Ataxia Telangiectasia - Pipeline Insight, 2024

This “Ataxia Telangiectasia- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Ataxia Telangiectasia 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

Ataxia Telangiectasia: Understanding

Ataxia Telangiectasia: Overview

Ataxia telangiectasia (AT) is a complex genetic neurodegenerative disorder that may become apparent during infancy or early childhood. The disorder is characterized by progressively impaired coordination of voluntary movements (ataxia), the development of reddish lesions of the skin and mucous membranes due to permanent widening of groups of blood vessels (telangiectasia), and impaired functioning of the immune system (i.e., cellular and humoral immunodeficiency), resulting in increased susceptibility to upper and lower respiratory infections (sinopulmonary infections). Individuals with AT also have an increased risk of developing certain malignancies, particularly of the lymphatic system (lymphomas), the blood-forming organs (e.g., leukemia), and the brain. In those with AT, progressive ataxia typically develops during infancy and may initially be characterized by abnormal swaying of the head and trunk. As the disease progresses, the condition leads to an inability to walk (ambulation) by late childhood or adolescence. Ataxia is often accompanied by difficulty speaking (dysarthria), drooling; and an impaired ability to coordinate certain eye movements (oculomotor apraxia), including the occurrence of involuntary, rapid, rhythmic motions (oscillations) of the eyes while attempting to focus upon certain objects (fixation nystagmus). Affected children may also develop an unusually stooped posture and irregular, rapid, jerky movements that may occur in association with relatively slow, writhing motions (choreoathetosis). In addition, telangiectasias may develop by mid-childhood, often appearing on sun-exposed areas of the skin, such as the bridge of the nose, the ears, and certain regions of the extremities, as well as the mucous membranes of the eyes (conjunctiva).AT is inherited as an autosomal recessive trait. The disorder is caused by changes (mutations) of a gene known as ATM (for “AT mutated”) that has been mapped to the long arm (q) of chromosome 11 (11q22.3). The ATM gene controls (encodes for) the production of an enzyme that plays a role in regulating cell division following DNA damage. Ataxia telangiectasia usually begins during infancy (between one and three years of age) and often affects more than one child in a family. Males and females may be affected in equal numbers. In the United States, the prevalence is approximately one in 40,000-100,000 live births. A diagnosis of ataxia telangiectasia is made based upon a detailed patient history, a thorough clinical evaluation, identification of characteristic symptoms, and a variety of specialized tests including blood tests, magnetic resonance imaging (MRI), and karyotyping.

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

Ataxia Telangiectasia Emerging Drugs Chapters

This segment of the Ataxia Telangiectasia 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.

Ataxia Telangiectasia Emerging Drugs

MBM-01: Matrix Biomed The active compound in MBM-01 has been shown to supplant the overall role of ATM by reducing oxidative stress, reducing DNA double strand breaks, and decreasing programmed cell death (in healthy cells). Accordingly, MBM-01 represents a potential breakthrough therapy for patients afflicted with A-T by activating the genes NRF2 and BDNF in the brain. NRF2 and BDNF are essential genes that are down regulated in A-T. Upregulation of the genes BDNF and NRF2 have the potential to prevent the DNA damage, oxidative stress, and neurodegeneration observed in A-T patients. Currently, the drug is being developed in the Phase II/III stage of clinical trial evaluation for the treatment of Ataxia Telangiectasia.

IB-1001: IntraBio Inc IntraBio is conducting multinational clinical trials with its lead compound, IB1001 (N-acetyl-L-leucine) for the treatment of three orphan diseases: Niemann-Pick disease type C (NPC); GM2 Gangliosidosis (Tay-Sachs and Sandhoff); Ataxia-Telangiectasia (A-T). Currently, the drug is being developed in the Phase II stage of clinical trial evaluation for the treatment of AtaxiaTelangiectasia.

Ataxia Telangiectasia: Therapeutic Assessment

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

Major Players in Ataxia Telangiectasia

There are approx. 5+ key companies which are developing the therapies for Ataxia Telangiectasia. The companies which have their Ataxia Telangiectasia drug candidates in the most advanced stage, i.e. phase II /III include, Matrix Biomed.

Phases

This report covers around 5+ 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

Ataxia Telangiectasia 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.

Ataxia Telangiectasia: Pipeline Development Activities

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

Ataxia Telangiectasia Report Insights

• Ataxia Telangiectasia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Matrix Biomed
• IntraBio Inc
• EryDel
• Acasti Pharma

Key Products

• EryDex
• MBM-01
• IB-1001
• GTX-102

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