SCAN is core circuit affected in Parkinson’s disease
Parkinson’s disease (PD) is a neurodegenerative disorder that affects movement, and tremor is one of its signatures. But it is a much more wide-ranging disorder, and patients experience problems with cognitive and emotional processes as well. SCAN, the somato-cognitive action network identified in 2023, could reshape the definition of PD. Treating this circuit can improve outcomes.

ALTx formed to drug pathway by which cancer cells become immortal
Newco ALTx Therapeutics Ltd. has launched with a £12.55 million (US$17.1 million) seed round to develop inhibitors of the alternative lengthening of telomeres (ALT) pathway, by which 10% to 15% of cancers become immortal.

SCAN is core circuit affected in Parkinson’s disease
Parkinson’s disease (PD) is a neurodegenerative disorder that affects movement, and tremor is one of its signatures. But it is a much more wide-ranging disorder, and patients experience problems with cognitive and emotional processes as well. SCAN, the somato-cognitive action network identified in 2023, could reshape the definition of PD. Treating this circuit can improve outcomes.
ALTx formed to drug pathway by which cancer cells become immortal
Newco ALTx Therapeutics Ltd. has launched with a £12.55 million (US$17.1 million) seed round to develop inhibitors of the alternative lengthening of telomeres (ALT) pathway, by which 10% to 15% of cancers become immortal.
Insilico Medicine selects NLRP3 inhibitor preclinical candidate
Insilico Medicine Cayman Topco has nominated ISM-5059, a peripherally restricted small-molecule NLRP3 inhibitor, as a preclinical candidate.
Astellas’ ASP-2246 shows promise in stroke management
Astellas Pharma Inc. recently presented data regarding ASP-2246, a drug candidate comprised of an mRNA encoding the transcription factor NeuroD1 encapsulated in lipid nanoparticles.
Nb20 nanobody reverses depressive-like behavior in mice
Researchers from the University of Miami Miller School of Medicine and collaborators described the preclinical efficacy of Nb20.
Tangram’s TGM-148 demonstrates pan-bleeding disorder therapeutic potential
Researchers at Tangram Therapeutics Inc. have presented preclinical safety and efficacy data for TGM-148 in a model of von Willebrand disease.
Jeil Pharmaceutical patents PDE4 inhibitors
Jeil Pharmaceutical Co. Ltd. has disclosed dihydrothiopyranopyrimidine compounds acting as phosphodiesterase PDE4 inhibitors reported to be useful for the treatment of inflammatory joint disease, neurological disorders and more.

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Conferences

GLUT5 identified as a target in ischemic stroke
Cardiovascular
Solute carrier family 2, member 5 (GLUT5) is known to be upregulated in metabolic disorders and cancer, but its potential role in ischemic stroke is not well defined. Japanese researchers have now explored the association of GLUT5 expression with oxidative stress in ischemic stroke.
KLF15 shows potential as marker, target for cholangiocarcinoma
EASL
Researchers at the Biodonostia Health Research Institute reported on the role of KLF15 in cholangiocarcinogenesis and its potential as a therapeutic target in this disease.
Serotonin receptor modulators for the treatment of OUD
Substance use and poisoning
Opioid use disorder (OUD) causes high morbidity and mortality rates, with fentanyl driving unprecedented overdose rates. Researchers from the University of California have used an AI-based drug discovery platform with the aim of identifying preclinical drug candidates for OUD.
Study identifies CAND1 as therapeutic target in hepatoblastoma
EASL
Hepatoblastoma is the most common liver cancer during childhood, with limited therapeutic options in aggressive or relapsed cases. NEDDylation is a post-translational modification that modulates cullin-RING ligases and has arisen as a crucial regulator of protein turnover in cancer.

Cancer

siRNA-mediated targeting of IRS2 in TNBC optimized with albumin-binding dendrimers
RNA
Effective targeted therapies against aggressive breast cancer subtypes, such as triple-negative breast cancer (TNBC), are still lacking. Developing therapeutics targeting nonenzymatic, intracellular proteins with causal roles in TNBC progression remains a significant challenge.

Infection

A humanized mouse mimics Fcγ receptor biology in antibody assays
Immune
Scientists at Ghent University have created a mouse model that incorporates human versions of the receptors that recognize the fragment crystallizable (Fc) region of immunoglobulin G (IgG), one of the most abundant antibodies in the blood and a key mediator of essential immune functions such as...

Intranasal adenoviral H5N1 vaccine protects naive and previously vaccinated rodents
Immune
A recent publication in Cell Reports Medicine from researchers at the Washington University School of Medicine and the La Jolla Institute for Immunology presents a promising new strategy for H5N1 vaccination.
BARDA offers $100M in prize money for antiviral development
Regulatory
Looking to fill a treatment gap, the U.S. Biomedical Advanced Research and Development Authority (BARDA) is launching the first stage of a $100 million prize competition to support development of broad-spectrum, small-molecule antiviral therapies targeting viruses in the Togaviridae and Flaviviridae...

Neurology/psychiatric

New ASOs achieve multigene silencing in cellular models of Alzheimer’s
Researchers from the University of Oxford and the Health Research Institute La Fe (Spain) investigated the potential of multigene RNA-based therapeutics in Alzheimer’s disease, aiming to overcome potential compensatory mechanisms and patient heterogeneity.
Biocells’ BXOS-110 confers neuroprotection in ischemic stroke
Cardiovascular
Acute ischemic stroke still represents a public health challenge due to its high incidence and mortality rate. Current treatments may effectively restore the blood flow, but recanalization by opening occluded vessels does not guarantee a favorable prognosis.
Shenzhen Hanlin Biomedical Technology reports urolithin A derivatives
Patents
Shenzhen Hanlin Biomedical Technology Co. Ltd. has identified new urolithin A derivatives. They are reported to be useful for the treatment of neurological disorder, aging, muscle disorder and metabolic diseases.
Nav1.8 blockers revealed in Alicorn Pharmaceutical patent
Patents
Work at Alicorn Pharmaceutical Co. Ltd. has led to the synthesis of new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, respiratory and neurological disorders.
Connecta Therapeutics discloses 3-phenoxy-3-phenylpropanamine derivatives
Patents
Connecta Therapeutics SL has discovered 3-phenoxy-3-phenylpropanamine derivatives described as potentially useful for the treatment of fragile X and Rett syndrome.

Endocrine/metabolic

Cyclodextrin-based therapy improves outcomes in Krabbe disease models
Krabbe disease, also called globoid-cell leukodystrophy, is caused by a deficiency of the lysosomal enzyme β-galactosylceramidase (GALC), resulting from pathogenic variants in both copies of the GALC gene. The GALC deficiency leads to increased levels of the primary galactosphingolipid substrates,...

Biomarkers

MJFF grant supports Bexorg’s translational biomarker work
Financings
Bexorg Inc. has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) through the Targets to Therapies initiative to identify translational biomarkers for TRPML1-targeted therapies.

REST emerges as biomarker in ALS, knockdown improves ALS symptoms
Neurology/psychiatric
About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).
MicroRNAs in circulating extracellular vesicles as IPF biomarkers
Inflammatory
MicroRNAs (miRNAs) are small noncoding RNAs gaining increasing attention due to their crucial role in gene expression regulation and influence in various cellular processes and diseases. miRNAs can be encapsulated in extracellular vesicles (EVs), which are released by most cell types to modulate...

Gastrointestinal

Novel JAK1/2 PROTAC degrader effectively improves IBD
Degradation inducer
Inflammatory bowel disease (IBD), including ulcerative colitis and Crohn’s disease, is a chronic immune-mediated inflammatory disorder with limited long-term therapeutic options. Proteolysis-targeting chimeras (PROTACs) offer a promising strategy...
Sinorda Biomedicine and Wuxi Biologics collaborate on SND-006
Collaboration
Wuxi Biologics Co. Ltd. and Sinorda Biomedicine have established a strategic collaboration for the development and manufacturing of SND-006, a novel bispecific antibody, for the treatment of inflammatory bowel disease and other autoimmune diseases.
Boehringer Ingelheim and Simcere partner on SIM-0709
Deals and M&A
Boehringer Ingelheim International GmbH and Simcere Pharmaceutical Group Ltd. have entered into a license and collaboration agreement to develop SIM-0709 for the treatment of inflammatory bowel disease.
NIK inhibitor shows potential for IBD and sepsis
Infection
NF-κB-inducing kinase (NIK), also known as MAP3K14, is the key kinase driving noncanonical NF-κB signaling and p100 processing. Researchers from China Pharmaceutical University reported the discovery and preclinical evaluation of a novel NIK...
RIPK1 arises as target in primary sclerosing cholangitis
Inflammatory
Increasing evidence exists regarding receptor-interacting protein kinase 1 (RIPK1), a necroptosis regulator, being involved in inflammation and fibrosis in chronic liver disorders. The relationship between necroptosis and the inhibition of RIPK1...
Nanjing Chia Tai Tianqing Pharmaceutical identifies new somatostatin SST2 receptor agonists
Patents
Nanjing Chia Tai Tianqing Pharmaceutical Co. Ltd. has discovered somatostatin SST2 receptor agonists described as potentially useful for the treatment of acute pancreatitis and peptic ulcer bleeding.

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