Globoid Cell Leukodystrophy (Krabbe Disease) – Drugs in Development, 2021

Global Markets Direct’s, ‘Globoid Cell Leukodystrophy (Krabbe Disease) – Drugs in Development, 2021’, provides an overview of the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Globoid Cell Leukodystrophy (Krabbe Disease), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Globoid Cell Leukodystrophy (Krabbe Disease) and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Globoid Cell Leukodystrophy (Krabbe Disease)

– The report reviews pipeline therapeutics for Globoid Cell Leukodystrophy (Krabbe Disease) by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Globoid Cell Leukodystrophy (Krabbe Disease) therapeutics and enlists all their major and minor projects

– The report assesses Globoid Cell Leukodystrophy (Krabbe Disease) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Globoid Cell Leukodystrophy (Krabbe Disease)

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Globoid Cell Leukodystrophy (Krabbe Disease)

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Globoid Cell Leukodystrophy (Krabbe Disease) pipeline depth and focus of Indication therapeutics

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Bial – Portela & Ca SA

BioXcel Corp

Forge Biologics Inc

Gain Therapeutics Inc

Kyorin Pharmaceutical Co Ltd

M6P Therapeutics

Neurogene Inc

Passage Bio Inc

Polaryx Therapeutics Inc

Vascumab LLC

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Globoid Cell Leukodystrophy (Krabbe Disease) – Overview

Globoid Cell Leukodystrophy (Krabbe Disease) – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Globoid Cell Leukodystrophy (Krabbe Disease) – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Globoid Cell Leukodystrophy (Krabbe Disease) – Companies Involved in Therapeutics Development

Bial - Portela & Ca SA

BioXcel Corp

Forge Biologics Inc

Gain Therapeutics Inc

Kyorin Pharmaceutical Co Ltd

M6P Therapeutics

Neurogene Inc

Passage Bio Inc

Polaryx Therapeutics Inc

Vascumab LLC

Globoid Cell Leukodystrophy (Krabbe Disease) – Drug Profiles

Drug for Krabbe Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DUOC-01 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

FBX-101 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Galactosylceramidase Replacement for Krabbe Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

gemfibrozil – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Globoid Cell Leukodystrophy – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate GALC for Globoid Cell Leukodystrophy (Krabbe Disease) – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ibudilast – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

M-031 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

M-033 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PBKR-03 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PLX-300 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecule to Inhibit Acid Ceramidase for Gaucher's and Krabbe's Diseases – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Activate GALC for Krabbe Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Globoid Cell Leukodystrophy (Krabbe Disease) – Dormant Projects

Globoid Cell Leukodystrophy (Krabbe Disease) – Discontinued Products

Globoid Cell Leukodystrophy (Krabbe Disease) – Product Development Milestones

Featured News & Press Releases

Sep 20, 2021: Forge Biologics announces regulatory updates from FDA and EMA, accelerating manufacturing and clinical trial momentum

Apr 05, 2021: Passage Bio receives European Commission Orphan Designation for PBKR03 for treatment of Krabbe Disease

Mar 08, 2021: Passage Bio annouced that U.S. Food and Drug Administration grants Fast Track Designation to its gene therapy candidate PBKR03

Feb 16, 2021: Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease

Feb 10, 2021: Polaryx Therapeutics receives both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Krabbe Disease with PLX-300

Feb 08, 2021: FDA clears IND application for Passage Bio’s gene therapy candidate PBKR03 for treatment of patients with early infantile Krabbe disease, a rare pediatric disorder with no approved disease-modifying treatment options

Jan 04, 2021: Forge Biologics announces FDA clearance of Investigational New Drug application for phase 1/2 clinical trial (RESKUE) of FBX-101 gene therapy for patients with Krabbe disease

Oct 28, 2020: Passage Bio’s PBKR03 receives Orphan Drug and Rare Pediatric Disease Designations from FDA for treatment of Krabbe Disease

May 12, 2020: Passage Bio announces presentation of data from animal models of Krabbe disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting

May 30, 2019: Passage Bio announces third gene therapy development program in Krabbe Disease and supports million dreams fundraising gala

Jan 18, 2016: FDA Grants Rare Pediatric Disease Designation to MediciNova’s MN-166 (ibudilast) for the Treatment of Krabbe Disease

Jun 03, 2015: MediciNova Announces FDA Granted Orphan Drug Designation to MN-166 (ibudilast) for Krabbe Disease

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Products under Development by Companies, 2021

Products under Development by Universities/Institutes, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Bial – Portela & Ca SA, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by BioXcel Corp, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Forge Biologics Inc, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Gain Therapeutics Inc, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Kyorin Pharmaceutical Co Ltd, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by M6P Therapeutics, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Neurogene Inc, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Passage Bio Inc, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Polaryx Therapeutics Inc, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline by Vascumab LLC, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Dormant Projects, 2021

Globoid Cell Leukodystrophy (Krabbe Disease) – Discontinued Products, 2021

List of Figures

List of Figures

Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), 2021

Number of Products under Development by Companies, 2021

Number of Products under Development by Universities/Institutes, 2021

Number of Products by Targets, 2021

Number of Products by Stage and Targets, 2021

Number of Products by Mechanism of Actions, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

    Pricing

Discounts available for multiple purchases.

reportstore@globalmarketsdirect.com
+44 20 7947 2960

Saved reports