Adrenoleukodystrophy - Pipeline Insight, 2024

This “Adrenoleukodystrophy (ALD) - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Adrenoleukodystrophy (ALD) 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

Adrenoleukodystrophy (ALD) Understanding

Adrenoleukodystrophy (ALD): Overview

Adrenoleukodystrophy (ALD) is a rare, X-linked metabolic disorder. ALD is caused by mutations in the ABCD1 gene that affect the production of adrenoleukodystrophy protein (ALDP) and subsequently cause toxic accumulation of very long-chain fatty acids (VLCFAs) primarily in the adrenal cortex and white matter of the brain and spinal cord. This brain disorder destroys myelin, the protective sheath that surrounds the brain’s neurons. Childhood Cerebral ALD is the most devastating form of adrenoleukodystrophy. Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Diagnosis of ALD is based on physical examination and various tests like blood testing, MRI, vision screening, skin biopsy and fibroblast cell culture. Stem cell transplant effective treatment option for cerebral ALD. Gene therapy is being evaluated as a potential treatment for cerebral ALD.

Adrenoleukodystrophy (ALD) - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Adrenoleukodystrophy (ALD) pipeline landscape is provided which includes the disease overview and Adrenoleukodystrophy (ALD) treatment guidelines. The assessment part of the report embraces, in depth Adrenoleukodystrophy (ALD) 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, Adrenoleukodystrophy (ALD) 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 Adrenoleukodystrophy (ALD) R&D. The therapies under development are focused on novel approaches to treat/improve Adrenoleukodystrophy (ALD).

Adrenoleukodystrophy (ALD) Emerging Drugs Chapters

This segment of the Adrenoleukodystrophy (ALD) 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.

Adrenoleukodystrophy (ALD) Emerging Drugs

Elivaldogene autotemcel: bluebird bioEli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of a functional gene allows patients to produce the adrenoleukodystrophy protein (ALDP). In October 2020, Bluebird bio announced that the European Medicines Agency (EMA) accepted the company’s marketing authorization application (MAA) for elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). The EMA granted eli-cel gene therapy Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel. In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the EMA granted an accelerated assessment to eli-cel. The U.S. FDA granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD.

Leriglitazone: Minoryx TherapeuticsLeriglitazone (MIN-102) is a novel, orally bioavailable and selective PPAR gamma agonist. Leriglitazone (MIN-102), by activating PPAR gamma, modulates the expression of genes involved in mitochondrial biogenesis (PGC-1a) and therefore restores the lost energy balance, decreases oxidative stress and restores mitochondrial function caused by accumulation of VLCFAs. The drug is in Phase II/III clinical development for the treatment of adrenoleukodystrophy, a registration enabling Phase 2 in cerebral ALD (cALD) and in a Phase 2 in Friedreich’s ataxia.

Adrenoleukodystrophy (ALD): Therapeutic Assessment

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

Major Players in Adrenoleukodystrophy (ALD)

There are approx. 8+ key companies which are developing the therapies for Adrenoleukodystrophy (ALD). The companies which have their Adrenoleukodystrophy (ALD) drug candidates in the most advanced stage, i.e. Phase III include, bluebird bio.

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

Adrenoleukodystrophy (ALD) 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
• Parenteral
• Intravitreal
• Subretinal
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Adrenoleukodystrophy (ALD): Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Adrenoleukodystrophy (ALD) 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 Adrenoleukodystrophy (ALD) drugs.

Adrenoleukodystrophy (ALD) Report Insights

• Adrenoleukodystrophy (ALD) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• bluebird bio
• Applied Genetic Technologies Corporation
• Poxel
• Minoryx Therapeutics
• Orpheris
• MedDay Pharmaceuticals
• Viking Therapeutics

Key Products

• Elivaldogene autotemcel

Research programme: Adrenoleukodystrophy gene therapy- Research programme: AMP activated protein kinase therapeutics

• Leriglitazone
• OP 101
• Biotin
• VK 0214

Please note: The report will be dispatched within 2-3 days