Krabbe Disease - Pipeline Insight, 2024

This Krabbe Disease - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ 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 comprises a spectrum ranging from infantile-onset disease (i.e., onset of extreme irritability, spasticity, and developmental delay before age 12 months) to later-onset disease (i.e., onset of manifestations after age 12 months and as late as the seventh decade). Although historically 85%-90% of symptomatic individuals with Krabbe disease diagnosed by enzyme activity alone have infantile-onset Krabbe disease and 10%-15% have later-onset Krabbe disease, the experience with newborn screening (NBS) suggests that the proportion of individuals with possible later-onset Krabbe disease is higher than previously thought. Infantile-onset Krabbe disease is characterized by normal development in the first few months followed by rapid severe neurologic deterioration; the average age of death is 24 months (range 8 months to 9 years). Later-onset Krabbe disease is much more variable in its presentation and disease course.

Krabbe Disease - Pipeline Insight, 2024 report 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 R&D Krabbe Disease. The therapies under development are focused on novel approaches to treat/improve Krabbe Disease.

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 I/II 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).

Krabbe Disease: Therapeutic Assessment

This segment of the report provides insights about the 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. 8+ key companies which are developing the therapies Krabbe Disease. The companies which have their Krabbe Disease drug candidates in the most advanced stage, i.e Phase I/II include Passage Bio

Phases

This report covers around 8+ 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
• 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.

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 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
• Passage Bio
• Sanbio
• Polaryx
• Gain Therapeutics

Key Products

• FBX 101
• PBKR 03
• MSC 2
• PLX 300
• PLX-200
• Liposomal Storage Disorder research project