Metachromatic Leukodystrophy (MLD) - Pipeline Insight, 2024

This “Metachromatic Leukodystrophy - Pipeline Insight, 2024,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Metachromatic Leukodystrophy 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

Metachromatic Leukodystrophy Understanding

Metachromatic Leukodystrophy: Overview

Metachromatic leukodystrophy (MLD) is a rare hereditary disease characterized by accumulation of fats called sulfatides. This causes the destruction of the protective fatty layer (myelin sheath) surrounding the nerves in both the central nervous system and the peripheral nervous system. There are three types of MLD based on the age symptoms appear: late-infantile MLD, juvenile MLD, and adult MLD.

MLD is caused by changes (mutations) in the ASA gene (also known as ARSA) and, in rare cases, the PSAP gene. MLD is first suspected by recognizing the characteristic pattern of progressive impairment. In the late-infantile form, the first signs are often difficult walking, which can present as new inability to fully lift the feet while walking (foot drop) or by toe walking. For adult MLD, the first signs are slurred speech and behavioral issues that include difficulty in school, behavior changes, and decreased ability in school. Individuals with juvenile MLD can present with motor or cognitive symptoms. In pre- or minimally symptomatic children, stem cell transplantation can be considered. Otherwise, the main treatment is supportive and focused on symptomatic relief.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Metachromatic Leukodystrophy R&D. The therapies under development are focused on novel approaches for Metachromatic Leukodystrophy.

Metachromatic Leukodystrophy Emerging Drugs Chapters

This segment of the Metachromatic Leukodystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Metachromatic Leukodystrophy Emerging Drugs

OTL-200: Orchard Therapeutics OTL-200 (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), referred to as Libmeldy in the European Union, is an ex vivo autologous hematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020. OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) or any other health authority.

TAK-611: Takeda TAK-611, a recombinant cerebroside sulfatase is being developed by Takeda as an enzyme replacement therapy for the treatment of metachromatic leukodystrophy (MLD), a rare hereditary disease characterised by the accumulation of toxic lipids that destroy the myelin sheath surrounding neurons. It is currently in phase II stage of development for metachromaticleukodystrophy.

Metachromatic Leukodystrophy: Therapeutic Assessment

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

Major Players working on Metachromatic Leukodystrophy

There are approx. 6+ key companies which are developing the Metachromatic Leukodystrophy. The companies which have their Metachromatic Leukodystrophy drug candidates in the most advanced stage, i.e. Registered include, Orchard Therapeutics.

Phases

This report covers around 6+ products under different phases of clinical development like

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

Route of Administration

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

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule

Product Type

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

Metachromatic Leukodystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Metachromatic Leukodystrophy 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 Metachromatic Leukodystrophy drugs.

Metachromatic Leukodystrophy Report Insights

• Metachromatic Leukodystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Metachromatic Leukodystrophy Report Assessment

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

Key Questions

Current Scenario and Emerging Therapies:

• How many companies are developing Metachromatic Leukodystrophy drugs?
• How many Metachromatic Leukodystrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for Metachromatic Leukodystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Metachromatic Leukodystrophy 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 Metachromatic Leukodystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Takeda
• Denali Therapeutics
• Orchard Therapeutics
• Homology Medicines
• Passage Bio
• ArmaGen Technologies

Key Products

• TAK-611
• ETV-ARSA
• OTL-200
• HMI-202
• PBML-04
• AGT 183