DelveInsight’s, “Adrenoleukodystrophy Pipeline Insight, 2023,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in the Adrenoleukodystrophy pipeline landscape. It covers the Adrenoleukodystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Adrenoleukodystrophy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key Takeaways from the Adrenoleukodystrophy Pipeline Report

• DelveInsight’s Adrenoleukodystrophy pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Adrenoleukodystrophy treatment.
• The leading companies working in the Adrenoleukodystrophy Market include Bluebird bio, Applied Genetic Technologies Corporation, Poxel, Minoryx Therapeutics, Orpheris, MedDay Pharmaceuticals, Viking Therapeutics, and others.
• Promising Adrenoleukodystrophy Pipeline Therapies in the various stages of development include Leriglitazone, MIN-102, VK0214, MD1003 100 mg capsule, SBT101, Cholic Acids, OP-101, and others.
• May 2023: Viking Therapeutics Inc. announced a study of Phase 1 clinical trials for VK0214. Subjects will be administered multiple doses of VK0214 in a cohort dose escalation process. A total of up to 36 subjects will be enrolled in the study, into one of three dose cohorts. For each cohort, up to 12 subjects will be randomized to receive VK0214 or placebo in a 3:1 ratio so that there will be a total of up to 9 subjects for each of the active doses and up to 3 subjects dosed with placebo in each cohort.
• September 2023: SwanBio Therapeutics Inc. announced a study of Phase 1 & 2 clinical trials for SBT101. This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years.
• September 2023: BlueBird Bio announced a study of Phase 3 clinical trials of Lenti-D. The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant’s blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing.
• October 2023: Minoryx Therapeutics S.L announced a study of Phase 3 clinical trials for Leriglitazone. A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy. A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy.
• October 2023: Mirium Pharmaceuticals Inc. announced a study of Phase 3 clinical trials for Cholic Acids. A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism. The study was begun with a single study site at Cincinnati Children’s Hospital Medical Center (CCHMC), but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the center’s screening/diagnostic program. Patients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study.

 

Request a sample and discover the recent advances in Adrenoleukodystrophy Treatment Drugs @ Adrenoleukodystrophy Pipeline Report

 

In the Adrenoleukodystrophy pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, Adrenoleukodystrophy 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.

 

Adrenoleukodystrophy 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.

 

Find out more about Adrenoleukodystrophy Therapeutics Assessment @ Adrenoleukodystrophy Preclinical and Discovery Stage Products

 

Adrenoleukodystrophy Emerging Drugs Profile

• Elivaldogene autotemcel: bluebird bio
• Leriglitazone: Minoryx Therapeutics

 

Adrenoleukodystrophy Pipeline Therapeutics Assessment

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

 

DelveInsight’s Adrenoleukodystrophy pipeline 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 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

 

Adrenoleukodystrophy Pipeline Products have been categorized under various Molecule types such as

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

 

Learn more about the emerging Adrenoleukodystrophy Pipeline Therapies @ Adrenoleukodystrophy Clinical Trials Assessment

 

Scope of the Adrenoleukodystrophy Pipeline Report

• Coverage- Global
• Adrenoleukodystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Adrenoleukodystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Adrenoleukodystrophy Companies- Bluebird bio, Applied Genetic Technologies Corporation, Poxel, Minoryx Therapeutics, Orpheris, MedDay Pharmaceuticals, Viking Therapeutics, and others.
• Adrenoleukodystrophy Pipeline Therapies- Leriglitazone, MIN-102, VK0214, MD1003 100 mg capsule, SBT101, Cholic Acids, OP-101, and others.

 

Dive deep into rich insights for new drugs for Adrenoleukodystrophy Treatment, Visit @ Adrenoleukodystrophy Market Drivers and Barriers, and Future Perspective

 

Table of Content

1. Introduction
2. Executive Summary
3. Adrenoleukodystrophy (ALD): Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Adrenoleukodystrophy (ALD) – DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Adrenoleukodystrophy (ALD) Collaboration Deals
9. Late Stage Products (Preregistration)
10. Elivaldogene autotemcel: bluebird bio
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II/III)
13. Leriglitazone: Minoryx Therapeutics
14. Drug profiles in the detailed report…..
15. Early Stage Products (Phase I)
16. OP 101: Orpheris
17. Drug profiles in the detailed report…..
18. Preclinical/Discovery Stage Products
19. Research programme: Adrenoleukodystrophy gene therapy: AGTC
20. Drug profiles in the detailed report…..
21. Inactive Products
22. Adrenoleukodystrophy (ALD) Key Companies
23. Adrenoleukodystrophy (ALD) Key Products
24. Adrenoleukodystrophy (ALD)- Unmet Needs
25. Adrenoleukodystrophy (ALD)- Market Drivers and Barriers
26. Adrenoleukodystrophy (ALD)- Future Perspectives and Conclusion
27. Adrenoleukodystrophy (ALD) Analyst Views
28. Adrenoleukodystrophy (ALD) Key Companies
29. Appendix

 

For further information on the Adrenoleukodystrophy Pipeline therapeutics, reach out to Adrenoleukodystrophy Unmet Needs and Analyst Views

 

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