Achromatopsia - Pipeline Insight, 2024

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

Achromatopsia Understanding

Achromatopsia: Overview

Achromatopsia is characterized by reduced visual acuity, pendular nystagmus, increased sensitivity to light (photophobia), a small central scotoma, eccentric fixation, and reduced or complete loss of color discrimination. All individuals with achromatopsia (achromats) have impaired color discrimination along all three axes of color vision corresponding to the three cone classes: the protan or long-wavelength-sensitive cone axis (red), the deutan or middle-wavelength-sensitive cone axis (green), and the tritan or short-wavelength-sensitive cone axis (blue). Most individuals have complete achromatopsia, with total lack of function of all three types of cones. Rarely, individuals have incomplete achromatopsia, in which one or more cone types may be partially functioning. The manifestations are similar to those of individuals with complete achromatopsia, but generally less severe.

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

This segment of the Achromatopsia 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.

Achromatopsia Emerging Drugs

AAV-CNGA3: Janssen Pharmaceutical AAV-CNGA3, gene therapy candidate designed to restore cone function, are delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. Janssen Enters Worldwide Collaboration and License Agreement with Meira GTx to Develop Gene Therapy Programs for Inherited Retinal Diseases. Under the terms of the agreement, MeiraGTx to grant Janssen an exclusive worldwide license to certain clinical assets in MeiraGTx’s inherited retinal disease portfolio. The companies have also formed a research collaboration to develop a pipeline of products addressing novel gene targets, giving Janssen the exclusive option to license new treatments for other inheritedretinaldiseases.

Achromatopsia: Therapeutic Assessment

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

Major Players in Achromatopsia

There are approx. 5+ key companies which are developing the therapies for Achromatopsia. The companies which have their Achromatopsia drug candidates in the most advanced stage, i.e. Phase I/II include, Janssen Pharmaceutical.

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

Achromatopsia 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.

Achromatopsia : Pipeline Development Activities

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

Achromatopsia Report Insights

• Achromatopsia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• MeiraGTx
• Janssen Pharmaceuticals
• Applied Genetic Technologies Corporation

Key Products

• Entacingene turiparvovec
• Aguracingene cadoparvovec
• rAAV2tYF-PR.1-hCNGA3

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