Histiocytosis, Langerhans-Cell

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

DUBLIN, Jan. 13, 2023 /PRNewswire/ -- The "Global Lysosomal Disease Treatment Market Size, Share & Industry Trends Analysis Report By Disease Type (Gaucher's Diseases, Fabry Diseases, Mucopolysaccharidosis, Pompe's Syndrome), By Type of Therapy By End User, By Regional Outlook and Forecast, 2022 - 2028" report has been added to ResearchAndMarkets.com's offering. The Global Lysosomal Disease Treatment Market size is expected to reach $11 billion by 2028, rising at a market growth of 6.3% CAGR during the forecast period. Lysosomal diseases (LSDs) are a complex group of metabolic disorders that are often inherited in an autosomal recessive manner. There are currently 45 known diseases. They are caused by malfunctions in various lysosomal processes, most often by abnormalities in genes that produce catabolic enzymes responsible for the breakdown of macromolecules. As the faulty enzyme accumulates substrate, cell malfunction and clinical symptoms escalate. Other types of lysosomal dysfunction that can lead to lysosomal storage include defects in post-translational processing of lysosomal enzymes, errors in enzyme trafficking or targeting, and dysfunction of non-enzymatic transmembrane and solubilize. Lysosomal hydrolase mutations, which are implicated in the breakdown of cellular macromolecules, are the primary cause of LSDs, accounting for more than two-thirds of cases. As a result, there are many different types of lipids, glycogen, mucopolysaccharides, and glycoproteins among the storage products in LSDs that are biochemical in origin. The LSDs are all inherited as autosomal recessive characteristics, except the X-linked Fabry, Danon, and Hunter diseases. The LSDs can affect the majority of organs, either alone or as part of a multisystem illness, and are clinically very heterogeneous. Even though lysosomal enzymes are housekeeping genes and expressed in the majority of cells, the tissues on which they act are distributed much less uniformly, and this determines which tissues in any individual are affected. COVID-19 Impact Analysis The COVID-19 public health crisis has affected almost every industry. The COVID-19 outbreak has caused a significant decrease in demand for the lysosomal disease treatment size of the market across several sectors, especially the health and pharmaceutical sector, as coronavirus crises sweep the globe and force healthcare providers to devote a large percentage of their funds to fighting COVID-19. The risk of infection was the primary factor contributing to the delays in treatment that patients with lysosomal storage disorder encountered in hospitals. The COVID-19 outbreak harms the market's value in 2022 and beyond because it will delay the development of the worldwide market for lysosomal disease treatments. Market Growth Factors Incidence of Lysosomal Disorders Are Rising The primary driving forces in the enzyme replacement therapy market are the expanding research and development for lysosomal disease diagnosis and drug development for treatment. The increasing incidence of lysosomal disease will be a key factor contributing to the acceleration of the market's growth rate. According to a report published by the National Center for Biotechnology Information, the prevalence of Fabry disease in communities of white, male individuals ranged from 1:17,000 to 1:117,000 in January 2020. Atypical presentations were associated with around 1:6000-1:40,000 females and 1:1000-1:3000 males, whereas 1:22,000-1:40,000 males had the classic Fabry disease mutations. Financial Incentives for Developing Orphan Drugs to Recoup R&D Expenses The increases in healthcare spending, which helps to improve its infrastructure, is a crucial element affecting the market's growth rate for therapies for lysosomal diseases. The market dynamics will be further impacted by various government organizations' efforts to strengthen the healthcare infrastructure by boosting funding. Additionally, the market for lysosomal disease therapies will grow as a result of the rising acceptance rate of early diagnosis and the increasing number of government programs to raise awareness. Along with this, expanding government benevolent programs and an increase in disposable money is expected to accelerate market expansion. Marketing Restraining Factor: The Treatment of Stem Cells Is Fraught with Issues Many of the issues that can arise soon after the transplant are caused by the bone marrow being destroyed just before it by drugs or radiation. Some may be unintended consequences of the conditioning procedures themselves. The patient can get support from the transplant team to manage side effects. Most can be addressed to make the patient feel better, and some can be prevented. This is not a comprehensive list, so please inform the physician or the transplant team of any issues or adjustments that experience. Key Topics Covered: Chapter 1. Market Scope & Methodology Chapter 2. Market Overview 2.1 Introduction 2.1.1 Overview 2.1.1.1 Market composition & scenarios 2.2 Key Factors Impacting the Market 2.2.1 Market Drivers 2.2.2 Market Restraints Chapter 3. Global Lysosomal Disease Treatment Market by Disease Type 3.1 Global Gaucher's Diseases Market by Region 3.2 Global Fabry Diseases Market by Region 3.3 Global Mucopolysaccharidosis Market by Region 3.4 Global Pompe's Syndrome Market by Region 3.5 Global Others Market by Region Chapter 4. Global Lysosomal Disease Treatment Market by Type of Therapy 4.1 Global Enzyme Replacement Therapy Market by Region 4.2 Global Substrate Reduction Therapy Market by Region 4.3 Global Stem Cell Therapy Market by Region 4.4 Global Others Market by Region Chapter 5. Global Lysosomal Disease Treatment Market by End User 5.1 Global Hospitals Market by Region 5.2 Global Clinics Market by Region 5.3 Global Others Market by Region Chapter 6. Global Lysosomal Disease Treatment Market by Region Chapter 7. Company Profiles 7.1 Merck & Co., Inc. 7.1.1 Company Overview 7.1.2 Financial Analysis 7.1.3 Segmental and Regional Analysis 7.1.4 Research & Development Expenses 7.2 Johnson & Johnson (Janssen Global Services, LLC) 7.2.1 Company Overview 7.2.2 Financial Analysis 7.2.3 Segmental &Regional Analysis 7.2.4 Research & Development Expenses 7.3 Eli Lilly And Company 7.3.1 Company Overview 7.3.2 Financial Analysis 7.3.3 Regional Analysis 7.3.4 Research & Development Expenses 7.3.5 Recent strategies and developments: 7.3.5.1 Acquisition and Mergers: 7.4 Pfizer, Inc. 7.4.1 Company Overview 7.4.2 Financial Analysis 7.4.3 Regional & Segmental Analysis 7.4.4 Research & Development Expense 7.4.5 Recent strategies and developments: 7.4.5.1 Partnerships, Collaborations, and Agreements: 7.5 Takeda Pharmaceutical Company Limited 7.5.1 Company Overview 7.5.2 Financial Analysis 7.5.3 Regional Analysis 7.5.4 Research & Development Expense 7.6 Sanofi S.A. 7.6.1 Company Overview 7.6.2 Financial Analysis 7.6.3 Segmental and Regional Analysis 7.6.4 Research & Development Expense 7.6.5 Recent strategies and developments: 7.6.5.1 Approvals and Trials: 7.7 Novartis AG 7.7.1 Company Overview 7.7.2 Financial Analysis 7.7.3 Segmental and Regional Analysis 7.7.4 Research & Development Expense 7.8 AstraZeneca PLC (Alexion Pharmaceuticals, Inc.) 7.8.1 Company Overview 7.8.2 Financial Analysis 7.8.3 Regional Analysis 7.8.4 Research & Development Expenses 7.9 BioMarin Pharmaceutical Inc. 7.9.1 Company Overview 7.9.2 Financial Analysis 7.9.3 Regional Analysis 7.9.4 Research & Development Expenses 7.9.5 Recent strategies and developments: 7.9.5.1 Approvals and Trials: 7.10. Sigilon Therapeutics, Inc. 7.10.1 Company Overview 7.10.2 Financial Analysis 7.10.3 Research & Development Expenses For more information about this report visit Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

The origins of the serious cancer-like disease LCH have been identified by researchers. The findings may lead to new, targeted treatments. The origins of the serious cancer-like disease LCH have been identified by researchers from Karolinska Institutet in collaboration with Karolinska University Hospital. The findings presented in Science Immunology may lead to new, targeted treatments. Langerhans' Cell Histiocytosis (LCH) is a serious type of cancer-like disease that mainly affects children and can be fatal in severe cases. About five to ten children get the disease in Sweden every year, usually before the age of ten. LCH is a disease in which the cancer mutation occurs in the immune cells, which otherwise have the task of detecting and eliminating cancer cells. "The origin of the LCH cells has been discussed for decades. Some researchers are convinced that LCH is derived from a certain type of immune cell called dendritic cells, while others believe that they come from related cells called monocytes," says Egle Kvedaraite, a doctor and researcher at the Department of Medical Biochemistry and Biophysics at Karolinska Institutet and first author of the new study. The researchers from Karolinska Institutet together with scientists from Singapore Immunology Network and Newcastle University have now been able to show that both theories are close to the truth. The researchers combined so-called single-cell sequencing, the microscopy of samples and the tracking of cells from patients recruited from, among others, Karolinska University Hospital. They discovered that mutated LCH cells had properties similar to both monocytes and dendritic cells, as well as a relatively newly discovered type of dendritic cell, so-called dendritic cell type 3 (DC3). "Today we know that DC3 has a separate pathway of development, separate from other dendritic cells and monocytes, and knowledge of this was crucial in our study," says Egle Kvedaraite. Researchers discovered that the different cell types could communicate with each other to promote the development of LCH and thereby create a self-reinforcing effect. "Among the treatment options for LCH, targeted therapy can be successfully applied, but the disease comes back when the targeted treatment is discontinued. This poses a serious challenge for patients, as a lifelong treatment for children is not a good option given the side effects," says Egle Kvedaraite. This new understanding of the origin of this type of cancer has the potential to contribute to the development of new targeted treatments. "The findings could lead to a treatment aimed at eliminating the pathological cells," says Egle Kvedaraite. The research was funded by grants from the Erik and Edith Fernström Foundation for Medical Research, the Swedish Childhood Cancer Foundation, the Histiocytosis Association, the VIVA Foundation for children with cancer, the Wellcome Trust, the CRUK Biomarker Project, Histio UK and Bright Red.