Choroideremia - Pipeline Insight, 2024

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

Choroideremia Understanding

Choroideremia (CHM) is an uncommon heredodystrophy with an estimated prevalence of 1 in 50,000 patients. This disorder mainly involves males because of its X-linked inheritance pattern1 and is dependent on mutations in the CHM gene. This gene is known to be related to membrane transportation protein in the retina and retinal pigment epithelium (RPE).

Choroideremia is an X-linked recessive inherited disorder due to mutation in the CHM gene (OMIM 303390), which is placed on chromosome X at position q21.2. CHM messenger RNA (m RNA) is responsible for the creation of the Rab escort protein (REP)-1 which is ubiquitously expressed. This protein has 653 amino acids and is involved in intracellular migration of organelles and molecules. Especially, REP-1 manages the process aimed at attaching the unprenylated Rab proteins to the geranyl-geranyl transferase 2enzyme.

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

• In February 2019, Roche entered into a definitive merger agreement to acquire Spark Therapeutics and its clinical assets, including SPK-7001 for choroideremia in Phase I/II.
• 4D therapeutics is developing and investigating its lead product candidate 4D-110 in an ongoing Phase I dose-escalation clinical trial in patients with choroideremia related to mutations in the CHM gene.

Choroideremia Emerging Drugs

AAV2-h CHM: SparkTherapeutics
Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators.

Choroideremia: Therapeutic Assessment

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

Major Players in Choroideremia

There are approx. 5+ key companies which are developing the therapies Choroideremia. The companies which have their Choroideremia drug candidates in the most advanced stage, i.e phase I/II include Spark Therapeutics

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

Choroideremia 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
• Intravenous
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Choroideremia: Pipeline Development Activities

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

Choroideremia Report Insights

• Choroideremia Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Spark Therapeutics
• Molecular Therapeutics
• Curative Biotechnology
• Ray Therapeutics

Key Products

• AAV2-hCHM
• 4D 110
• Metformin
• RAY 001

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