Krabbe Disease - Pipeline Insight, 2021

Attention: There is an updated edition available for this report.

DelveInsight’s, “Krabbe Disease - Pipeline Insight, 2021,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Krabbe 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.

Geography Covered
• Global coverage

Krabbe Disease Understanding
Krabbe Disease: Overview
Krabbe disease is a rare leukodystrophy affecting about 1 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase (GALC) gene, a lysosomal enzyme responsible for the breakdown of certain types of lipids such as psychosine. Without functional GALC, psychosine accumulates to toxic levels in cells. The psychosine toxicity is most severe in the protective cells surrounding the nerves in the brain and throughout the body (peripheral nervous system), eventually leading to the death of these cells. The progressive neuronal cell death manifest over the course of the disease: initially presenting as mental and physical delays in development, muscle weakness and irritability and advancing rapidly to vision and hearing loss and difficulty swallowing and breathing. Early onset, “Early Infantile”, Krabbe cases usually results in death by age 2, while later onset “Late Infantile” cases have a more variable course of progressive decline. There is currently no cure for either form of Krabbe disease.

""Krabbe Disease - Pipeline Insight, 2021"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Krabbe Disease pipeline landscape is provided which includes the disease overview and Krabbe Disease treatment guidelines. The assessment part of the report embraces, in depth Krabbe 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, Krabbe 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 Krabbe Disease R&D. The therapies under development are focused on novel approaches to treat/improve Krabbe Disease.

Krabbe Disease Emerging Drugs Chapters
This segment of the Krabbe 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.

Krabbe Disease Emerging Drugs
• FBX-101: Forge Biologics
FBX-101 utilizes adeno-associated viral (AAV) gene therapy after hematopoietic stem cell transplant (HSCT) to deliver a functioning copy of the GALC gene, which encodes an enzyme needed to prevent the buildup of psychosine in myelinated cells of both the central and peripheral nervous system. FBX-101 has been shown to functionally correct the central and peripheral myelination deficits, significantly improve the behavioral impairments associated with Krabbe disease in animal models, and drastically improve the lifespan of treated animal models of the disease. The use of transplant and intravenous AAV gene therapy infusion has the potential to overcome some of the immunological safety challenges of traditional AAV gene therapies. FBX-101, for the treatment of patients with Krabbe disease, is currently in the early phase of clinical development, and the focus of the initial “RESKUE” Phase 1/2 clinical trial will be an evaluation for safety of FBX-101.

• PBKR03: Passage Bio
The gene therapy product candidate, PBKR03, utilizes an AAVhu68 viral vector to deliver a functional GALC enzyme gene that codes for galactosylceramidase (GAL-C). The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection), with the goal of increasing levels of the GALC enzyme in both the central nervous system (CNS) and the peripheral nervous system (PNS).
Further product details are provided in the report……..

Krabbe Disease: Therapeutic Assessment
This segment of the report provides insights about the different Krabbe Disease drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Krabbe Disease
There are approx. 7+ key companies which are developing the therapies for Krabbe Disease. The companies which have their Krabbe Disease drug candidates in the most advanced stage, i.e. phase I/II includes Forge Biologics.

• Phases
DelveInsight’s report covers around 7+ 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
Krabbe 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
• 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.

Krabbe Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Krabbe 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 Krabbe Disease drugs.

Krabbe Disease Report Insights
• Krabbe Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players
• Forge Biologics
• Magenta Therapeutics
• Passage Bio
• Sanbio
• BioXcel Corporation
• Fondazione Telethon
• Polaryx
• Gain therapeutics
• Zymenex

Key Products
• FBX-101
• MGTA-456
• PBKR 03
• MSC 2
• BXCL 904
• Research programme: rare genetic diseases gene thera
• PLX-200

Executive Summary
Krabbe Disease: Overview
• Causes
• Mechanism of Action
• Signs and Symptoms
• Diagnosis
• Disease Management
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Krabbe Disease – DelveInsight’s Analytical Perspective
Mid Stage Products (Phase II)
• Comparative Analysis
MGTA-456: Magenta Therapeutics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase Iand I/II)
• Comparative Analysis
FBX-101: Forge Biologics
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Krabbe Disease Key Companies
Krabbe Disease Key Products
Krabbe Disease- Unmet Needs
Krabbe Disease- Market Drivers and Barriers
Krabbe Disease- Future Perspectives and Conclusion
Krabbe Disease Analyst Views
Krabbe Disease Key Companies
Table 1 Total Products for Krabbe Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Figure 1 Total Products for Krabbe Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

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