X-linked retinitis pigmentosa - Pipeline Insight, 2024

This “X-linked Retinitis Pigmentosa - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in X-linked Retinitis Pigmentosa 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

X-linked Retinitis Pigmentosa Understanding

X-linked Retinitis Pigmentosa: Overview

X-linked Retinitis Pigmentosa is a rare, hereditary progressive genetic disorder associated with the gene mutation in chromosome X of an individual. This disorder is characterized by the progressive degeneration of photoreceptor cells in the retina and gradually leading to peripheral vision loss. Total population affected by X-linked Retinitis Pigmentosa is 1 in 4000 people across the world. Also, as it is a recessive disorder, males are more affected as compared to females, who have two X chromosomes making them carrier for the disorder. Early symptoms for X-linked Retinitis Pigmentosa includes night blindness, and progressive loss of visual field.

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

X-linked Retinitis Pigmentosa Emerging Drugs Chapters

This segment of the X-linked Retinitis Pigmentosa 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.

X-linked Retinitis Pigmentosa Emerging Drugs

BIIB 112 (NSR-RPGR): Biogen Biogen’s lead drug candidate, BIIB 112 (NSR-RPGR), is a class of gene therapy molecule which consists of standard AAV8 vector carrying a codon-optimized RPGR gene. The drug is in Phase II/III stage for the treatment of rare X-linked Retinitis Pigmentosa and has been designated orphan drug status. The trial is carried out to evaluate the tolerability as well as the stability of the drug in improving centralvision.

4D-125: 4 D Molecular Therapeutics4D Molecular Therapeutics “lead” drug 4D-125 is currently being evaluated for the safety and maximum tolerance dose for the treatment of X-linked Retinitis Pigmentosa. 4D-125 is an AAV gene therapy with an optimized vector designed to deliver a functional RPGR gene copy to the photoreceptors in retina.

X-linked Retinitis Pigmentosa: Therapeutic Assessment

This segment of the report provides insights about the different X-linked Retinitis Pigmentosa drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in X-linked Retinitis Pigmentosa

There are approx. 5+ key companies which are developing the therapies for X-linked Retinitis Pigmentosa. The companies which have their X-linked Retinitis Pigmentosa drug candidates in the mid to advanced stage, Phase II/III include, Biogen and others.

Phases

This report covers around 5+ products under different phases of clinical development like

• Late-stage products (Phase II/III)
• Mid-stage products (Phase II and Phase I/II)
• 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

X-linked Retinitis Pigmentosa pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Subcutaneous
• Intravenous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecules
• Natural metabolites
• Monoclonal antibodies

Product Type

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

X-linked Retinitis Pigmentosa: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses X-linked Retinitis Pigmentosa 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 X-linked Retinitis Pigmentosa drugs.

X-linked Retinitis Pigmentosa Report Insights

• X-linked Retinitis Pigmentosa Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Biogen
• 4 D Molecular therapeutics
• Applied Genetics Technology Corporation

Key Products

• BIIB-112 (NSR-RPGR)
• 4D-125
• AGTC501

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