A Double Blind, Placebo Controlled Trial to Investigate the Efficacy and Safety of Two Concentrations of Juvidex (Mannose-6-Phosphate) in Accelerating the Healing of Split Thickness Skin Graft Donor Sites Using Different Dosing Regimes

The objective of this study is to compare the efficacy of two concentrations of Juvidex with placebo on the healing of split thickness skin graft donor sites

Start Date01 Feb 2008

Sponsor / Collaborator

Renovo Ltd.

NCT00663780 / CompletedNot Applicable

Pharmacogenomic Analysis of Blood Samples to Identify Polymorphisms That Segregate Responders From Non-Responders Following Treatment With Juvidex of Split Skin Donor Sites in Renovo's Clinical Study RN1004-0082

The objective of the study is to identify variations in subjects genetic makeup that segregate responders from non-responders in respect of response to Juvidex in the clinical trial RN1004-0082

Start Date01 Feb 2008

Sponsor / Collaborator

Renovo Ltd.

EUCTR2007-004155-11-GB / Not yet recruitingNot Applicable

A double blind, placebo controlled trial to investigate the efficacy and safety of two concentrations of Juvidex in accelerating the healing of split thickness skin graft donor sites using different dosing regimes - Juvidex - SGDS

Start Date17 Jan 2008

Sponsor / Collaborator

Renovo Ltd.

100 Clinical Results associated with Mannose phosphate

100 Translational Medicine associated with Mannose phosphate

100 Patents (Medical) associated with Mannose phosphate

131

Literatures (Medical) associated with Mannose phosphate

01 Dec 2024·SCIENCE OF THE TOTAL ENVIRONMENT

Spherical covalent-organic framework-assisted laser desorption ionization mass spectrometry reveals the promotional effect of triphenyl phosphate on breast cancer in mice

Article

Author: Lin, Zian ; Dan, Akang ; Cai, Zongwei ; Ouyang, Dan

Triphenyl phosphate (TPP), a wide-used organophosphate flame retardants (OPFRs), is suspected to be a risk factor for the female-specific cancers, but underlying toxicity mechanisms of environmentally relevant dose exposure remain unclear. Herein, a strategy of spherical covalent organic framework (TPB-BPTP-COF)-assisted laser desorption ionization mass spectrometry (LDI-MS), which benefited from fast analysis speed, facile sample preprocessing, and high throughput, was proposed for unveiling the biomarkers of breast cancer (BC) and the relationship between TPP exposure and progression of BC in mice by serum metabolism analysis. The results displayed that 13 metabolites associated with BC development were up-regulated in experimental group versus healthy control mice. Moreover, long-term exposure to environmentally relevant doses of TPP was found to promote BC, mainly by affecting glycolysis/gluconeogenesis, pyrimidine metabolism, pantothenic acid and CoA biosynthesis, and β-alanine metabolism. This work proved the potential application of COFs as LDI-MS substrates in analyzing complex biological samples, and also revealed the risk of long-term low-dose exposure to TPP in the development of BC.

01 Sep 2024·ENVIRONMENT INTERNATIONAL

Chemical and non-chemical stressors in a postpartum cohort through wristband and self report data: Links between increased chemical burden, economic, and racial stress

Article

Author: Frey, Jenna ; Mills-Koonce, W Roger ; Fry, Rebecca C ; Short, Sarah J. ; Stapleton, Heather M ; Rager, Julia E. ; Stapleton, Heather M. ; Rager, Julia E ; Wylie, Amanda ; Herkert, Nicholas J. ; Hartwell, Hadley J ; Herkert, Nicholas J ; Hickman, Elise ; Hartwell, Hadley J. ; Short, Sarah J ; Mills-Koonce, W. Roger ; Propper, Cathi ; Fry, Rebecca C.

Multiple external stressors are known to have adverse impacts on health and development. Certain groups are more vulnerable and/or more likely to be exposed toenvironmental, psychological, and social stressors simultaneously. Yet, few studies have examined combined exposure to environmental toxicants and psychosocial stress. Here, we integrated environmental chemical exposure data collected using silicone wristbands and self-report social stressor data within the Brain and Early Experience (BEE) perinatal cohort to understand co-exposure to environmental chemicals and social stress. Silicone wristbands were worn for one week by mothers throughout central North Carolina who were 6 months postpartum (n = 97). Exposure to 110 environmental chemicals across eight chemical classes was quantified on silicone wristbands using gas chromatography mass spectrometry. Social stress was evaluated using eight established self-report questionnaires (e.g., Brief Symptom Inventory, Perceived Stress Scale), quantifying experiences such as race-related stress, economic strain, and relationship conflict. Hair cortisol levels were measured as an additional metric of stress. The chemical exposure landscape and associations among chemical exposure, demographic characteristics, and social stress were characterized through individual variable analyses, cluster and data reduction, and compiled scoring approaches to comprehensively evaluate chemical and social stress burdens. We found that chemicals contain co-occurring patterns largely based on chemical class, with phthalates representing the chemical class with highest exposure and polychlorinated biphenyls the lowest. Chemicals showed differential exposure across racial groups, with diethyl phthalate, triphenyl phosphate, and tris(3,5-dimethyl phenyl) phosphate at higher levels in Black participants compared with White participants. Integrating social stressor profiling with chemical exposure data identified one particularly vulnerable subset of participants in which high chemical exposure burden coincided with high experiences of racism and economic stress. These findings demonstrate co-occurring chemical and social stress, warranting further investigation to better understand how these combined stressors may contribute to disparities in maternal and child health.

01 Aug 2024·REGULATORY TOXICOLOGY AND PHARMACOLOGY

Can next generation ecological risk assessment decisions be made today?―A case study of regulatory risk assessment in the United States

Article

Author: DeLeo, Paul C

We examined the need for new in vivo avian toxicity testing for three common industrial chemicals (1,2 dichloropropane, 1,1,2-trichloroethane and triphenyl phosphate) based on estimated avian exposures using fugacity and multimedia fate models for current conditions of use compared to hazard information including existing in vivo test data for the chemicals and analogs, interspecies correlation estimates and results from hundreds of acute avian dietary toxicity studies. The data indicated that acute avian toxicity is not likely to be observed below 10 ppm in the diet for any chemical with the exception of those with a specific mode of toxic action. Modeling indicated low exposure potential for terrestrial birds to any of the three chemicals, with estimated dietary concentration of less than 0.001 ppm. Despite uncertainty associated with the underlying data sources, the four order of magnitude gap between potential exposure and a minimum hazard threshold suggests that additional avian in vivo testing would not generate valuable data. However, a weight of evidence approach for integrating data is necessary to engender greater confidence among government decision-makers in cases where data from a particular in vivo study is not expected to improve risk decision-making and an existing data gap can remain unfilled.

News (Medical) associated with Mannose phosphate

21 Feb 2023

·www.globenewswire.com

M6P Therapeutics Presents Promising Preclinical Data In Lysosomal Storage Disorders At The 19th Annual WORLDSymposium™ 2023

Results Highlight Potential Utility of Targeting Mannose 6-Phosphate Pathway to Treat Multiple Lysosomal Storage DisordersST. LOUIS, Mo., Feb. 21, 2023 (GLOBE NEWSWIRE) -- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced the presentation of promising preclinical data at the 19th Annual WORLDSymposium™, a research conference dedicated to lysosomal diseases being held in Orlando, FL, and virtually Feb. 22-26, 2023. In five poster presentations, including two Contemporary Forum presentations, M6P Therapeutics’ researchers report preclinical efficacy results for LSDs, including Gaucher disease and Pompe disease. “We are thrilled with the continued development of our S1S3 phosphotransferase platform for producing lysosomal enzymes with high levels of mannose 6-phosphate to enable much better drug targeting and delivery of therapeutic lysosomal enzymes to all affected cells and tissues,” said Cuong Do, Chairman of M6P Therapeutics. “We believe that this approach has the potential for creating more potent and effective enzyme replacement therapies and therapeutic proteins from gene therapies for the treatment of lysosomal storage disorders.” The following forum presentations and posters will be presented at the WORLDSymposium: Poster #160: S1S3 Gene Therapy for the Potential Treatment of Lysosomal Disorders Title: Novel dual promoter AAV gene therapy platform ensures production of therapeutic soluble lysosomal enzymes with high M6P content to enable broad cellular uptake and cross-correction in vivo Date and Time: Contemporary Forum Talk: Saturday, February 25, from 6:45 – 9:00 AM ET followed by:Poster Session: Saturday, February 25, 3:00 PM - 4:00 PM ETSession: Contemporary Forum Poster PresentationsPresenter (In-person): Andrew Hedman, M6PT Scientist Poster #142: M021 for the Potential Treatment of Pompe Disease Title: M021: rhGAA with optimal glycosylation profile containing very high levels of bis-phosphorylated N-glycans clears accumulated glycogen and rapidly normalizes muscle strength in treated Pompe mice Date and Time: Contemporary Forum Talk: Saturday, February 25, from 10:30 – 11:30 AM ET followed by:Poster Session: Saturday, February 25, 3:00 PM - 4:00 PM ETSession: Contemporary Forum Poster PresentationsPresenter (In-person): Russell Gotschall, Vice-President, Research & Development, M6PT Poster #227: Novel Dual-Promoter Expression Vector for Gaucher Gene Therapy Title: Novel AAV gene therapy produces β-glucocerebrosidase with high levels of M6P to enable cellular uptake and cross-correction in the CNS as a potential treatment for Type 2/3 Gaucher disease Date and Time: Saturday, February 25, 3:00 PM - 4:00 PM ETSession: Contemporary ForumPresenter (In-person): Lin Liu, Director, Research & Development, M6PT Poster #96: Intra-Nasal Delivery of M011 for Gaucher Type 2/3 Title: Highly phosphorylated β-glucocerebrosidase (M011) that targets central nervous system neurons as a potential treatment for neuronopathic Gaucher disease type 2 and 3 Date and Time: Saturday, February 25, 3:00 PM - 4:00 PM ETSession: Contemporary ForumPresenter (In-person): Michael Digruccio, M6PT Scientist Poster #99: M011 May Enable Alternative, Less-frequent ERT Dosing Strategies for Gaucher Disease Type 1 Title: Highly phosphorylated β-glucocerebrosidase (M011) has much broader tissue distribution and superior substrate reduction with potential for alternative dosing strategies for the treatment of Gaucher type 1 Date and Time: Saturday, February 25, 3:00 PM - 4:00 PM ETSession: Contemporary ForumPresenter (In-person): Hung Do, CSO, M6PT For more information, registration details and to access the live streamed events, presentations and Q&A sessions, please go to WorldSymposium. The abstracts will also be made available on the “Publications” section of M6PT’s corporate website following the presentations. About the Annual WORLDSymposium™ The WORLDSymposium™ is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 18th Annual WORLDSymposium™, please visit https://worldsymposia.org/. About M6P Therapeutics M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics’ proprietary bicistronic-S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics’ team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its bicistronic-S1S3 platform. M6P Therapeutics’ mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: www.m6ptherapeutics.com. Contact us to learn about partnering opportunities with M6P Therapeutics: M6P Therapeutics:314-236-9694info@m6ptherapeutics.com Media:Alex Van Rees, SmithSolve973-442-1555 x111alex.vanrees@smithsolve.com

Gene TherapyClinical Study

11 Oct 2022

·www.biospace.com

M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress

- Preclinical data provide proof of concept for co-expression of GBA gene with the truncated S1S3 PTase to produce highly phosphorylated GCase (β-glucocerebrosidase) - - Company’s M012 gene therapy had high M6P content and enhanced binding to CI-MPR as compared to the enzyme produced without S1S3 PTase - - Results suggest that M012 AAV can enable effective cross-correction in the CNS, and M012 AAV is a potential novel gene therapy for Gaucher disease, including neuronopathic phenotypes - ST. LOUIS--(BUSINESS WIRE)-- M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced a poster presentation titled, “A novel AAV9 gene therapy for producing β-glucocerebrosidase enzyme with high mannose 6-phosphate content to treat Gaucher disease,” by Lin Liu, Ph.D., Director, R&D of M6P Therapeutics, at the 29th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre. “The data from this preclinical study illustrate the potential of our AAV-GBA-S1S3 gene therapy candidate for the treatment of Gaucher disease, including neuronopathic phenotypes,” said Pawel Krysiak, President and CEO of M6P Therapeutics. “M012 is part of our pipeline of candidates leveraging a first-in-class co-expression S1S3 platform technology for the treatment of lysosomal storage disorders. Using our technology platform, we enhance mannose 6-phosphate content on lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved enzyme uptake across target tissues.” Gaucher disease is a rare inherited metabolic disorder of defective lipid catabolism caused by deficient β-glucocerebrosidase (GCase) encoding by the GBA1 gene resulting in accumulation of glycosphingolipids in the periphery and central nervous system (CNS). Multiple recombinant human GCase enzyme replacement therapies have been approved to treat Gaucher disease type 1, but there are no effective treatments to address the neuronopathic manifestations for Gaucher disease types 2 and 3. Gene therapy could be a potential therapeutic approach to treat all three types of Gaucher disease. "In this study, gene copy and transcriptome analysis showed that the dosed novel M012 AAV vector enabled transduction of peripheral tissues and various regions of brain in wild-type mice post-intravenous injection,” said Dr. Liu. “We observed increased GCase enzyme activity with enhanced CI-MPR affinity in peripheral tissues of treated animals and noted strong GCase protein signal with broad distribution detected in M012 AAV vector-treated brain samples. These results strongly suggest that M012 AAV enables effective GCase cross-correction in the CNS. We look forward to sharing additional updates and data as the program progresses.” Presentation highlights from the poster include: Data from previous publications show that co-expression of truncated GlcNAc-1-phosphotransferase (designated as S1S3 PTase) greatly increases the levels of mannose 6-phosphate (M6P) on lysosomal enzymes, which enables efficient cellular uptake via the cation-independent mannose 6-phosphate receptor (CI-MPR) that is the broadly expressed on most cells including neurons and other cell types in the CNS The study demonstrated M6P Therapeutics’ ability to regulate and enhance M6P content on lysosomal enzymes for both enzyme replacement and gene therapies, which improves enzyme uptake across target tissues, restoring the enzymatic activity within this natural pathway The M012’s novel AAV gene therapy approach uses a dual promoter construct design for co-expression of GBA gene with the truncated S1S3 PTase to produce highly phosphorylated GCase as a potential treatment for Gaucher disease The produced hGCase with S1S3 PTase co-expression was shown to have high M6P content and enhanced binding to CI-MPR as compared to the enzyme produced without S1S3 PTase GCase uptake and distribution in the CNS was also evaluated by immunohistochemical staining About M6P Therapeutics M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted enzyme replacement and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics’ proprietary co-expression S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics’ team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of enzyme replacement and gene therapies by harnessing the power of protein phosphorylation using its co-expression S1S3 platform. M6P Therapeutics’ mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: .

Gene TherapyFirst in ClassCell Therapy

100 Deals associated with Mannose phosphate

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Indication	Highest Phase	Country/Location	Organization	Date
Cicatrix	Phase 3	-	Renovo Ltd.	-
Wounds and Injuries	Phase 2	GB	Renovo Ltd.	01 Aug 2004

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