Galactosemia Drug Pipeline Market Report 2022 Featuring Applied Therapeutics, Jaguar Gene Therapy, & BridgeBio Pharma - ResearchAndMarkets.com

The "Galactosemia- Pipeline Insight, 2022" clinical trials has been added to ResearchAndMarkets.com's offering.

This Galactosemia- Pipeline Insight, 2022 report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Galactosemia 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.

"Galactosemia- Pipeline Insight, 2022" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Galactosemia pipeline landscape is provided which includes the disease overview and Galactosemia treatment guidelines.

The assessment part of the report embraces, in depth Galactosemia 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, Galactosemia 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 Galactosemia R&D. The therapies under development are focused on novel approaches to treat/improve Galactosemia.

Galactosemia Emerging Drugs Chapters

This segment of the Galactosemia 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.

Galactosemia Emerging Drugs

AT-007: Applied therapeutics

AT-007 is an investigational, novel Aldose Reductase Inhibitor (ARI) being developed for the treatment of several rare diseases. AT-007 is a potent and selective compound, which crosses the blood brain barrier into the Central Nervous System (CNS penetrant). AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the FDA for the treatment of Galactosemia, a rare metabolic disease, and Phosphomannomutase 2 Deficiency - a Congenital Disorder of Glycosylation (PMM2-CDG). AT-007 is a once-daily oral compound that inhibits the enzyme Aldose Reductase. Aldose Reductase plays a crucial role in the pathogenesis of Galactosemia. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Galactosemia.

JAG 101: Jaguar Gene Therapy

Jaguar is advancing JAG101, an investigational gene therapy currently in preclinical development that aims to deliver a gene replacement solution to address the root cause of Type 1 galactosemia by delivering the functional GALT gene via the AAV9 vector.

Galactosemia: Therapeutic Assessment

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

Major Players in Galactosemia

There are approx. 5+ key companies which are developing the therapies for Galactosemia. The companies which have their Galactosemia drug candidates in the most advanced stage, i.e. phase III include, Applied therapeutics.

Phases

The 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

Galactosemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal
• Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule
• Product Type

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

Galactosemia: Pipeline Development Activities

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

Galactosemia Report Insights

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

Galactosemia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Players

• Applied therapeutics
• Jaguar Gene Therapy
• BridgeBio Pharma

Key Products

• AT-007
• JAG 101
• BBP-818

For more information about this clinical trials report visit https://www.researchandmarkets.com/r/kwgwz9

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