Mucopolysaccharidosis Type I - Pipeline Insight, 2024

This “Mucopolysaccharidosis Type I- Pipeline Insight, 2024” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Mucopolysaccharidosis Type I pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Mucopolysaccharidosis Type I: Understanding

Mucopolysaccharidosis Type I: Overview

Mucopolysaccharidosis Type I (MPS I) is a rare genetic disorder that falls under the larger group of lysosomal storage diseases. It is caused by a deficiency of the enzyme alpha-L-iduronidase, which is crucial for breaking down glycosaminoglycans (GAGs), previously known as mucopolysaccharides. The accumulation of GAGs in various tissues and organs leads to the symptoms and complications associated with MPS I. The disorder can present in a spectrum of severity, traditionally classified into three subtypes: Hurler syndrome (severe), Hurler-Scheie syndrome (intermediate), and Scheie syndrome (mild).

The signs and symptoms of MPS I vary widely depending on the severity of the enzyme deficiency. In its most severe form, Hurler syndrome, symptoms typically appear within the first year of life. These can include coarse facial features, frequent respiratory infections, hepatosplenomegaly (enlarged liver and spleen), and developmental delays. Children with Hurler syndrome often experience progressive physical and mental deterioration, leading to severe disability and a significantly shortened life expectancy. In milder forms, such as Scheie syndrome, symptoms might not manifest until later in childhood or even adulthood and are generally less severe. Common symptoms across the spectrum include joint stiffness, vision and hearing impairments, heart valve abnormalities, and skeletal deformities.

MPS I is caused by mutations in the IDUA gene, which provides instructions for making the alpha-L-iduronidase enzyme. The lack of functional enzyme results in the accumulation of dermatan sulfate and heparan sulfate, types of GAGs, in the lysosomes of cells. This buildup disrupts normal cellular function, leading to the progressive damage of tissues and organs. The pathophysiology of MPS I involves a complex interplay of cellular and molecular mechanisms that lead to inflammation, fibrosis, and cellular dysfunction across multiple systems.

Diagnosis of MPS I typically involves a combination of clinical evaluation, biochemical testing, and genetic analysis. Initial clinical suspicion might arise from observing characteristic physical symptoms and developmental delays. Biochemical tests, such as enzyme assays, can confirm reduced alpha-L-iduronidase activity. Genetic testing is used to identify mutations in the IDUA gene, providing a definitive diagnosis. Early diagnosis is critical for managing the disease effectively and improving outcomes.

Treatment for MPS I includes enzyme replacement therapy (ERT), hematopoietic stem cell transplantation (HSCT), and supportive care to manage symptoms and improve quality of life. ERT with laronidase (Aldurazyme) can help reduce the accumulation of GAGs and alleviate some symptoms, particularly if started early. HSCT, typically performed in severe cases like Hurler syndrome, can provide a source of healthy enzyme-producing cells but carries significant risks and is most effective when done before the onset of severe symptoms. Supportive treatments might include surgeries for heart or skeletal issues, physical therapy, and interventions for respiratory and hearing problems. Ongoing research aims to improve existing therapies and develop new approaches, such as gene therapy, to address the underlying genetic defect.

"Mucopolysaccharidosis Type I- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Mucopolysaccharidosis Type I pipeline landscape is provided which includes the disease overview and Mucopolysaccharidosis Type I treatment guidelines. The assessment part of the report embraces, in depth Mucopolysaccharidosis Type I commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Mucopolysaccharidosis Type I collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Mucopolysaccharidosis Type I R&D. The therapies under development are focused on novel approaches to treat/improve Mucopolysaccharidosis Type I.

Mucopolysaccharidosis Type I Emerging Drugs Chapters

This segment of the Mucopolysaccharidosis Type I report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Mucopolysaccharidosis Type I Emerging Drugs

OTL 203: Orchard Therapeutics

OTL-203 is an investigational ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare pediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority. Currently, the drug is in Phase III stage of its development for the treatment of Mucopolysaccharidosis Type I.

JR 171: JCR Pharmaceuticals

JR-171, is an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® BBB technology. JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. The trial is also scheduled for enrolling patients in the US. Currently, the drug is in Phase I/II stage of its development for the treatment of Mucopolysaccharidosis Type I.

Mucopolysaccharidosis Type I: Therapeutic Assessment

This segment of the report provides insights about the different Mucopolysaccharidosis Type I drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Mucopolysaccharidosis Type I

• There are approx. 8+ key companies which are developing the therapies for Mucopolysaccharidosis Type I. The companies which have their Mucopolysaccharidosis Type I drug candidates in the most advanced stage, i.e. phase III include, Orchard Therapeutics.

Phases

This report covers around 8+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Mucopolysaccharidosis Type I pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Mucopolysaccharidosis Type I: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Mucopolysaccharidosis Type I therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Mucopolysaccharidosis Type I drugs.

Mucopolysaccharidosis Type I Report Insights

• Mucopolysaccharidosis Type I Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Mucopolysaccharidosis Type I Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Mucopolysaccharidosis Type I drugs?
• How many Mucopolysaccharidosis Type I drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Mucopolysaccharidosis Type I?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Mucopolysaccharidosis Type I therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Mucopolysaccharidosis Type I and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Orchard Therapeutics
• JCR Pharmaceuticals
• REGENXBIO

Key Products

• OTL 203
• JR 171
• RGX 111

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