At AACR-NCI-EORTC 2025, new strategies reshape the field
During the first poster session of the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held in Boston, several presentations highlighted novel strategies that move beyond traditional antibody-drug conjugate (ADC) payloads and targets.

At AACR-NCI-EORTC 2025, new strategies reshape the field
During the first poster session of the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held in Boston, several presentations highlighted novel strategies that move beyond traditional antibody-drug conjugate (ADC) payloads and targets.
Multivalent aptamer oligonucleotide complex boosts CAR-NK efficacy
Adoptive cell therapy represents a major step forward in treating hematological cancers. Among its different approaches, chimeric antigen receptor natural killer (CAR-NK) cells are drawing growing interest.
BBOT presents preclinical data on KRAS inhibitor BBO-11818
At the ongoing AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics 2025 in Boston, Bridgebio Oncology Therapeutics Inc. (BBOT) presented data on BBO-11818, a potent and selective KRAS inhibitor with activity against several KRAS mutants both in active (ON) and inactive (OFF) forms.
JAB-23E73 shows promise as pan-KRAS inhibitor in cancer
Mutant KRAS is a well-known oncogenic driver and has remained undruggable for many decades. The development of pan-KRAS inhibitors that target a broad range of mutations is a promising approach to cancer treatment.
Gilead Sciences patents new PI3Kα inhibitors
Gilead Sciences Inc. has reported new compounds acting as phosphatidylinositol 3-kinase α (PI3Kα) inhibitors reported to be useful for the treatment of cancer.
New off-the-shelf CAR-NKT therapy shows potential against TNBC
Researchers at the University of California, Los Angeles (UCLA), have developed a new type of allogeneic immune cell therapy that demonstrated potent antitumor activity against triple-negative breast cancer (TNBC) in preclinical studies.
EED inhibitor exhibits strong efficacy across multiple prostate cancer models
Preclinical results were recently presented from studies conducted by Fulcrum Therapeutics Inc. evaluating FTX-6274, a novel, orally bioavailable embryonic ectoderm development (EED) inhibitor that targets the PRC2 complex, in models of castration-resistant prostate cancer.

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Conferences

FGF5 gene involved in excessive hair growth
Biomarkers
Hirsutism is the excessive hair growth in women in male-typical areas, usually associated with androgen excess and other conditions such as polycystic ovary syndrome (PCOS). The genetic basis of hirsutism remains poorly understood; researchers from the University of Oulu and collaborators performed...
Preclinical data on single-dose gene editing system for AATD
Endocrine/metabolic
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
New link between PRRX1 gene and open-angle glaucoma
Biomarkers
Primary open-angle glaucoma (POAG) is among the leading causes of irreversible blindness. Early detection and treatment are crucial for vision preservation, and research to identify genes associated with POAG is ongoing.
rAAV2-U1a-hELP1 rescues RGC function in familial dysautonomia
Genetic/congenital
Familial dysautonomia (FD) is an autosomal recessive neurodevelopmental disorder.

Cancer

Transcriptomics and AI for drug discovery by reading cell states
Science
A technology that combines transcriptomic data and AI enables a novel approach to drug discovery based on the state of cells, how they behave and which genes they express. The Drugreflector model, developed by scientists at Cellarity Inc., learns from gene expression profiles and predicts which...

Infection

Smartbax raises funding to advance antibiotics for MDR bacteria
Financings
Smartbax GmbH has announced the successful first closing of its €4.7 million (US$5.4 million) pre-series A financing round to support development of its next-generation antibiotics against multidrug-resistant (MDR) bacteria. A second closing of the round remains open to investors.

Combining multiple HIV epitopes elicits broad antibody responses
Drug design, drug delivery and technologies
In vaccine development, one might think that targeting multiple epitopes increases the likelihood of improving outcomes. However, when several immunogens are administered together, the immune system does not always generate antibodies against all of them. Two parallel studies have overcome this...
Novel antibiotic based on dimethylcysteamine pleuromutilin
Antibiotic
As part of global efforts to develop next-generation antibiotics to overcome the growing problem of bacterial resistance to existing drugs, researchers at the Lanzhou Institute of Husbandry and Pharmaceutical Sciences prepared various derivatives of the dimethylcysteamine form of pleuromutilin, a...

Neurology/psychiatric

Biphenyl-based M4 receptor PAMs to treat schizophrenia
Positive allosteric modulators (PAMs) of the muscarinic acetylcholine M4 receptor show potential against schizophrenia because they can enhance endogenous acetylcholine signaling, which in turn may mitigate psychotic symptoms as well as improve attention and working memory in individuals with the...
N-benzoyl tryptamine derivatives against Alzheimer’s disease
Researchers at University of South China and collaborators have generated numerous carbamate-based N-benzoyl tryptamine derivatives for Alzheimer’s disease.
Lundbeck and Contera Pharma partner in neurological disorders
Deals and M&A
H. Lundbeck A/S and Contera Pharma A/S have entered into a strategic research collaboration designed to accelerate the discovery and development of innovative oligonucleotide-based medicines for serious neurological conditions with unmet needs.
Study identifies REST as biomarker and siRNA target in ALS
Biomarkers
Restrictive element-1 silencing transcription factor (REST) has key roles in neuronal differentiation, structural remodeling and plasticity, contributing to neuronal homeostasis in postnatal neurons. It acts as a suppressor of neuronal gene expression in stem and progenitor cells, and abnormal...
Women more prone to AD even though their brains age better
Aging
Do men’s and women’s brains age equally? Women are more often diagnosed with Alzheimer’s disease (AD) than men. Age is the primary known risk factor for AD prevalence, and both aging and AD are associated with brain atrophy, but it is still not clear whether men and women differ regarding brain...

Endocrine/metabolic

Genescience Pharmaceuticals describes new PAH stabilizers for PKU
Genetic/congenital
Genescience Pharmaceuticals Co. Ltd. has identified phenylalanine hydroxylase (PAH) (R408W mutant) stabilizers reported to be useful for the treatment of phenylketonuria (PKU).

Biomarkers

miR-1265 emerges as a biomarker of meningioma
Conferences
Meningiomas are usually grade I benign tumors that can turn into aggressive grade II or III malignant tumors. Genome-wide methylation analysis has shown that microRNA 1265 (miR-1265) is hypermethylated in meningiomas.

AKR1B1 and AKR1B10 as prognostic markers in endometrial cancer
Conferences
Endometrial cancer is the most prevalent gynecological malignancy in high-income countries, and the discovery of new biomarkers may aid in earlier detection and improve the prognosis of patients.
MFAP5: Potential biomarker of interstitial lung disease
Respiratory
To identify potential biomarkers of interstitial lung disease, researchers at West China Hospital of Sichuan University and Minda Hospital of Hubei Minzu University mined the Gene Expression Omnibus database of transcriptomes as well as compared tissues across healthy individuals and patients with...

Gastrointestinal

Daewon Pharm patent reveals new H+/K+-ATPase inhibitors
Patents
Daewon Pharmaceutical Co. Ltd. has reported new compounds acting as H+/K+-ATPase inhibitors useful for the treatment of reflux esophagitis.
Proqr’s AX-0810 gains European clinical trial clearance
Regulatory
Proqr Therapeutics NV has received clinical trial application (CTA) authorization under the EMA’s new centralized review process for a phase I study of AX-0810, which is being developed for the treatment of cholestatic diseases such as primary...
New mPGES-1 inhibitors disclosed in Nippon Shinyaku patent
Patents
Nippon Shinyaku Co. Ltd. has divulged quinazoline compounds acting as microsomal prostaglandin E2 synthase-1 (mPGES-1) inhibitors reported to be useful for the treatment of irritable bowel syndrome, psoriasis, arthritis, bacterial infection,...
Salubris Pharmaceuticals divulges new MIR124 activators
Patents
Shenzhen Salubris Pharmaceuticals Co. Ltd. has synthesized compounds acting as MIR124 activators reported to be useful for the treatment of inflammatory bowel disease and inflammatory disorders.
Esperion nominates ESP-2001 as preclinical candidate for PSC
Esperion Therapeutics Inc. has nominated ESP-2001 as a preclinical development candidate for the treatment of primary sclerosing cholangitis (PSC). The company will now begin IND-enabling studies with the aim of filing an IND application with the...
Orchard’s OTL-104 is safe and efficacious in Crohn’s disease model
Conferences
NOD2 deficiency is involved in the pathogenesis of Crohn’s disease (CD) due to failure of gut innate immunity and loss of intestinal tissue homeostasis. Orchard Therapeutics Ltd. has presented preclinical data on OTL-104, human hematopoietic stem...

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