Dublin, Nov. 21, 2024 (GLOBE NEWSWIRE) -- The "Pompe Disease - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.
The report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Pompe 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.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Pompe Disease R&D. The therapies under development are focused on novel approaches to treat/improve Pompe Disease.
Pompe Disease Emerging Drugs Chapters
This segment of the Pompe Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.
Pompe Disease Emerging Drugs
GC301: GeneCradle Therapeutics
- GC301 injection is an AAV gene therapy drug designed and developed by Genecradle Therapeutics for the treatment of Pompe disease. It adopts a one-time intravenous injection strategy for widespread systemic expression, aiming to directly compensate for the GAA gene deficiency in tissues such as the liver, heart, skeletal muscles, and central nervous system. In previous clinical trials conducted (IIT+IND), IOPD subjects successfully discontinued enzyme replacement therapy after gene therapy, with varying degrees of improvement in motor ability. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Pompe disease.
MZE001: Maze Therapeutics
- MZE001 is an oral glycogen synthase (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. GYS1 is an enzyme responsible for glycogen production. MZE001 is currently being evaluated as a potential oral treatment for patients with Pompe disease, as well as other glycogen storage disorders. Currently, the drug is in Phase I stage of its clinical trial for the treatment of pompe disease.
Pompe Disease: Therapeutic Assessment
This segment of the report provides insights about the different Pompe Disease drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Pompe Disease
- There are approx. 15+ key companies which are developing the therapies for Pompe Disease. The companies which have their Pompe Disease drug candidates in the most advanced stage, i.e. Phase I/II include, GeneCradle Therapeutics.
The 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
The Pompe 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
- 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
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Pompe Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Pompe 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 Pompe Disease drugs.
Pompe Disease Report Insights
- Pompe Disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Pompe 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 Pompe Disease drugs?
- How many Pompe Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pompe Disease?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pompe 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 Pompe Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Asklepios Biopharmaceutical
- GeneCradle Therapeutics
- Astellas Gene Therapies
- Maze Therapeutics
- M6P Therapeutics
- Entrada Therapeutics
Key Products
- AAV2/8LSPhGAA
- GC301
- AT845
- MZE 001
- M 021
- ERT/Oligonucleotide
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/pt0ejo
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