ribitol (BBP-418) / BridgeBio - LARVOL DELTA

Omic evaluation of G-CSF-induced changes in bone marrow: A focus on lipid metabolism (ASH 2025) - "Metabolomic analysis revealed statistically significant increases in 3-hydroxypropanoic acid, fucose,glutamic acid, O-phospho-L-serine, phosphoric acid, ribitol, trimethyllysine, myo-inositol, and oxaloaceticacid in the group exposed to G-CSF. Conversely, levels of alpha-glucosamine 1-phosphate, proline, andthreonine were decreased in the G-CSF-exposed group. The most prominent changes observed in thelipidomic analysis involved ether-linked phosphatidylcholines, which are essential components of the cellmembrane and lipid rafts."

Hematological Disorders • Inflammation • Metabolic Disorders • CSF3R

Rewiring central metabolism in Komagataella phaffii for efficient mannose synthesis. (PubMed, Microb Cell Fact) - "This study achieved efficient mannose production in K. phaffii by remodeling central metabolism. It not only offers a new route for mannose biosynthesis but also establishes a model framework for engineering K. phaffii to produce other high-value bioactive compounds."

Journal

Novel causal associations between plasma metabolites and prostate cancer risk revealed by mendelian randomization. (PubMed, Metabol Open) - "Ribitol was associated with a reduced risk, while N2,N5-diacetylornithine, N-acetylarginine, and N-acetylcitrulline were associated with an elevated risk...The incorporation of a male-exclusive metabolomic dataset (METSIM) strengthens the validity of our findings for this male-specific cancer. These metabolites represent promising candidates for further mechanistic investigation into prostate cancer etiology and potential translation into clinical biomarkers."

Journal • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor

Causal insights into immune cell profiles, plasma metabolites, and bladder cancer: A Mendelian randomization approach. (PubMed, Medicine (Baltimore)) - "CD19 on IgD- CD27- was linked to BC, mediated by ribitol levels, with a mediation ratio of 6.58%...This study identified 5 immune cell phenotypes that are causally associated with BC, mediated through 5 plasma metabolites. These findings enhance our understanding of the biological pathways involved, provide potential biomarkers for identifying at-risk populations, and offer insights into strategies for early prevention and diagnosis of BC."

IO biomarker • Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • CD24 • CD27

CLINICAL IMPACT OF UNTARGETED BASELINE PLASMA METABOLOMICS IN NEWLY DIAGNOSED DIFFUSE LARGE B CELL LYMPHOMA (SIE 2025) - "The study relies on a real-world cohort of 96 R-CHOP treated DLBCL provided with baseline plasma samples...4-hydroxybenzeneacetic acid and ribitol are associated with changes in intestinal microbiome composition...Baseline plasma metabolites harbor a promising prognostic value in DLBCL and exhibit distinct distributions across molecular clusters. If validated, these findings may provide new insights into DLBCL biology and may identify novel potential therapeutic targets."

Clinical • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma

Alkyne-tagged ribitol-5-phosphate derivatives for metabolic labelling of alpha-dystroglycan. (PubMed, RSC Chem Biol) - "Novel tools are needed to study its rare and intriguing O-mannosyl glycans. This report describes the synthesis and evaluation of alkyne-tagged ribitol-5-phosphate derivatives for the metabolic labelling of α-dystroglycan in mammalian cells."

Journal • Muscular Dystrophy

The effect of treatment with a non-ionic surfactant vesicular formulation of sodium stibogluconate on host immune responses and serum metabolites in a murine model of Leishmania donovani. (PubMed, Front Immunol) - "Visceral leishmaniasis (VL), caused by Leishmania donovani, is associated with parasite-induced immunological and physiological changes that ensure the survival of amastigotes within the host. Additionally, glycine and ribitol show promise as immune correlates for antiparasitic therapies. These findings highlight the diagnostic and prognostic potential of serum-derived metabolites in monitoring host immune responses to VL and treatment."

Biomarker • Journal • Preclinical • Infectious Disease • CD4 • CD8 • IFNG • IL10 • IL12A • IL4 • TGFB1

Dysfunctional CRPPA is responsible for recessively inherited Hereford hydrocephalus with muscular dystrophy and retinal dysplasia. (PubMed, Vet Pathol) - "Analyses identified a splice site variant in CRPPA (CDP-L-ribitol pyrophosphorylase A or ISPD) for which the affected calves were homozygous...This phenotype is like Walker-Warburg syndrome in humans, some cases of which are attributed to mutations in CRPPA. This advancement allows for testing to eliminate carriers and potential for affected calves."

Journal • CNS Disorders • Inherited Retinal Dystrophy • Muscular Dystrophy • Ventriculomegaly

Altered lipidomic and metabolomic status in cerebrospinal fluid of children with myelin oligodendrocyte glycoprotein antibody-associated disorder. (PubMed, Biochem Biophys Rep) - "Among the 549 (0.03 %) hydrophilic molecules that were differentially presented, pyridoxine, ribitol, and isethionate levels were significantly lower in patients with MOGAD...In summary, CSF samples from children with MOGAD exhibit distinctive lipidomic and metabolomic profiles. These findings provide evidence for the diagnostic potential of CSF-based lipidomic and metabolomic analyses for childhood-onset MOGAD."

Journal • CNS Disorders • Epilepsy • Solid Tumor

Fukuyama congenital muscular dystrophy: Clinical features and therapeutic advances. (PubMed, Brain Dev) - "Additionally, it was clarified that the gene product of FKTN is a glycosyltransferase that transfers ribitol-5-phosphate from cytidine diphosphate ribitol, a precursor for the synthesis of the O-mannosyl glycans of α-dystroglycan, a cell membrane component...Thus, phase II clinical trials are underway with the aim of obtaining insurance approval. This review provides an overview of the clinical course and current status of treatments being developed for FCMD."

Journal • Review • Cardiomyopathy • Cardiovascular • Muscular Dystrophy • Respiratory Diseases

Selective wall teichoic acid labeling via metabolic incorporation (ACS-Fall 2025) - "In several pathogenic bacteria, including Staphylococcus aureus, WTAs consist of ribitol phosphate repeat units, constituting about half of cell wall mass...Additionally, we plan to investigate whether adaptations of S. aureus to cell wall stress cause changes in WTA abundance and localization. We anticipate that our results will provide new fundamental insights into the roles and synthesis of WTAs and may aid in developing new antibiotics to combat antimicrobial resistance."

Infectious Disease

Ribitol treatment rescues dystroglycanopathy mice with common L276I mutation. (PubMed, PLoS One) - "The higher level of restoration in matriglycan in L276I mice than in P448L mice is consistent with the hypothesis that therapeutic potential of ribitol treatment may depend on the remaining function of mutant FKRPs. These results support clinical trials of ribitol to the majority of patients with FKRP mutations."

Journal • Preclinical • Fibrosis • Immunology • Muscular Dystrophy • FKRP

Chemical Synthesis of Oligosaccharides Derived from Streptococcus Pneumoniae Serotype 35B and D Provides Molecular Insight in l-Ficolin Binding. (PubMed, J Am Chem Soc) - "Solution-phase NMR experiments and computational modeling demonstrate that Galf(OAc)-1,1-Ribitol of the repeating unit of 35B CPS constitutes the minimal motif for binding to l-ficolin, and the acetyl ester is a key recognition motif...The oligosaccharides were employed to investigate binding to pneumococcal serum factors 35a and 29b, which indicates that immunization with 35B CPS will not provide protection against 35D. Antibodies that can bind 35D can, however, recognize 35B, and thus, 35D CPS may provide cross-protection."

Journal • Infectious Disease • Pneumococcal Infections • Pneumonia

LC-QTOF-MSE with MS1-based precursor ion quantification and SiMD-assisted identification enhances human urine metabolite analysis. (PubMed, Comput Struct Biotechnol J) - "Untargeted retrospective profiling revealed level 1 seven metabolites, including ribitol, creatine, glucuronic acid, trans-ferulic acid, succinic acid, dimethylglycine, and 3-hydroxyphenylacetic acid related to sex variation (VIP > 1.5)...It enables reliable target quantification and enhances confidence in metabolite identification while also reducing sample and instrumental demands. These features make it particularly well-suited for clinical metabolomics studies."

Journal

Baseline predictors for 28-day COVID-19 severity and mortality among hospitalized patients: results from the IMPACC study. (PubMed, Front Med (Lausanne)) - "Adding laboratory results such as TNFRSF11B and plasma ribitol count increased the prediction model by 3.5%...Several biomarkers were identified to modestly improve the predictions. The results also provide a baseline of SARS-CoV-2 infection during the early stages of the coronavirus emergence and can serve as a baseline for future studies that inform how the genetic evolution of the coronavirus affects the host response to new variants."

Journal • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases • FGF23 • IL6 • TNFRSF11B

Symmetrospora pini sp. nov., an anamorphic yeast species isolated from plant leaves. (PubMed, Int J Syst Evol Microbiol) - "Phenotypically, the two strains can be distinguished from S. clarorosea and S. gracilis by their ability to assimilate d-glucosamine and grow at 30 °C, as well as their inability to assimilate ribitol...nov. is proposed with holotype CICC 33601T (MycoBank MB 857729)."

Journal

CLINICAL IMPACT OF UNTARGETED BASELINE PLASMA METABOLOMICS IN NEWLY DIAGNOSED DIFFUSE LARGE B CELL LYMPHOMA (EHA 2025) - "The maximally selected rank statistics was used to identify the best cutoff values in predicting progression free survival (PFS) for each metabolite.The cohort included 96 newly diagnosed DLBCL homogenously treated with R-CHOP. Baseline plasma metabolites harbor a promising prognostic value in DLBCL, and some metabolites are upregulated in specific DLBCL molecular clusters. Interestingly, L-threonine and ribitol are mainly involved in glycolysis, tricarboxylic acid cycle and ATP synthesis, therefore providing new insights into DLBCL biology and identifying novel potential therapeutic targets."

Clinical • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Leaf functional metabolic traits reveal the adaptation strategies of larch trees along the R/B ratio gradient at the stand level. (PubMed, Sci Rep) - "Metabolite analysis revealed that, under 1.5R:1B light conditions, the upregulation of sucrose synthase (SS) and sucrose-phosphate synthase (SPS) enzyme activities accelerated the consumption of maltose in leaves, led to the accumulation of ribitol and d-glucitol, and increased the levels of organic acids, thereby promoting the accumulation of flavonoids. These findings suggest that 0.6R:1B favors a resource acquisition strategy (rapid growth), while 1.5R:1B leans towards a resource conservation strategy (slow growth). This study provides a new perspective on the effects of light conditions on understory vegetation regeneration."

Journal

Targeting of ribitol metabolic pathway using AAV to enhance glycosylation and therapeutic efficacy of metabolites in the FKRP-P448L mutant mouse. (ASGCT 2025) - "By targeting the biosynthetic pathway of ribitol-5-phosphate the effectiveness of ribitol treatment can potentially be improved for treating FKRP-related muscular dystrophies. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Muscular Dystrophy • AKR1B1 • FKRP

Design and Evaluation of FKRP Dimer Expression for Restoration of Functional Matriglycan in a LGMD 2I/R9 Mouse Model (ASGCT 2025) - "Of these, Limb Girdle Muscular Dystrophy 2I/R9 results from mutation-bearing Fukutin-related protein (FKRP) having little or no functional capacity as ribitol-phosphate (RboP) transferase to elongate the core M3 glycan (matriglycan) which is required to bind laminin and other proteins, securing the muscle membrane and extracellular matrix...This novel approach to FKRP gene replacement has the potential to restore greater levels of matriglycan expression at lower doses of AAV thereby reducing dose related toxicity. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Fibrosis • Immunology • Inflammation • Muscular Dystrophy • FKRP

Meniscomyces senecionis sp. nov., a novel anamorphic basidiomycete yeast species. (PubMed, Int J Syst Evol Microbiol) - "The novel strain also exhibits distinct physiological traits, including an inability to assimilate maltose and cellobiose and the ability to utilize d-xylose, d-ribose, glycerol, ribitol, d-mannitol, dl-lactate and d-gluconate...nov. (Holotype: CICC 33585; MycoBank: MB 857392) is proposed."

Journal

ELUCIDATING THE ROLE AND MECHANISM OF TUFM IN LGR5 ACTIVATION WITHIN WNT SIGNALING IN NEUROGENIC TUMOR CELLS AND ENHANCING OF NEURAL STEM CELLS USING TUFM (ADPD 2025) - "Through proteomics analysis, common changes in protoporphyrinogen oxidase (PPO), suppressor APC domain - containing protein 2 (SAPCD2), katanin p60 ATPase -containing subunit A -like 2 (KATNAL2), Rab GTPase - binding effector protein 2 (RABEP2), ribitol -5-phosphate xylosyltransferase 1 (RXYLT1), and organic solute carrier partner 1 (OSCP1) were observed in the two cell types under TUFM regulation... The cellular activities of genes and proteins associated with the TUFM, Lgr5, and Wnt signaling pathway were validated in SH -SY5Y cells and hNSCs. Based on our previous research findings, we have confirmed that substantial involvement of splicing and mRNA transcription among various mechanisms. Modulating splicing factors triggers the activation of TUFM and Lgr5 within the Wnt signaling pathway, consequently enhancing the functionality of hNSCs."

Tumor cell • Brain Cancer • CNS Disorders • Oncology • PPOX • SAPCD2 • SPON1 • TUFM

Synergistic Effect of Ribitol and Shikonin Promotes Apoptosis in Breast Cancer Cells. (PubMed, Int J Mol Sci) - "Metabolomic analysis supports the premise that inhibition of the Warburg effect is involved in shikonin-induced cell death, which is likely further enhanced by dysregulation of glycolysis and the tricarboxylic acid (TCA) cycle, afflicted by ribitol treatment. In conclusion, the present study demonstrates that the metabolite ribitol selectively enhances the cytotoxic effect mediated by shikonin against breast cancer in vitro."

Journal • Breast Cancer • Oncology • Solid Tumor • BCL2L1 • MCL1 • MYC

Substrate flexibility of Mycoplasma fermentans mf1 phosphorylcholine transferase. (PubMed, Glycoconj J) - "Mf1 activity is inhibited by β-glycerophosphate, an isomer of part of CDP-glycerol which is known to compete with CDP-ribitol in enzymatic reactions catalyzed by fukutin, a human protein sharing structural homology with mf1. For the first time, a phosphorylcholine transferase, mf1, could be biochemically characterized in vitro and its lipid products with zwitterionic phosphodiesters attached could be detected specifically with the pentraxin serum amyloid P."

Journal

Unraveling the genome of Proteus mirabilis strain representing the O78 serogroup: Insights into the unique features of the O-antigen biosynthesis gene cluster. (PubMed, Infect Genet Evol) - "The function of licD in this strain appears to be linked to the ispD gene, potentially involved in the biosynthesis of CDP-ribitol. Additionally, we explored other genomic features, including the strain's genetic similarity to closely related microorganisms, the presence of antimicrobial resistance genes, and prophage elements. This study provides valuable insights into the genetic factors underlying the pathogenicity of the P. mirabilis 1B-m strain and its potential implications for hospital-acquired infections."

Journal • Infectious Disease • Inflammation