthritic bone damage
Musculoskeletal
In a recent study published in Cell Reports Medicine, researchers from the Institute of Chinese Materia Medica of the China Academy of Chinese Medical Sciences and Xi’an Jiao Tong University Health Science Center found that Mesothelin (MSLN) was significantly elevated in rheumatoid arthritis (RA)...

UK Biobank delivers largest metabolomics dataset of 500K profiles
Science
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
‘Most complete’ map of oral microbiome enables links to systemic disease
Artificial intelligence
South Korean researchers led by Lee In-suk of Yonsei University have reported the most complete oral microbiome catalog to date, with more than 72,000 genomes. Detailed in Cell Host & Microbe on Nov. 12, 2025, the database is expected to serve as a universal platform for academia and enable...

Gastrointestinal

Aerolysin-producing Aeromonas is behind ulcerative colitis
Infection
Researchers from the Medical School of Nanjing University hypothesized that in ulcerative colitis, the gut-resident macrophages may be compromised, leading to impaired integrity of the epithelial barrier. “From a basic science standpoint, our work...
Tangram seeks clinical trial clearance for TGM-312 for MASH
Regulatory
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Opko’s OPK-88006 shows hepatoprotection in MASH
Conferences
Opko Health Inc. has recently presented data for their GLP-1/glucagon receptor dual agonist OPK-88006, which is in preclinical development for the treatment of metabolic disease, including metabolic dysfunction-associated steatohepatitis (MASH)...
UK study reveals inconsistencies in global microbiome research
Regulatory
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical patents new compounds for MASH
Patents
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex patents new NOD2 activators for Crohn’s disease
Patents
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.

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