The domino effect of cutting mRNA vaccine research
In August, a press release from HHS announced the cancellation of 22 vaccine research projects based on mRNA, the latest available technology aimed at developing therapies for viral infections, cancer, and genetic conditions. What happens to mRNA innovation when funding dries up? This series explores how reductions in funding could impact mRNA technology, affecting innovation, research and future therapies.

The domino effect of cutting mRNA vaccine research
In August, a press release from HHS announced the cancellation of 22 vaccine research projects based on mRNA, the latest available technology aimed at developing therapies for viral infections, cancer, and genetic conditions. What happens to mRNA innovation when funding dries up? This series explores how reductions in funding could impact mRNA technology, affecting innovation, research and future therapies.
Merck’s MK-8527 enables long-acting HIV-1 prevention
Nucleoside reverse transcriptase translocation inhibitors (NRTTIs) are a novel class of antiretroviral agents that inhibit HIV replication by targeting the viral reverse transcriptase enzyme and specifically blocking its translocation step during DNA synthesis, a critical process in the viral replication cycle.
CSPC’s SYH-2066 gains trial clearance in China for RSV
CSPC Pharmaceutical Group Ltd.’s SYH-2066 tablets has obtained clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials for respiratory infections caused by respiratory syncytial virus (RSV).
Other news to note for Sept. 15, 2025
Additional early-stage research and drug discovery news in brief.
Selenized exosomes revealed as a novel therapy for CNS trauma
Neural stem cell-derived exosomes (Nexo) are small, membrane-bound extracellular vesicles secreted by neural stem cells. Due to their role in cell-to-cell communication within the central nervous system (CNS), they are considered a promising strategy for modulating neuronal function and promoting repair processes in conditions such as stroke, traumatic brain injury and neurodegenerative diseases. Researchers at the Chinese Academy of Sciences developed an advanced Nexo platform incorporating ultrasmall nanoselenium via lipid-mediated nucleation, termed Senexo.
Rezubio patents drug conjugates for type 2 diabetes
Rezubio Pharmaceuticals Co. Ltd. has disclosed drug conjugates comprising somatostatin receptor type 5 (SSTR5) antagonists covalently linked to a hydrophilic moiety through a linker reported to be useful for the treatment of type 2 diabetes.

Conferences

IAM-1363 outperforms standards in HER2+ NSCLC models
Cancer
Iambic Therapeutics Inc. presented preclinical data on IAM-1363, a selective and irreversible HER2 and pan-HER2 mutant inhibitor in HER2-driven NSCLC models. In vitro studies across a panel of HER2-altered NSCLC cell lines demonstrated that IAM-1363 exhibits potent antiproliferative activity in...
Vantictumab reverses bone defects in mice with osteopetrosis
Genetic/congenital
At the American Society for Bone and Mineral Research (ASBMR) in Seattle, researchers from Ashibio Inc. reported preclinical efficacy data on vantictumab, a human monoclonal IgG2 lambda antibody that binds to multiple frizzled (FZD) receptors.
Sangamo presents epigenetic therapy for small fiber neuropathy
Neurology/psychiatric
Small fiber neuropathy (SFN) is a chronic and debilitating condition caused by damage to peripheral sensory nerves, which are responsible for pain, temperature sensation and autonomic functions. Current treatments are limited, highlighting the need for better therapies.
CREB inhibitor protects rodents from PAH
European Society of Cardiology
Pulmonary arterial hypertension (PAH) is a condition that may lead to right heart dysfunction. Previous evidence has tied mitochondrial dynamics with the progression of PAH, but the mechanisms behind this are not well elucidated.

Cancer

Series D financing at Dewpoint to advance c-mod DPTX-3186
Financings
Dewpoint Therapeutics Inc. has closed its series D financing to advance DPTX-3186, a first-in-class condensate modulator (c-mod), into clinical trials by year-end and deliver early clinical proof of concept in gastric cancer by the end of next year.

Infection

Lasker Awards honor the fine compartmentalization of life
Drug design, drug delivery and technologies
A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and...

Blacksmith Medicines patent details bacterial LpxC inhibitors
Patents
Work at Blacksmith Medicines Inc. has led to the identification of new bacterial UDP-3-O-(R-3-hydroxymyristoyl)-N-acetylglucosamine deacetylase (LpxC) inhibitors reported to be useful for the treatment of Pseudomonas aeruginosa infection, lung diseases and infectious pneumonia.
Oregon Health & Science University and Washington University patent new antiviral agents
Patents
Oregon Health & Science University and Washington University have jointly developed new tetrahydrobenzothiophene and tetrahydropyridothiophene derivatives reported to be useful for the treatment of flavivirus and alphavirus infections.

Neurology/psychiatric

Arrowhead files to begin clinical trials of ARO-MAPT
Regulatory
Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.
Grant supports Nanonewron’s TNF-α inhibitors for Alzheimer’s
Grant
Nanonewron Inc. has been awarded a $2.5 million NIH STTR phase II grant to support development of its TNF-α inhibitor NN-840 program for Alzheimer’s disease and other neurodegenerative conditions. The company aims to submit an IND application next year.
Neuronal lipid metabolism plays role in ABCA7 Alzheimer’s risk
Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.
Loss of PPM1M function may contribute to Parkinson’s disease
Mutations that activate phosphorylation of certain Rab GTPases by LRRK2 have been linked to a subset of genetic cases of Parkinson’s disease, and PPM1M is one of the phosphatases responsible for reversing phosphorylation by LRRK2.
MAPT-directed miRNA broadly reduces tau pathology in Alzheimer’s
Gene therapy
Researchers at Sanofi SA have developed a promising gene therapy approach targeting the microtubule-associated protein tau (MAPT) for the treatment of Alzheimer’s disease.

Endocrine/metabolic

Remedium and Lilly partner on gene therapies for obesity and diabetes
Collaboration
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.

Biomarkers

CTRP7 predicts response to vasodilators in PAH
Conferences
Pulmonary arterial hypertension (PAH) is a life-threatening disease; vasodilators may aid in managing this condition, but their impact on prognosis is still limited, potentially due to a lack of biomarkers to guide therapy. Japanese researchers have presented results of their efforts to discover...

Genes for prognosis after oral squamous cell carcinoma treatment
Cancer
Researchers at Seoul National University and the National Cancer Center examined public datasets from patients as well as the whole-exome sequence from one of their own patients, who developed radiation-induced sarcoma after treatment.
Boost for Minovia Therapeutics’ mitochondrial biomarkers
Financings
Minovia Therapeutics Ltd. has announced a $350,000 grant from Countdown for a Cure for a research proposal related to the development of novel mitochondrial blood-based biomarkers.

Gastrointestinal

NIH support for Lactiga’s sIgA antibody for IBD
Financings
Lactiga US Inc. has received an award from the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to advance the development of its secretory IgA (sIgA) antibody for inflammatory bowel disease (IBD).
Multiomics reveals GPR31 as a druggable target for MASH
Metabolic dysfunction-associated steatotic liver disease (MASLD) now affects over a third of adults worldwide. Its progressive form, metabolic dysfunction-associated steatohepatitis (MASH), is a leading driver of cirrhosis, cancer and liver...
Omass and Genentech partner in inflammatory bowel disease
Deals and M&A
Omass Therapeutics Ltd. has entered into an exclusive collaboration and license agreement with Genentech Inc., a member of the Roche Group, for the rights to develop and commercialize Omass’ preclinical oral small-molecule program for inflammatory...
Emory University and Hadasit discover new ROR-α agonists
Patents
Scientists at Emory University and Hadasit Medical Research Services and Development Ltd. have described nuclear receptor ROR-α agonists reported to be useful for the treatment of liver cancer, pancreatitis, diabetes, obesity, immunological...
New GLP-2R agonists disclosed in Sciwind patent
Patents
Beijing Sciwind Biotechnology Co. Ltd. and Sciwind Biosciences Co. Ltd. have divulged glucagon-like peptide 2 receptor (GLP-2R) agonists reported to be useful for the treatment of Crohn’s disease, irritable bowel syndrome, malnutrition, ulcerative...
Gilead details discovery of oral integrin inhibitor GS-1427
Conferences
Gilead Sciences Inc. recently discussed the discovery of emvistegrast (GS-1427), a potent, selective, once-daily oral α4β7 integrin inhibitor currently in phase II trials for the treatment of inflammatory bowel disease (IBD) (NCT06290934).

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