Stargardt disease - Pipeline Insight, 2024

This “Stargardt disease - Pipeline Insight, 2024,” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in Stargardt disease 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

Stargardt disease Understanding

Stargardt disease: Overview

Stargardt disease is caused by inheritance of a faulty gene: the ABCA4 gene. Genes are in pairs, one inherited from the father and one from the mother. If abnormal ABCA4 genes are inherited from both parents, then the disease will occur. Because of these abnormal genes, a build-up of waste material from metabolism called lipofuscin occurs in the retina. This material should be cleared away but because of the disease it is not and damages the retina, and some of the retinal cells die. This causes loss of vision, but the amount of loss varies. There are many different forms of the faulty gene and, as a result, some people have more severe disease than others.

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

This segment of the Stargardt disease 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.

Stargardt disease Emerging Drugs

Tinlarebant: Belite BioLBS-008 or Tinlarebant is a potent small molecule RBP4 antagonist that has been specifically designed to reduce the delivery of retinol to the eye as a therapeutic approach towards reducing the accumulation of cytotoxic bisretinoids, preserving the integrity of retinal tissues, and ultimately slowing or preventing loss of vision. The delivery of retinol to the RPE requires RBP4 and the RPE expresses a specific RBP4 receptor (STRA6) to regulate vitamin A uptake. Other extrahepatic tissues do not require delivery of retinol bound to RBP4 and do not express the RBP4 receptor. These tissues are able to take up vitamin A bound to non-specific carriers such as lipoproteins, triglycerides, and albumin. The drug is in Phase III clinical evaluation for the treatment of Stargardt disease.

Stargardt disease: Therapeutic Assessment

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

Major Players in Stargardt disease

There are approx. 18+ key companies which are developing the therapies for Stargardt disease. The companies which have their Stargardt disease drug candidates in the most advanced stage, i.e. Phase III include, Belite Bio.

Phases

This report covers around 20+ 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

Stargardt disease 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
• Intravenous
• Subcutaneous
• 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.

Stargardt disease: Pipeline Development Activities

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

Stargardt disease Report Insights

• Stargardt disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• IVERIC bio
• Nanoscope Therapeutics
• Alkeus Pharmaceuticals
• Kubota Vision
• Astellas Pharma
• Stargazer Pharmaceuticals, Inc.
• Belite Bio
• ProQR Therapeutics
• ReVision Therapeutics

Key Products

• Avacincaptad pegol
• MCO 010
• Retinol deuterated
• Emixustat
• MA09 hRPE
• STG-001
• Tinlarebant
• QR1011
• REV 0100

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