Gaucher's Disease Type I - Pipeline Insight, 2025

This “Gaucher’s Disease Type I - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Gaucher’s Disease Type I 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.

Gaucher’s Disease Type I: Understanding

Gaucher’s Disease Type I: Overview

Gaucher's Disease Type I, also known as non-neuronopathic Gaucher disease, is the most prevalent form of this genetic disorder, particularly among individuals of Ashkenazi Jewish descent, affecting approximately 1 in 450 in this population. It is caused by a deficiency of the enzyme glucocerebrosidase, leading to the accumulation of glucocerebroside in various organs, particularly the spleen and liver, which can result in organ enlargement (hepatosplenomegaly), anemia, easy bruising due to low platelet counts, and bone pain or fractures. Symptoms can vary widely, from mild to severe, and may present at any age. Effective treatments, including enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), are available to manage symptoms and improve quality of life, allowing individuals to live well into old age with proper care and monitoring. Gaucher's Disease Type I presents with a wide range of signs and symptoms that can vary greatly in severity and onset. The most common manifestations include an enlarged spleen and liver (hepatosplenomegaly), which can lead to abdominal discomfort and early satiety. Anemia and low platelet counts are also common, resulting in easy bruising and bleeding. Bone involvement is another hallmark of the disease, with patients often experiencing bone pain, osteoporosis, and an increased risk of fractures.

Gaucher's Disease Type I is characterized by the accumulation of glucocerebroside in cells of the macrophage-monocyte system, particularly in the liver, spleen, bone marrow, and lymph nodes, due to a deficiency of the enzyme glucocerebrosidase. This leads to enlargement of the liver and spleen (hepatosplenomegaly), anemia, low platelet counts, and bone abnormalities such as pain, fractures, and osteoporosis. Histologically, the disease is marked by the presence of enlarged gaucher cells with a characteristic 'crinkled paper' cytoplasm, which can be seen in liver biopsies. Electron microscopy reveals angulated lysosomes within these cells.

The diagnosis of Gaucher's Disease Type I begins with a clinical evaluation of symptoms like splenomegaly, anemia, and bone pain. Laboratory tests measure glucocerebrosidase enzyme activity in blood, with levels below 15% indicating the disease. Genetic testing identifies mutations in the GBA gene for confirmation and family risk assessment. Imaging studies such as ultrasound or MRI evaluate organ enlargement and bone involvement. The treatment of Gaucher's Disease Type I has significantly improved in recent decades with the development of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). ERT involves intravenous infusions of recombinant glucocerebrosidase enzyme, which helps to reduce the accumulation of glucocerebroside in affected organs and tissues. SRT, on the other hand, aims to reduce the production of glucocerebroside by inhibiting the enzyme glucosylceramide synthase. Both therapies have been shown to effectively reduce spleen and liver size, improve hematological parameters, and alleviate bone pain and complications.

“Gaucher’s Disease Type I - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the mechanism of action. A detailed picture of the Gaucher’s Disease Type I pipeline landscape is provided which includes the disease overview and Gaucher’s Disease Type I treatment guidelines. The assessment part of the report embraces, in depth Gaucher’s Disease Type I 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, Gaucher’s Disease Type I 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 Gaucher’s Disease Type I R&D. The therapies under development are focused on novel approaches to treat/improve Gaucher’s Disease Type I.

Gaucher’s Disease Type I Emerging Drugs Chapters

This segment of the Gaucher’s Disease Type I report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

Gaucher’s Disease Type I Emerging Drugs

FLT 201: Spur Therapeutics

FLT201 is an investigational liver-directed AAV gene therapy in preclinical development for the treatment of gaucher disease Type 1. FLT201 contains a liver-specific promoter and a GBA1 sequence that expresses Freeline’s novel, proprietary GCase variant 85, which has a 20-fold longer half-life at lysosomal pH conditions than wild-type GCase protein. Freeline’s high-transducing AAVS3 capsid advances our goal to address unmet needs for those affected by Gaucher disease by enabling sustained, endogenous production of GCase following one-time intravenous infusion. Currently the drug is in Phase I stage of Clinical trial evaluation for the treatment of Gaucher's Disease Type I.

Gaucher’s Disease Type I: Therapeutic Assessment

This segment of the report provides insights about the different Gaucher’s Disease Type I drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Gaucher’s Disease Type I

There are approx. 3+ key companies which are developing the therapies for Gaucher’s Disease Type I. The companies which have their Gaucher’s Disease Type I drug candidates in the most advanced stage, i.e. Phase I include Spur Therapeutics.

Phases

The report covers around 3+ 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

Gaucher’s Disease Type I 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
• 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.

Gaucher’s Disease Type I: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Gaucher’s Disease Type I 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 Gaucher’s Disease Type I drugs.

Gaucher’s Disease Type I Report Insights

• Gaucher’s Disease Type I Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Eli Lilly and Company
• Spur Therapeutics
• AceLink Therapeutics

Key Products

• LY-3884961
• FLT 201
• AL 1211

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