雅虎香港財經 Stargardt Disease Drug Pipeline Research 2024: Therapeutic Assessment by Product Type, Stage, Route of Administration, and Molecule Type
Dublin, June 26, 2024 (GLOBE NEWSWIRE) -- The "Stargardt Disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.
This report provides comprehensive insights about 20+ 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.
The 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, Stargardt Disease 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 Stargardt Disease R&D. The therapies under development are focused on novel approaches to treat/improve Stargardt Disease.
Stargardt Disease Emerging Drugs Chapters
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 Bio
Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye.
By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the US, and Orphan Drug Designation in the U.S., Europe, and Japan for the treatment of STGD1. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Stargardt disease.
KIO-301: Kiora Pharmaceuticals
KIO-301 is a molecular photoswitch designed to confer light-sensing capabilities to Retinal Ganglion Cells (RGCs). It targets patients with inherited retinal diseases where mutations lead to photoreceptor death, causing vision loss. Unlike healthy eyes where photoreceptors perform light detection, KIO-301 selectively enters RGCs downstream of degenerated photoreceptors. Inside RGCs, it interacts with voltage-gated ion channels.
When exposed to light, KIO-301 changes shape, altering current flow and activating neurons to signal the brain. Removal of light causes KIO-301 to revert to its original shape, halting signaling. Essentially, KIO-301 acts as a light switch within the eye, restoring light sensitivity in patients with retinal degeneration. Currently, the drug is in Phase II stage of its clinical trial for the treatment of stargardt disease.
OCU410: Ocugen
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies.
These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the drug is in Phase I/II stage of its clinical trial 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. 20+ 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.
Key Questions
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 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 Stargardt Disease and their status?
What are the key designations that have been granted to the emerging drugs?
Key Players
Belite Bio
Ocugen
Alkeus Pharmaceuticals
Astellas Pharma
SalioGen Therapeutics
Ascidian
Biogen
Splice Bio
Key Products
Tinlarebant
OCU410ST
ALK-001
ASP-7317
SGT-1001
ABCA4
NSR-ABCA4
SB-007
Phases
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
Oral
Intravenous
Subcutaneous
Parenteral
Topical
Molecule Type
Products have been categorized under various Molecule types such as
Recombinant fusion proteins
Small molecule
Monoclonal antibody
Peptide
Polymer
Gene therapy
Product Type
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
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/qbhkng
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