As per Global Insight services’ assessment, about 20+ prominent pharma and biotech giants are working on 20+ drugs in the Progressive Mucopolysaccharidosis – pipeline landscape globally. Progressive Mucopolysaccharidosis Pipeline Insight, 2023” report by Global Insight Services provides a comprehensive analysis of the ongoing clinical development activities and growth prospect.
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Mucopolysaccharidosis is a group of metabolic inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). There are seven types of mucopolysaccharidosis : mps type I (hurler syndrome), mps type II (hunter syndrome), mps type III (sanfilippo syndrome), mps type IV (morquio syndrome), mps type VI (maroteaux-lamy syndrome), mps type VII (sly syndrome), and mps type IX (hyaluronidase deficiency).
Mucopolysaccharidosis is caused by the absence or malfunctioning of certain enzymes in the body needs to break down molecules called glycosaminoglycans.
Mucopolysaccharidosis main symptoms are abnormal bones in the spine, claw hands, cloudy corneas, deafness, heart valve problems, liver and spleen enlargement, and changing facial features.
Mucopolysaccharidosis is diagnosed by low iduronate 2-sulfatase enzyme activity and elevated plasma and or urine GAGs.
Mucopolysaccharidosis is treated by enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation.
The pipeline of Mucopolysaccharidosis treatment market consists of a great variable of drugs. N-Sulfoglucosamine Sulfohydrolase (SGSH) Replacements, Iduronate-2-Sulfatase (IDS) Gene Replacement, and some others are the most highlighted class of this indication. Ongoing research and current trials have the potential to change the market.
More than 200 key players, including pharma giants such as Denali Therapeutics Inc, REGENXBIO Inc, JCR Pharmaceuticals Co Ltd, ArmaGen Inc, and others are working to bring novel therapies that overcome the roadblocks of AML treatment pipeline.
Report Highlights
Global Insight Service’s, “Mucopolysaccharidosis – Pipeline Landscape, 2023,” an overview of the Mucopolysaccharidosis pipeline drugs. This report covers detailed insights on Mucopolysaccharidosis drugs under development, assessment by target, mechanism of action, route of administration and molecule type. Product pipeline by companies, stage of development and key regulatory designations, deals and milestones have been presented to gain insights and thus help industry participants for decision making. Mucopolysaccharidosis pipeline report helps gaining insights on drugs which are under development stage of drug development process across globally. Global Insight Services aims to cover all the drug attributes, deals, regulatory milestones and all the upcoming milestones.
Recent Developments
In February 2023, Regenxbio Inc. announced additional positive interim data from the Phase I/II/ III CAMSIITE trial of RGX-121 for the treatment of patients up to 5 years of age diagnosed with MPS II. The results were presented at the 19th Annual WORLD Symposium.
In February 2023, Denali Therapeutics Inc. presented interim results from the ongoing open-label, single-arm Phase I/II study of DNL310 in children with MPS II, including data from additional participants and up to 104 weeks of treatment, and preclinical data on DNL126 for MPS IIIA presented at the 19th Annual WORLD Symposium.
In January 2023, Immunosoft received USD 8 million from the California Institute for Regenerative Medicine (CIRM). This funding is to support the Phase I study of ISP-001 in MSP I.
In September 2022, Immusoft received US FDA clearance for an Investigational New Drug Application (IND) for ISP-001 in the treatment of MPSI.
In October 2019, Allievex Corp. entered into a worldwide licence agreement with BioMarin Pharmaceutical Inc. for the development and commercialization of AX 250 to treat mucopolysaccharidosis type IIIb (MPS IIIB).
Key Products
ABO-102 (UX111) is under development for the treatment of mucopolysaccharidosis (MPS) IIIA. In May 2022, Ultragenyx Pharmaceutical Inc. and Abeona Therapeutics Inc. entered into a licence agreement for AAV gene therapy ABO-102 for the treatment of Sanfilippo syndrome type A (MPS IIIA). The US FDA granted Abeona Therapeutics Inc Regenerative Medicine Advanced Therapy (RMAT) designation in April 2018. Abeona Therapeutics Inc received fast track designation from the US FDA in October 2016. In October 2016, Abeona Therapeutics Inc. received orphan drug designation from the European Medicines Agency (EMA) and also received rare paediatric disease designations.
AGT-184 is under development for the treatment of mucopolysaccharidosis I. In August 2018, ArmaGen received orphan drug designation from the US FDA for AGT-184.
HMI-203 is under development for the treatment of mucopolysaccharidosis type I. In 2021, Homology Medicines Inc received Orphan Medicinal Product Designations (OMPD) and Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) for HMI-203 for mucopolysaccharidosis type I (MPS I).
Methodology
The research process includes extensive secondary research on public domain and other authentic sources carried out to add or update the pipeline products information. The secondary research sources include, but are not limited to company websites, annual reports, financial reports, company pipeline chart, investor presentations and SEC filings, journals and conferences, clinical trials registries.
Scope
The pipeline landscape report consists of pipeline product based on several stages of development ranging from discovery to Pre-Registration. The report provides a review of pipeline therapeutics for Mucopolysaccharidosis (Hormonal Disorders) by companies based on information derived from company and industry-specific sources. The pipeline report covers assessment of Mucopolysaccharidosis (Hormonal Disorders) therapeutics by mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. Comprehensive profiles of the pipeline products with details such as company overview, development stage; molecule type, target, mechanism of action, route of administration, dosage form, regulatory designations, key deals and key upcoming milestones.
Report Covers the Emerging Pipeline Products Under Different Phases of Clinical Development Like –
– Late-stage products (Phase III)
– Mid-stage products (Phase II)
– Early-stage product (Phase I)
– Pre-clinical and Discovery stage candidates
– Discontinued & Inactive candidates
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