Lamellar Ichthyosis Pipeline Insight 2025: Topical Gene Therapies, Barrier Repair Innovations, and Novel Retinoids Reshape Management of Rare Congenital Skin Disorders | DelveInsight

DelveInsight's "Lamellar Ichthyosis - Pipeline Insight, 2025" provides an in-depth analysis of the emerging therapeutic landscape for this debilitating autosomal recessive skin disorder. Characterized by generalized, plate-like scaling and erythroderma from birth, Lamellar Ichthyosis (LI) stems primarily from TGM1 gene mutations that impair epidermal barrier formation. With no curative treatment and current care relying heavily on emollients, keratolytics, and systemic retinoids, the need for disease-modifying therapies remains urgent.

The 2025 pipeline reveals momentum toward targeted topical gene therapies, protein replacement approaches, and barrier repair formulations. Notably, Krystal Biotech's KB105, a redosable, HSV-1-based topical gene therapy delivering functional TGM1, has shown encouraging safety and protein expression data in early trials. Meanwhile, Timber Pharmaceuticals' TMB-001, a topical isotretinoin formulated using patented IPEGTM delivery, has advanced into Phase III trials, aiming to reduce systemic retinoid exposure while maintaining efficacy.

Emerging therapies are also exploring ARCI subtype-specific pathways, with candidates targeting lipid biosynthesis, skin microbiome modulation, and Nrf2 pathway activation to enhance skin integrity and reduce inflammation. Additionally, RNA-based therapeutics and non-viral gene editing platforms are being evaluated preclinically to provide long-term correction with fewer safety risks.

From a regulatory standpoint, the FDA and EMA are supporting orphan drug designations, fast-track pathways, and real-world evidence collection to accelerate access. Incorporation of patient-centric endpoints-such as pruritus relief, mobility, and psychosocial impact-into trial designs is also redefining treatment benchmarks.

As rare dermatological diseases gain broader investment and innovation focus, Lamellar Ichthyosis stands at the forefront of first-in-class therapies that could transform both disease course and patient quality of life.

Interested in learning more about the current treatment landscape and the key drivers shaping the Lamellar Icthyosis pipeline? Click here: https://www.delveinsight.com/report-store/lamellar-icthyosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Lamellar Icthyosis Pipeline Report
• DelveInsight's Lamellar Icthyosis pipeline analysis depicts a strong space with 4+ active players working to develop 5+ pipeline drugs for Lamellar Icthyosis treatment.
• The leading Lamellar Icthyosis companies include Timber Pharmaceuticals, Krystal Biotech, Janssen, and others are evaluating their lead assets to improve the Lamellar Icthyosis treatment landscape.
• Key Lamellar Icthyosis pipeline therapies in various stages of development include TMB-001, KB-105, Ustekinumab, and others.
• Ongoing (first submitted July 29, 2019; primary completion October 2022; study completion March 2025): The Phase I/II clinical trial (NCT04047732) is active in the U.S. to evaluate safety and efficacy of KB105 topical gene therapy in TGM1‐deficient ARCI patients. Initial enrollment and dosing have occurred at Northwestern University and Paddington Testing Company, with early safety data showing promise
• In May 2022, the FDA awarded Breakthrough Therapy Designation to TMB‐001 for the same indication, reinforcing the urgent unmet need and potential impact of this topical therapy.
• In April 2022, the FDA granted Fast Track Designation to TMB‐001, Timber Pharmaceuticals' topical isotretinoin therapy delivered via their IPEGTM system, for treatment of moderate-to-severe congenital ichthyosis-including lamellar ichthyosis.

Request a sample and discover the recent breakthroughs happening in the Lamellar Icthyosis pipeline landscape at https://www.delveinsight.com/report-store/lamellar-icthyosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Lamellar Icthyosis Overview
Lamellar Ichthyosis is a rare inherited skin disorder characterized by widespread scaling, thickened skin, and dryness due to a genetic mutation affecting skin barrier function. It typically presents at birth with a tight, shiny, and thickened outer layer of skin, often described as a "collodion membrane." This condition leads to impaired shedding of skin cells, resulting in large, plate-like scales over the body. Lamellar Ichthyosis is chronic and lifelong, with symptoms varying in severity. Treatment focuses on managing symptoms through regular moisturizing, keratolytic agents to reduce scaling, and supportive skin care to prevent infections and complications.

Find out more about Lamellar Icthyosis medication at https://www.delveinsight.com/report-store/lamellar-icthyosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Lamellar Icthyosis Treatment Analysis: Drug Profile
TMB-001: Timber Pharmaceuticals
TMB-001, available in 0.05% and 0.1% isotretinoin formulations, is being developed as a topical treatment for congenital ichthyosis, including recessive X-linked ichthyosis and autosomal recessive congenital ichthyosis (lamellar ichthyosis subtype). Both the US FDA and EMA have granted orphan drug designation to TMB-001 for this indication. In 2018, the FDA awarded $1.5 million through its Orphan Products Grant program to support Phase IIa and IIb clinical trials. In late 2021, Timber reported positive top-line results from its Phase IIb CONTROL Study, showing clinically meaningful efficacy and a favorable safety profile. TMB-001 is currently being evaluated in Phase III clinical trials.

KB-105: Krystal Biotech
KB-105 is a gene therapy in Phase II clinical trials for treating TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI). It uses a replication-defective, non-integrating HSV-1 viral vector engineered via Krystal's technology platform to deliver a functional human TGM1 gene directly to dividing and non-dividing skin cells. This vector penetrates skin cells more efficiently than other viral vectors, offering a promising therapeutic approach.

Learn more about the novel and emerging Lamellar Icthyosis pipeline therapies at https://www.delveinsight.com/report-store/lamellar-icthyosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Lamellar Icthyosis Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• 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

By Route of Administration
• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Lamellar Icthyosis Pipeline Report
• Coverage: Global
• Key Lamellar Icthyosis Companies: Timber Pharmaceuticals, Krystal Biotech, Janssen, and others.
• Key Lamellar Icthyosis Pipeline Therapies: TMB-001, KB-105, Ustekinumab, and others.

To dive deep into rich insights for drugs used for Lamellar Icthyosis treatment, visit: https://www.delveinsight.com/report-store/lamellar-icthyosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Lamellar Icthyosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Lamellar Icthyosis Pipeline Therapeutics
6. Lamellar Icthyosis Pipeline: Late-Stage Products (Phase III)
7. Lamellar Icthyosis Pipeline: Mid-Stage Products (Phase II)
8. Lamellar Icthyosis Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

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This release was published on openPR.