Mucopolysaccharidosis type I (MPS I) - Pipeline Insight, 2024

This “Mucopolysaccharidosis type I (MPS I)- Pipeline Insight, 2024” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Mucopolysaccharidosis type I (MPS 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 (MPS I): Understanding

Mucopolysaccharidosis type I (MPS I): Overview

Mucopolysaccharidosis type I (MPS I) is a progressive multisystem disorder with features ranging over a continuum of severity. While affected individuals have traditionally been classified as having one of three MPS I syndromes (Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome), no easily measurable biochemical differences have been identified and the clinical findings overlap. Affected individuals are best described as having either a phenotype consistent with either severe (Hurler syndrome) or attenuated MPS I, a distinction that influences therapeutic options. Subdivisions of Mucopolysaccharidosis Type I includes: attenuated mucopolysaccharidosis type I, severe mucopolysaccharidosis type I, Hurler syndrome (reserved for severely affected individuals), Hurler-Scheie syndrome (preferred term is attenuated mucopolysaccharidosis type I), Scheie syndrome (preferred term is attenuated mucopolysaccharidosis type I). MPS I is caused by genetic changes in the IDUA gene. These genetic changes lead to reduced levels or the complete lack of the IDUA enzyme. Without the proper amount of this enzyme, large sugar molecules called glycosaminoglycans (GAGs) accumulate within cells called lysosomes. This causes the lysosomes to increase in size, causing many different organs and tissues of the body to become enlarged. This leads to the medical problems seen in the condition. The specific signs and symptoms seen in MPS I are highly variable and depends on numerous factors including what form of MPS I a child has, when treatment was first started, and how an individual child responds to various treatment options. It is important to note that MPS I is a progressive disorder and therefore the symptoms that a child has are also related to the age of the child at the time of diagnosis. The age of onset, disease severity, degree of intellectual disability, and rate of progression vary significantly among affected individuals. A diagnosis of mucopolysaccharidosis type I is based upon identification of characteristic symptoms, a detailed patient and family history, a thorough clinical evaluation and a variety of specialized tests. A diagnosis may be suspected in infants with characteristic early signs. Currently newborn screening for MPS I is being implemented in many areas. There are three components in the treatment of MPS I: replacing the enzyme that is missing, alleviating specific symptoms of disease and providing the family with genetic counseling.

Mucopolysaccharidosis type I (MPS 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 (MPS I) pipeline landscape is provided which includes the disease overview and Mucopolysaccharidosis type I (MPS I) treatment guidelines. The assessment part of the report embraces, in depth Mucopolysaccharidosis type I (MPS 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 (MPS 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 (MPS I) R&D. The therapies under development are focused on novel approaches to treat/improve Mucopolysaccharidosis type I (MPS I).

Mucopolysaccharidosis type I (MPS I) Emerging Drugs Chapters

This segment of the Mucopolysaccharidosis type I (MPS 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 (MPS I) Emerging Drugs

JR 171: JCR PharmaceuticalsJR-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 a-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. Currently JR-171 is undergoing a global Phase 1/2 clinical trial and the first patient in Japan was dosed in October, 2020 and in Brazil in March, 2021. The trial is also scheduled for enrolling patients in the US.

RGX 111: REGENXBIORGX-111 is designed to use the AAV9 vector to deliver the human a-l-iduronidase (IDUA) gene to the central nervous system (CNS). RGX-111 is REGENXBIO product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I). Currently, REGENXBIO is enrolling patients in the Phase I/II trial of RGX-111 for the treatment of MPS I. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designation from the FDA.

Mucopolysaccharidosis type I (MPS I): Therapeutic Assessment

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

Major Players in Mucopolysaccharidosis type I (MPS I)

There are approx. 12+ key companies which are developing the therapies for Mucopolysaccharidosis type I (MPS I). The companies which have their Mucopolysaccharidosis type I (MPS I) drug candidates in the most advanced stage, i.e. Phase I/II, JCR Pharmaceuticals.

Phases

This report covers around 12+ 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 (MPS 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Mucopolysaccharidosis type I (MPS 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 (MPS 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 (MPS I) drugs.

Mucopolysaccharidosis type I (MPS I) Report Insights

• Mucopolysaccharidosis type I (MPS I) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Mucopolysaccharidosis type I (MPS 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 (MPS I) drugs?
• How many Mucopolysaccharidosis type I (MPS 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 (MPS I)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Mucopolysaccharidosis type I (MPS 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 (MPS I) and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• JCR Pharmaceuticals
• REGENXBIO
• Talaris Therapeutics Inc.
• Orchard Therapeutics
• Paradigm Biopharma
• Jupiter Neurosciences

Key Products

• JR 171
• RGX 111
• hematopoietic stem cell infusion
• OTL 203
• Pentosan polysulfate
• JNS 101

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