Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024

Powered by

Unlock hidden opportunities in the Pharmaceuticals industry

$2000
Buy Report

Published: June 05, 2024 Report Code: GDGMDHC23088IDB-L5

Select Item

Empower your strategies with our Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024 report and make more profitable business decisions.

Lipid storage disorders, or lipidoses, encompass inherited metabolic conditions where fats accumulate due to enzyme deficiencies, impacting various organs. Gaucher, Niemann-Pick, Tay-Sachs, Fabry, and Krabbe diseases are examples. Symptoms, varying by disorder, may include neurological problems, organ enlargement, and skeletal abnormalities. Treatments focus on symptom management, employing enzyme replacement, substrate reduction therapies, and supportive care. Managing these complex conditions requires tailored approaches; however, some lack specific treatments. Genetic counseling aids in understanding inheritance patterns and risks for affected families, offering guidance amidst these rare and diverse disorders.

The Lipid Storage Disorders (Lipidoses) drugs in development market research report provide comprehensive information on the therapeutics under development for Lipid Storage Disorders (Lipidoses), 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 and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Lipid Storage Disorders (Lipidoses) and features dormant and discontinued products.

Note:

* This is an “on-demand” report and will be delivered within 2 business days (excluding weekends and holidays) of the purchase.

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

Quick View – Lipid Storage Disorders (Lipidoses)
Key Targets
· Alpha Galactosidase A; Lysosomal Acid Glucosylceramidase; Ceramide Glucosyltransferase; Beta Galactosidase; NPC Intracellular Cholesterol Transporter 1; Peroxisome Proliferator Activated Receptor Alpha; Sphingomyelin Phosphodiesterase; Acid Ceramidase; Coagulation Factor IX; and B Cell Receptor CD22
Key Mechanisms of Action
· Alpha Galactosidase A Replacement; Lysosomal Acid Glucosylceramidase Activator; Ceramide Glucosyltransferase Inhibitor; Alpha Galactosidase A Activator; Lysosomal Acid Glucosylceramidase Replacement; Beta Galactosidase Activator; NPC Intracellular Cholesterol Transporter 1 Activator; Peroxisome Proliferator Activated Receptor Alpha Agonist; Sphingomyelin Phosphodiesterase Activator; and Beta Galactosidase Replacement
Key Routes of Administration
· Intravenous; Oral; Subcutaneous; Parenteral; Nasal; Intrathecal; Intracisternal; Inhalational; and Intracerebral
Key Molecule Types
· Small Molecule; Gene Therapy; Recombinant Enzyme; Gene-Modified Cell Therapy; Enzyme; Protein; Fusion Protein; Monoclonal Antibody; Biologic; and Recombinant Protein
Major Companies
· Gain Therapeutics Inc; M6P Therapeutics; Takeda Pharmaceutical Co Ltd; Polaryx Therapeutics Inc; Sanofi; Yuhan Corp; IntraBio Ltd; AceLink Therapeutics Inc; CANbridge Life Sciences Ltd; and Erad Therapeutics Inc

Scope

  • Therapeutics in Development: Covering 138 molecules, with 123 developed by companies and the rest by universities/institutes.

  • Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.

  • Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.

  • Development History: Access the complete research and development history.

  • Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.

Reasons to Buy

  • Holistic insights: Understand the broad spectrum of Lipid Storage Disorders (Lipidoses) therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.

  • Pipeline Exploration: Explore the detailed Lipid Storage Disorders (Lipidoses) pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.

  • Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Lipid Storage Disorders (Lipidoses) treatments for adaptable decision-making.

  • Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.

Key Players

4D Molecular Therapeutics Inc
AceLink Therapeutics Inc
Aceragen Inc
Adienne Pharma & Biotech SA
Allievex Corp
Allife Medical Science and Technology Co Ltd
Amicus Therapeutics Inc
Apteeus SAS
Azafaros BV
Be Biopharma Inc
Belrose Pharma Inc
Bial - Portela & Ca SA
BioArctic AB
Bioasis Technologies Inc
Biosidus SA
BioStrategies LC
Bloomsbury Genetic Therapies Ltd
Blue Turtle Bio Technologies Inc
CANbridge Life Sciences Ltd
CellGenTech Inc
Centogene NV
Chameleon Biosciences Inc
Chiesi Farmaceutici SpA
Coave Therapeutics
Cyclo Therapeutics Inc
CycloLab Cyclodextrin Research and Development Laboratory Ltd
Deep Genomics Inc
Denali Therapeutics Inc
Dorphan SA
Duke University
Edenbridge Pharmaceuticals LLC
Eleva GmbH
Eli Lilly and Co
Endece LLC
Equaly SA
Erad Therapeutics Inc
Evox Therapeutics Ltd
Freeline Therapeutics Holdings Plc
Gain Therapeutics Inc
GC Biopharma Corp
Gene Therapy Research Institution Co Ltd
Generation Bio Co
Generium
Hanmi Pharmaceuticals Co Ltd
Idorsia Pharmaceutical Ltd
ILIAS Biologics Inc
Immorna Hangzhou Biotechnology Co Ltd
IntraBio Ltd
ISU ABXIS Co Ltd
Jasper Therapeutics Inc
Johnson & Johnson
Kashiv BioSciences LLC
Kumamoto University
KYORIN Pharmaceutical Co Ltd
Kyoto Prefectural University of Medicine
Liberyx Therapeutics Ltd
Lysogene SAS
M6P Therapeutics
Mandos LLC
Merck & Co Inc
MOGAM Biotechnology Research Institute
NeuImmune Inc
Northwestern University
Okklo Life Sciences BV
Oragenics Inc
Oraxion Therapeutics Inc
Oregon State University
Orphazyme A/S
Passage Bio Inc
Perlara PBC
Pfizer Inc
Pharming Group NV
Polaryx Therapeutics Inc
Prevacus Inc
Prevail Therapeutics Inc
ProfoundBio Suzhou Co Ltd
Pronacera Therapeutics SL
Recursion Pharmaceuticals Inc
Sangamo Therapeutics Inc
Sanofi
Scenic Biotech BV
Shanghai Lingyi Biotechnology Co Ltd
Shanghai Pharmaceuticals Holding Co Ltd
Sichuan Zhishan Weixin Biotechnology Co Ltd
Sigilon Therapeutics Inc
SmartPharm Therapeutics Inc
SOM Biotech SL
Spanish National Research Council
StrideBio Inc
Synaptogenix Inc
Takeda Pharmaceutical Co Ltd
Taysha Gene Therapies Inc
The Hospital for Sick Children
Tokyo Medical and Dental University
U.S. National Institutes of Health
UniQure NV
Universiteit Utrecht
University Hospitals Leuven
University of California San Francisco
Vanqua Bio Inc
Walking Fish Therapeutics Inc
Yuhan Corp

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Overview

Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Drugs Under Development by Companies

Drugs under Development by Universities/Institutes

Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Companies Involved in Therapeutic Development

Drug Profiles

Dormant Projects

Discontinued Drugs

Product Development Milestones

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Drugs under Development by Companies, 2024

Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Stage and Target, 2024

Number of Drugs by Stage and Mechanism of Action, 2024

Number of Drugs by Stage and Route of Administration, 2024

Number of Drugs by Stage and Molecule Type, 2024

Dormant Projects, 2024

Discontinued Drugs, 2024

Figures

List of Figures

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Top 10 Targets, 2024

Number of Drugs by Stage and Top 10 Targets, 2024

Number of Drugs by Top 10 Mechanism of Actions, 2024

Number of Drugs by Stage and Top 10 Mechanism of Actions, 2024

Number of Drugs by Top 10 Routes of Administration, 2024

Number of Drugs by Stage and Top 10 Routes of Administration, 2024

Number of Drugs by Top 10 Molecule Types, 2024

Number of Drugs by Stage and Top 10 Molecule Types, 2024

Frequently asked questions

Currency USD
$2,000

Can be used by individual purchaser only

Most Popular
$4,000

Can be shared by unlimited users within one corporate location e.g. a regional office

$6,000

Can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

Add to Basket

Undecided about purchasing this report?

Enquire Before Buying

Get in touch to find out about multi-purchase discounts

reportstore@globaldata.com
Tel +44 20 7947 2745

See what our customers are saying
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image
testimonial-image

“The GlobalData platform is our go-to tool for intelligence services. GlobalData provides an easy way to access comprehensive intelligence data around multiple sectors, which essentially makes it a one-for-all intelligence platform, for tendering and approaching customers.

GlobalData is very customer orientated, with a high degree of personalised services, which benefits everyday use. The highly detailed project intelligence and forecast reports can be utilised across multiple departments and workflow scopes, from operational to strategic level, and often support strategic decisions. GlobalData Analytics and visualisation solutions has contributed positively when preparing management presentations and strategic papers.”

 Business Intelligence & Marketing Manager, SAL Heavy Lift

“COVID-19 has caused significant interference to our business and the COVID-19 intelligence from GlobalData has helped us reach better decisions around strategy. These two highlights have helped enormously to understand the projections into the future concerning our business units, we also utilise the project database to source new projects for Liebherr-Werk to use as an additional source to pitch for new business.”

 Market Analyst & Management, Liebherr-Werk

Your daily news has saved me a lot of time and keeps me up-to-date with what is happening in the market, I like that you almost always have a link to the source origin. We also use your market data in our Strategic Business Process to support our business decisions. By having everything in one place on the Intelligence Center it has saved me a lot of time versus looking on different sources, the alert function also helps with this.

 Head of Key Accounts, Saab AB

Having used several other market research companies, I find that GlobalData manages to provide that ‘difficult-to-get’ market data that others can’t, as well as very diverse and complete consumer surveys.

 Marketing Intelligence Manager, Portugal Foods

Our experience with GlobalData has been very good, from the platform itself to the people. I find that the analysts and the account team have a high level of customer focus and responsiveness and therefore I can always rely on. The platform is more holistic than other providers. It is convenient and almost like a one stop shop. The pricing suite is highly competitive and value for our organisation.

I like reports that inform new segments such as the analysis on generation Z, millennials, the impact of COVID 19 to our banking customers and their new channel habits. Secondly the specialist insight on affluent sector significantly increases our understanding about this group of customers. The combination of those give us depth and breadth of the evolving market.

I’m in the business of answering and helping people make decisions so with the intelligence center I can do that, effectively and efficiently. I can share quickly key insights that answer and satisfy our country stakeholders by giving them many quality studies and primary research about competitive landscape beyond the outlook of our bank. It helps me be seen as an advisory partner and that makes a big difference. A big benefit of our subscription is that no one holds the whole data and because it allows so many people, so many different parts of our organisation have access, it enables all teams to have the same level of knowledge and decision support.

 Head of Customer Insight and Research, Standard Chartered

“I know that I can always rely on Globaldata’s work when I’m searching for the right consumer and market insights. I use Globaldata insights to understand the changing market & consumer landscape and help create better taste & wellbeing solutions for our customers in food, beverage and healthcare industries.

Globaldata has the right data and the reports are of very high quality compared to your competitors. Globaldata not only has overall market sizes & consumer insights on food & beverages but also provides insights at the ingredient & flavour level. That is key for B2B companies like Givaudan. This way we understand our customers’ business and also gain insight to our unique industry”

 Head of Consumer Sensory Insights, Givaudan

GlobalData provides a great range of information and reports on various sectors that is highly relevant, timely, easy to access and utilise.  The reports and data dashboards help engagement with clients; they provide valuable industry and market insights that can enrich client conversations and can help in the shaping of value propositions. Moreover, using GlobalData products has helped increase my knowledge of the finance sector, the players within it, and the general threats and opportunities.

I find the consumer surveys that are carried out to be extremely beneficial and not something I have seen anywhere else. They provided an insightful view of why and which consumers take (or don’t) particular financial products. This can help shape conversations with clients to ensure they make the right strategic decisions for their business.

One of the challenges I have found is that data in the payments space is often piecemeal. With GD all of the data I need is in one place, but it also comes with additional market reports that provide useful extra context and information. Having the ability to set-up alerts on relevant movements in the industry, be it competitors or customers, and have them emailed directly to me, ensures I get early sight of industry activity and don’t have to search for news.

 Senior Account Manager, TSYS

Related reports

View more Lipid Storage Disorders (Lipidoses) reports
Pharmaceuticals
New
Global M&A Deals in H1 2026 - Top Themes by…
$495 | August 2026
Pharmaceuticals
New
Pharmaceutical Drug Development Annual Review 2025
$1,200 | August 2026
Pharmaceuticals
New
Q2 2026 CRO Activity and Intel Report
$1,870 | July 2026
Go even deeper with GlobalData Intelligence Center

Every Company Report we produce is powered by the GlobalData Intelligence Center.

Subscribing to our intelligence platform means you can monitor developments at Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024 in real time.

  • Access a live Lipid Storage Disorders (Lipidoses) – Drugs In Development, 2024 dashboard for 12 months, with up-to-the-minute insights.
  • Fuel your decision making with real-time deal coverage and media activity.
  • Turn insights on financials, deals, products and pipelines into powerful agents of commercial advantage.
Tell me more