US Leukocyte Adhesion Deficiency (LAD-I) Market Report 2023 to 2030

US Leukocyte Adhesion Deficiency (LAD-I) Market Analysis

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US Leukocyte Adhesion Deficiency (LAD-I) therapeutics Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 - 2030. An uncommon genetic condition called leukocyte adhesion deficiency (LAD-I) impairs the immune system's capacity to combat infections. There is currently no cure for LAD-I, and there are few effective treatments available. To fill this unmet medical need, new medicines are becoming more and more popular. Vertex Pharmaceuticals, Orpha Labs, Avalo Therapeutics, Inc., Rocket Pharmaceuticals, Inc., Aspen Neuroscience, Magenta Therapeutics, Rubius Therapeutics, Enochian Biosciences, Sana Biotechnology, and Sigma-Aldrich are major global players.

ID: IN10USRD096 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Sakshi Mantri

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US Leukocyte Adhesion Deficiency (LAD-I) Market Analysis Summary

US Leukocyte Adhesion Deficiency (LAD-I) Market is valued at around $3.15 Bn in 2022 and is projected to reach $4.38 Bn by 2030, exhibiting a CAGR of 4.2% during the forecast period 2023-2030.

An uncommon autosomal recessive paediatric condition called Leukocyte Adhesion Deficiency-I (LAD-I) is brought on by mutations in the ITGB2 gene, which codes for the protein CD18, a component of beta-2 integrins. Leukocyte adhesion and extravasation from blood arteries to fight infections are made possible by the crucial protein CD18. Children with severe LAD-I therefore frequently have symptoms soon after birth. They experience repeated, sometimes fatal bacterial and fungal infections during infancy, which necessitate many hospitalizations and poor treatment response. Recurrent severe infections in infants who survive infancy include pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Patients with severe LAD-I have a death rate of 60–75 per cent before the age of 2, and survival past the age of five is unusual without a successful bone marrow transplant. Patients with severe LAD-I have a large unmet medical need.

There is currently no cure for LAD-I, and there are few effective treatments available. To fill this unmet medical need, new medicines are becoming more and more popular. There is optimism that novel medicines for LAD-I will be licensed soon given the rising interest in rare diseases and the expanding number of incentives provided by regulatory agencies.

Vertex Pharmaceuticals, Orpha Labs, Avalo Therapeutics, Inc., Rocket Pharmaceuticals, Inc., Aspen Neuroscience, Magenta Therapeutics, Rubius Therapeutics, Enochian Biosciences, Sana Biotechnology, and Sigma-Aldrich are major global players.

Market Dynamics

Market Drivers

There is currently no cure for LAD-I, and there are few effective treatments available. To fill this unmet medical need, new medicines are becoming more and more popular. Currently, a number of businesses are working to develop gene therapies and biologics as LAD-I treatments. However, there is hope that new treatments for LAD-I will be approved soon due to the rising interest in rare diseases and the growing number of incentives provided by regulatory agencies. Therefore, the LAD-I market offers a compelling prospect for businesses willing to make an investment in this field.

Market Development

For severe Leukocyte Adhesion Deficiency-I, Rocket's ex vivo lentiviral gene therapy candidate is called RP-L201 (LAD-I).
All nine LAD-I patients with 12 to 24 months of follow-up available had observed 100% overall survival through Kaplan Meier estimate at 12 months post-infusion. The data also showed improvement in wound healing skills and remission of a skin rash associated with LAD-I.
There have been no significant adverse events associated with RP-L201 to date, but all patients had a very positive safety profile for the drug. It has been previously reported that adverse effects connected to other study techniques, such as busulfan conditioning, are consistent with the safety profiles of those agents and techniques.
Rocket expects to submit the Biologics License Application (BLA) with the FDA in the second quarter of 2023 in light of the promising efficacy and safety data from the Phase 2 pivotal LAD-I study.

Restraints of US Leukocyte Adhesion Deficiency (LAD-I) Therapeutics Market

Because of the high cost of development and the small patient population, companies are likely to encounter difficulties while bringing their medications to market.

Key players

Rocket Pharmaceuticals, Inc. Grifols International S.A. Freeline Therapeutics Ltd. Orchard Therapeutics plc uniQure N.V. Takeda Pharmaceutical Company Limited Pfizer Inc. CSL Behring Roche Holding AG BioMarin Pharmaceutical Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For US Leukocyte Adhesion Deficiency (LAD-I) Therapeutics Market

By Treatment

Hematopoietic Stem Cell Transplantation
Recombinant Human Interferon-gamma Treatment
Prophylactic Immunoglobulin Therapy
Antimicrobial Therapy
Prophylactic Therapy
Fucose Supplementation
Monoclonal Antibodies
Coagulation Factors

By End Users

Hospitals
Specialty Clinics
Others

Methodology for Database Creation

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Combining Secondary and Primary Research

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