Pandemic potential is plentiful, but the next bug’s specifics are known unknown
Compared to other forms of prevention, a unique issue for pandemic preparedness is that it is forever unclear what pathogen, exactly, the world needs to be prepared for. There are an estimated 300,000 viruses that infect mammals; add in birds, and the estimate grows to more than half a million. Some of those viruses are much greater threats than others.

Pandemic potential is plentiful, but the next bug’s specifics are known unknown
Compared to other forms of prevention, a unique issue for pandemic preparedness is that it is forever unclear what pathogen, exactly, the world needs to be prepared for. There are an estimated 300,000 viruses that infect mammals; add in birds, and the estimate grows to more than half a million. Some of those viruses are much greater threats than others.
UCB-7853 shows promise for treating Parkinson’s disease
Parkinson’s disease (PD) is a neurodegenerative disease of the central nervous system characterized by the loss of dopaminergic neurons in the substantia nigra and the abnormal aggregation of α-synuclein. UCB Pharma Inc. has presented new preclinical data on their anti-α-synuclein antibody UCB-7853 for the potential treatment of PD.
Nissan Chemical, Sanwa Kagaku Kenkyusho to codevelop antisense drug candidate
Nissan Chemical Corporation and Sanwa Kagaku Kenkyusho Co. Ltd. (SKK) have entered an agreement to codevelop SK-2407/SN-001 for the treatment of dentatorubral-pallidoluysian atrophy (DRPLA) in Japan.
Tharimmune reports data on new biparatopic biologics
Tharimmune Inc. has announced preclinical data from its expanded pipeline with HS-1940, a dual-target multispecific biologic engineered to bind to both programmed cell death protein 1 (PD-1) and vascular endothelial growth factor (VEGF), and HS-3215, a dual-target biologic binding to HER2 and HER3 receptors. Both use the proprietary Epiclick technology, a multispecific antibody engineering platform.
Newave Pharmaceutical patents new GTPase KRAS inhibitors
Newave Pharmaceutical LLC has disclosed GTPase KRAS (mutant) inhibitors reported to be useful for the treatment of cancer, autoimmune disease and inflammatory disorders.
CSPG4 expression discerns EBSCC from conventional ESCC
Esophageal basaloid squamous cell carcinoma (EBSCC) is a rare type of esophageal squamous cell cancer (ESCC) characterized by basaloid features and considered to be more aggressive than ESCC. RNA sequencing was performed from 20 pure EBSCC and 11 poorly differentiated ESCC samples; researchers identified chondroitin sulfate proteoglycan 4 (CSPG4) as a potential marker of EBSCC at the mRNA level, which was verified by immunohistochemistry in these same samples.

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Conferences

ADPD 2025: Unfolding the field of neurolipidomics
Neurology/psychiatric
Neurodegenerative pathologies, once primarily associated with protein alterations, should be revisited in the context of lipidopathies, researchers argued at the 2025 International Conference of Alzheimer’s & Parkinson’s Disease and Related Neurological Disorders (AD/PD 2025).
L1CAM predicts unfavorable outcome in malignant melanoma
Biomarkers
Malignant melanoma (MM) is among the most lethal cutaneous neoplasms, frequently tied to metastasis and poor outcomes. Neural cell adhesion molecule L1 (L1CAM) is a protein that has been associated with poor prognosis in several cancer types, but its association with MM is poorly understood.
Pfizer’s PF-07853578 is candidate to treat MASH
Endocrine/metabolic
The I148M mutation in the PNPLA3 gene, which encodes patatin-like phospholipase domain-containing protein 3, is known to confer risk of fatty liver, cirrhosis and hepatic inflammation, which may lead to hepatocellular carcinoma or metabolic dysfunction-associated steatohepatitis (MASH).
Blacksmith presents FG-2101 for gram-negative bacterial infections
New compound
Using its proprietary FBDD platform, Blacksmith discovered FG-2101, the prodrug form of FG-960, the first known non-hydroxamate LpxC inhibitor that exerted activity against LpxC at the nanomolar range.

Cancer

FDA clears ALX Oncology’s IND for ALX-2004
Antibody-drug conjugate
ALX Oncology Holdings Inc. has received FDA clearance for the IND application for ALX-2004, the company’s potential best- and first-in-class antibody-drug conjugate (ADC) for the treatment of epidermal growth factor receptor (EGFR)-expressing solid tumors.

Infection

Arrepath’s antibiotic targeting Enterobacterales receives CARB-X funding
Antibiotic
Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) will award Arrepath Inc. $3.7 million to execute a lead optimization workplan for its first-in-class antibiotic targeting a clinically novel target for the treatment of complicated urinary tract infections caused by...

Shionogi’s S-880008 shows broad range of activity against SARS-CoV-2 variants
Conferences
COVID-19 has continued to alarm public health, and although several therapeutics and vaccines have been developed, the development of effective vaccines or antibodies is challenging due to mutations in the surface of the spike protein in the SARS-CoV-2 virus.
Syndax Pharmaceuticals patents new prodrugs for viral infections
Patents
Syndax Pharmaceuticals Inc. has disclosed extended purine tricyclic and bicyclic nucleosides as prodrugs reported to be useful for the treatment of viral infections.

Neurology/psychiatric

ACD-856 TRK PAM mitigates neuroinflammation in experimental Alzheimer's
Conferences
The survival and plasticity of neurons depends on the signaling of the nerve growth factors BDNF and NGF acting through TRK receptors, which is crucial in neurological disorders such as Alzheimer’s disease (AD). Alzecure Pharma’s ACD-856 is a positive allosteric modulator (PAM) of TRK receptors...
Lilly licenses rights to use Sangamo’s neurotropic AAV capsid in genomic medicines
Drug design, drug delivery and technologies
Sangamo Therapeutics Inc. has entered into a license agreement with Eli Lilly and Co., allowing Lilly to use Sangamo’s novel proprietary neurotropic AAV capsid, STAC-BBB, to deliver intravenously administered genomic medicines to treat certain diseases of the central nervous system.
Preclinical results of SPK-10005 as a potential miRNA-based therapeutic for synucleinopathies
Conferences
Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying...
DYRK1A inhibitors for treating Alzheimer’s disease presented at ACS Spring
Conferences
Dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A) is an attractive therapeutic target due to its involvement in cancer and neurodegenerative diseases. Researchers from the National Health Research Institutes and their collaborators have presented a series of DYRK1A inhibitors...
New SARM1 inhibitors disclosed in Tenvie patents
Patents
Tenvie Therapeutics Inc. has divulged NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of glaucoma, spinal cord injury, multiple sclerosis, Niemann-Pick disease, Alzheimer’s disease, amyotrophic lateral sclerosis, stroke and diabetic neuropathy, among others.

Endocrine/metabolic

Ibio reports preclinical results for antibodies in cardiometabolic and obesity pipeline
Antibody
Ibio Inc. has announced data from a non-GLP, nonhuman primate (NHP) pharmacokinetic (PK) study of IBIO-600, the company’s lead asset and a potentially best-in-class long-acting anti-myostatin antibody designed for subcutaneous administration. Results suggest the antibody could provide a...

Biomarkers

NIPAL4 correlates with poor survival in renal cancer
Cancer
Clear cell renal cell carcinoma (ccRCC) is still a clinical challenge due to its high metastasis rate at diagnosis and therapy resistance. It accounts for about 75%-80% of RCC cases. The magnesium transporter NIPA4 (NIPAL4) may influence tumor progression, but there is lack of knowledge on its...

Rare genetic variants in HECTD2 tied to bipolar disorder
Neurology/psychiatric
A variant burden analysis for bipolar disorder was performed using gene-based aggregation of loss-of-function variants in whole-genome sequencing data from Iceland and the UK Biobank; the association between bipolar disorder and the burden of loss-of-function variants was tested in 13,786 genes.
PRTFDC1 expression predicts prognosis, drug response of testicular germ cell tumors
Cancer
Researchers from Guangdong Medical University published data from a study that aimed to identify novel biomarkers that would allow for comprehensive assessment of prognosis and immunotherapy response in patients with testicular germ cell tumors (TGCT).

Gastrointestinal

Airna’s $155M series B advances RNA editing drug AIR-001 for AATD
Financings
As it prepares to advance its lead RNA editing candidate, AIR-001, into a phase I/II trial for alpha-1 antitrypsin deficiency, Airna Corp. Inc. closed an oversubscribed $155 million series B financing less than a year after completing its series A...
Validation of 3D bioprinted h-VIOS platform for ADME-toxicology studies
Systemic Bio LLC has developed a 3D bioprinted human Vascularized Integrated Organ System (h-VIOS) platform that better recapitulates the native-like liver architecture and vascularization, integrating PHHs and non-parenchymal cells.
ABHD17B identified as a target in liver fibrosis
RNAi
Scientists at Massachusetts General Hospital have revealed how chronic liver injury alters hepatic stellate cells (HSCs), triggering the formation of fibrotic scarring in the liver. The researchers have shown that this cell type...
Unlocking early genetic drivers of stomach cancer
Cancer
Research published online in Nature on March 19, 2025, closely examines the changes occurring in the gastric epithelium during the progression toward cancer development. Certain mutations that occur in normal, nonreproductive cells over time can...
ICB treatment induces colitis-related indicators in DSS drinking mouse model
Conferences
Researchers from Chinese Academy of Sciences detailed the creation of a new dextran sulfate sodium (DSS)-based mouse model of immune checkpoint blockade (ICB) therapy-associated colitis.
Domain Therapeutics nominates biased antagonist of PAR2 as drug candidate for inflammatory diseases
Dermatologic
Domain Therapeutics SA has nominated PAR2 antagonist DT-9046 as a drug candidate with potential to treat various inflammatory diseases, including atopic dermatitis, inflammatory bowel disease and arthritis, as well as neuroinflammatory conditions...

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