更新于：2024-11-01

MUT

更新于：2024-11-01

基本信息

别名

MCM、methylmalonyl Coenzyme A mutase、Methylmalonyl-CoA isomerase

+ [5]

简介

Catalyzes the reversible isomerization of methylmalonyl-CoA (MMCoA) (generated from branched-chain amino acid metabolism and degradation of dietary odd chain fatty acids and cholesterol) to succinyl-CoA (3-carboxypropionyl-CoA), a key intermediate of the tricarboxylic acid cycle.

关联

叶酸/维生素D3/维生素B12/枸橼酸钙

靶点

叶酸受体 x MUT x VDR

作用机制

叶酸受体调节剂 [+3]

在研机构

Syntex Pharmaceuticals Ltd.

原研机构-

在研适应症

发育障碍 [+1]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

韩国

首次获批日期2024-08-19

Sirolimus Albumin-Bound

西罗莫司(白蛋白结合型)

靶点

MUT x mTOR

作用机制

MUT刺激剂 [+3]

在研机构

Aadi Bioscience, Inc. [+5]

原研机构

Abraxis BioScience, Inc.

在研适应症

血管周围上皮样细胞肿瘤 [+19]

非在研适应症

晚期肉瘤 [+16]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2021-11-22

Cyanocobalamin/Vitamin B6/Thiamine Chloride Hydrochloride

维生素B12/维生素B6/盐酸硫胺素

靶点

MUT

作用机制

MUT刺激剂

在研机构

Aflofarm Farmacja Polska SP zoo

原研机构

Aflofarm Farmacja Polska SP zoo

在研适应症

维生素B12缺乏症 [+2]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区

波兰

首次获批日期2016-01-03

136

项与 MUT 相关的临床试验

CTRI/2024/01/062168 / Not yet recruitingN/A

Effect of perioperative intravenous vitamin B12 on chronic post surgical pain after modified radical mastectomy:A prospective randomiized double blind control study - Nil

开始日期2025-01-06

申办/合作机构-

CTRI/2024/10/074903 / Not yet recruiting临床4期IIT

Low versus high oral dose of vitamin B12 for treatment of macrocytic anemia: a randomised controlled trial in a District hospital - Nil

开始日期2024-10-15

申办/合作机构-

NCT06494150 / Not yet recruiting临床2期IIT

Phase II Study of Nab-sirolimus and Endocrine Therapy in Recurrent Low Grade Serous Ovarian Cancer

This single arm phase II study proposes to evaluate the efficacy and safety of nab-sirolimus + endocrine therapy (Fulvestrant) in patients with recurrent low grade serous ovarian cancer (LGSOC).

开始日期2024-10-01

申办/合作机构

University of Oklahoma

[+1]

100 项与 MUT 相关的临床结果

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100 项与 MUT 相关的转化医学

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0 项与 MUT 相关的专利（医药）

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2,376

项与 MUT 相关的文献（医药）

2024-12-01·Applied Microbiology and Biotechnology

Genetic optimization of the human gut bacterium Phocaeicola vulgatus for enhanced succinate production

Article

作者: Lück, Rebecca ; Gindt, Mélanie E ; Deppenmeier, Uwe

AbstractThe demand for sustainably produced bulk chemicals is constantly rising. Succinate serves as a fundamental component in various food, chemical, and pharmaceutical products. Succinate can be produced from sustainable raw materials using microbial fermentation and enzyme-based technologies. Bacteroides and Phocaeicola species, widely distributed and prevalent gut commensals, possess enzyme sets for the metabolization of complex plant polysaccharides and synthesize succinate as a fermentative end product. This study employed novel molecular techniques to enhance succinate yields in the natural succinate producer Phocaeicola vulgatus by directing the metabolic carbon flow toward succinate formation. The deletion of the gene encoding the methylmalonyl-CoA mutase (Δmcm, bvu_0309-0310) resulted in a 95% increase in succinate production, as metabolization to propionate was effectively blocked. Furthermore, deletion of genes encoding the lactate dehydrogenase (Δldh, bvu_2499) and the pyruvate:formate lyase (Δpfl, bvu_2880) eliminated the formation of fermentative end products lactate and formate. By overproducing the transketolase (TKT, BVU_2318) in the triple deletion mutant, succinate production increased from 3.9 mmol/g dry weight in the wild type to 10.9 mmol/g dry weight. Overall, succinate yield increased by 180% in the new mutant strain P. vulgatus Δmcm Δldh Δpfl pG106_tkt relative to the parent strain. This approach is a proof of concept, verifying the genetic accessibility of P. vulgatus, and forms the basis for targeted genetic optimization. The increase of efficiency highlights the huge potential of P. vulgatus as a succinate producer with applications in sustainable bioproduction processes.Key points• Deleting methylmalonyl-CoA mutase gene in P. vulgatus doubled succinate production• Triple deletion mutant with transketolase overexpression increased succinate yield by 180%• P. vulgatus shows high potential for sustainable bulk chemical production via genetic optimization

2024-11-01·Pathology - Research and Practice

Unraveling the genetic and singaling landscapes of pediatric cancer

Article

作者: Ankam, Krishna Veni ; Nashier, Disha ; Dakal, Tikam Chand ; Kumar, Abhishek ; Srihari Sharma, M V ; Mitra, Tamoghna ; Srihari Sharma, MV ; Manjunath, Gowrang Kasaba

Pediatric cancer (PAEC) arises from gene mutations and their disrupted pathways, often driven by genetic instability affecting cell signaling. These pathways can help identify cancer triggers. Genomic studies have examined PAEC gene etiologies and disorders, but further analysis is needed to understand tumor progression mechanisms. We systematically analyzed PAEC datasets from cBioPortal, encompassing thirteen studies with 6568 samples. We identified 827 PAEC genes with mutation frequencies over fifteen across four tiers (I-IV). Tier I (mutation frequency ≥1 %) includes 40 genes, while Tier II(0.90-0.70 %), Tier III(0.60-0.50 %), and Tier IV(0.40-0.10 %) comprise 126, 336, and 325 genes, respectively. Key Tier I genes include TP53(5 %), NRAS(2.2 %), KRAS(1.8 %), CTNNB1(1.4 %), ATM(1.3 %), CREBBP(1.2 %), JAK2 (1.1 %), PIK3CA(1 %), PTEN(1 %), BRAF(0.9 %), EGFR(0.9 %), PIK3R1(0.8 %), and PTPN11(0.8 %). These genes participate in various signaling pathways (PI3K/AKT/mTOR, RAS/RAF/MAPK, JAK/STAT, and WNT/β-catenin), which are interconnected. We compared several PAEC panels with Tier I genes, and we found that the most shared across PAEC panels were TP53 (8), PTEN (7), and ATM (4). We further examined roles of TP53 in normal cells versus PEAC tumors using digital cellular and pathological imaging data supported by Human Protein Atlas. TP53 is expressed in cytosol, nucleosol, and vesicles and during cell-cycle TP53 protein in key regulator and it is present during all major cell-cycle events. Balancing of TP53^WT and TP53^MUT is the hallmark of the TP53 pathophysiology with severe functional implications. Notably, genes linked to insulin metabolism disorders may be PAEC risk factors, suggesting metabolic pathways as key research targets. This study highlights the therapeutic, prognostic, and diagnostic significance of these genes and pathways, emphasizing the need for ongoing PAEC research.

2024-09-12·International Journal of Systematic and Evolutionary Microbiology

Streptomyces castrisilvae sp. nov. and Streptomyces glycanivorans sp. nov., novel soil streptomycetes metabolizing mutan and alternan

Article

作者: Lombard, Vincent ; Larsbrink, Johan ; Widén, Tove ; Mazurkewich, Scott ; Kerkhoven, Eduard J. ; Drula, Elodie ; Rangel, Albert Tafur ; Terrapon, Nicolas

Six bacterial strains, Mut1T, Mut2, Alt1, Alt2, Alt3T, and Alt4, were isolated from soil samples collected in parks in Gothenburg, Sweden, based on their ability to utilize the insoluble polysaccharides α−1,3-glucan (mutan; Mut strains) or the mixed-linkage α−1,3/α−1,6-glucan (alternan; Alt strains). Analysis of 16S rRNA gene sequences identified all strains as members of the genus Streptomyces. The genomes of the strains were sequenced and subsequent phylogenetic analyses identified Mut2 as a strain of Streptomyces laculatispora and Alt1, Alt2 and Alt4 as strains of Streptomyces poriferorum, while Mut1T and Alt3T were most closely related to the type strains Streptomyces drozdowiczii NBRC 101007T and Streptomyces atroolivaceus NRRL ISP-5137T, respectively. Comprehensive genomic and biochemical characterizations were conducted, highlighting typical features of Streptomyces, such as large genomes (8.0–9.6 Mb) with high G+C content (70.5–72.0%). All six strains also encode a wide repertoire of putative carbohydrate-active enzymes, indicating a capability to utilize various complex polysaccharides as carbon sources such as starch, mutan, and cellulose, which was confirmed experimentally. Based on phylogenetic and phenotypic characterization, our study suggests that strains Mut1T and Alt3T represent novel species in the genus Streptomyces for which the names Streptomyces castrisilvae sp. nov. and Streptomyces glycanivorans sp. nov. are proposed, with strains Mut1T (=DSM 117248T=CCUG 77596T) and Alt3T (=DSM 117252T=CCUG 77600T) representing the respective type strains.

项与 MUT 相关的新闻（医药）

2024-06-07

·FierceBiotech

Moderna's MMA candidate nabs spot in FDA pilot for rare disease drugs

Methylmalonic acidemia, or MMA, is a genetic liver condition that in most cases is caused by a deficiency in an enzyme called MUT. Moderna’s methylmalonic acidemia candidate, mRNA-3705, will be developed under the FDA’s START pilot program for rare disease, the company announced June 6. It joins therapies from Denali Therapeutics, Neurogene, Grace Sciences and Larimar Therapeutics in the initiative, which aims to do for rare disease drugs what Operation Warp Speed did for the COVID-19 vaccines. Methylmalonic acidemia, or MMA, is a genetic liver condition that in most cases is caused by a deficiency in an enzyme called MUT, which processes fatty acids. Without it, the acids build up and damage organs. The disease is usually diagnosed within the first few months of life, and there are currently no treatments. MRNA-3705 is an mRNA-based drug that encodes MUT, thereby restoring acid metabolism. It has orphan-drug, fast-track and rare pediatric disease designations from the FDA and is currently being tested in a 63-subject, phase 1/2 clinical trial. Under START, mRNA-2705 will get extra attention from FDA officials, who will guide Moderna through product development issues like designing studies, choosing control groups and selecting the right patient population. The effort is being carried out by the agency’s Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research, which is selecting six participants total for the first iteration of the program, according to a September 2023 announcement from the FDA. “This selection highlights the promise of Moderna’s innovative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA,” Kyle Holen, M.D., senior vice president and head of development in the therapeutics and oncology unit at Moderna, said in a press release. “Selection for this program will…[result] in acceleration of our development program as we prepare for pivotal study initiation for mRNA-3705 in 2024.” Moderna's other rare disease programs include drugs for propionic acidemia, glycogen storage disorder type 1a, ornithine transcarbamylase deficiency, phenylketonuria, Crigler-Najjar syndrome type 1 and cystic fibrosis. Also picked for the START program is Calico Life Sciences' eIF2B activator fosigotifator, which is under development for Vanishing White Matter disease. The condition is an ultra-rare disease of the brain's white matter caused by variations in an essential enzyme in the cells called eIF2B. This causes a reduction in enzymatic activity that can lead to chronic activation of the integrated stress response. This causes the white matter to degenerate, causing symptoms such as impaired muscle movement, cognitive decline and seizures. Patients have a shortened life span. Fosigotifator is being developed in a partnership between Calico and AbbVie through an agreement signed in 2014.

快速通道孤儿药

2024-06-06

·Firstwordpharma

FDA taps Moderna’s mRNA therapy as sixth programme for rare disease pilot

The FDA filled its second-to-last slot for a new rare disease accelerator with an mRNA therapy from Moderna, one of a dozen programmes that represent the biotech’s push to grow beyond its bread-and-butter of infectious diseases.The candidate, mRNA-3705, is in the Phase I/II Landmark trial to treat methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase deficiency in patients one year and older. The metabolic disorder, for which there are no approved therapies, is associated with significant mortality and can currently only be treated with a liver or combined liver and kidney transplants.Kyle Holen, Moderna's head of development for its therapeutics and oncology unit, said that mRNA-2705’s inclusion in the pilot "highlights the promise of Moderna's innovative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA.” He added that the firm plans to start a pivotal trial of the candidate this year. Boosting drug developmentIt’s the sixth experimental treatment to be announced for the FDA’s pilot programme, dubbed Support for Clinical Trials Advancing Rare Disease Therapeutics (START). Intended to speed up the development of mid- to late-stage drugs and biologics for rare diseases, participants can receive frequent advice from and communicate regularly with the agency’s staff on development issues such as study design and patient enrollment. Launched in September of last year, the initiative is designed to ensure “promising treatments advance efficiently through regulatory milestones.”Participating therapies will be split between drugs and biologics, with FDA’s Center for Drug Evaluation and Research in charge of selecting three products for rare neurodegenerative conditions, and the regulator’s Center for Biologics Evaluation and Research (CBER) responsible for picking four cell or gene therapies intended to treat a rare disease that is likely to lead to significant disability or death within the first decade of life. The slate of candidates, which were first revealed at the end of May, includes Larimar Therapeutics’ nomlabofusp, a protein replacement therapy for Friedreich’s ataxia; Grace Science’s GS-100, an AAV9 gene therapy for NGLY1 deficiency; Myrtelle’s rAAV-Olig001-ASPA, a gene therapy for Canavan disease; Denali Therapeutics’ DNL126, an enzyme replacement therapy for MPS IIIA; and Neurogene’s NGN-401, a gene therapy for Rett syndrome.One participant has yet to be announced.

基因疗法临床研究临床申请信使RNAsiRNA

2024-06-06

·Pharmaceutical Technology

Moderna and Myrtelle among those picked for FDA rare disease programme

The FDA’s pilot programme is set to accelerate the development of novel drug and biological products for rare diseases. Credit: Tada Images via Shutterstock. Moderna and Myrtelle’s rare programmes will both be included in a pilot programme by the US Food and Drug Administration (FDA) that aims to accelerate the development of novel drugs and biological products for rare diseases. Recommended Buyer's Guides Buyer's Guide Top Guide for Drug Delivery Systems Buyer's Guide Leading Guide to Clinical Packaging Companies in contract Manufacturing for the Pharmaceutical Industry Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. Thank you. You will receive an email shortly. Please check your inbox to download the Report. Go deeper with GlobalData Reports LOA and PTSR Model - Gene Therapy For Melanoma And Multiple Myeloma... Reports NPVM: MeiraGTx Holdings Plc's Cevaretigene Ritoparvovec Premium Insights The gold standard of business intelligence. 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Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Announced in September 2023, the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) programme aims to expedite new drug development by providing sponsors with frequent advice and regular communication with the FDA to advance their programme as quickly as possible. Moderna’s START programme evaluates mRNA-3705, an investigational therapeutic for methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. The candidate is being investigated in a Phase I/II Landmark study (NCT04899310), designed to evaluate the safety and tolerability of Mrna-3705 administered via intravenous infusion (IV) in patients one year and older with isolated MMA due to methylmalonyl-CoA mutase (hMUT) deficiency. Myrtelle is developing rAAV-Olig001 for Canavan disease (CD), a fatal childhood genetic disorder characterised by the collapse of white matter in the brain due to a mutation in the aspartoacylase gene (ASPA). This affects the creation of myelin, leading to a deficiency in the aspartoacylase enzyme. The gene therapy is a new vector from a class of recombinant adeno-associated viruses (rAAVs) that selectively target oligodendrocytes, the cells in the brain that produce myelin. See Also: The tiny pills that may send shockwaves across immunology Roche’s PI3K inhibitor secures breakthrough status in breast cancer It is being evaluated in a Phase l/ll clinical trial (NCT04833907) enrolling 24 patients with CD. It involves administering the gene therapy to patients using neurosurgical procedures, which involves direct administration of gene therapy to affected regions of the brain. Outcome measures for the open-label clinical trial include longitudinal clinical assessments and brain imaging. In the announcement accompanying the acceptance, Myrtelle global regulatory affairs senior vice president Nancy Barone Kribbssaid: “Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application.” Applicants had to meet certain criteria from the Center for Biologics Evaluation and Research (CBER), which has selected four participants for the pilot programme;, and the Center for Drug Evaluation and Research (CDER), which has selected three. Earlier this week, Denali Therapeutics , Neurogene, Larimar Therapeutics and Grace Science all announced that programmes will be included in the pilot. Neurogene’s gene therapy NGN-401 is currently under investigation to treat Rett syndrome. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. Thank you. You will receive an email shortly. Please check your inbox to download the Report. 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