Limb Girdle Muscular Dystrophy Pipeline Insight 2025: Advancing Genetic and Regenerative Therapies for Rare Muscular Disorders | DelveInsight

DelveInsight's "Limb Girdle Muscular Dystrophy (LGMD) - Pipeline Insight, 2025" report provides a deep dive into the evolving therapeutic landscape of LGMD, a group of genetically inherited muscular disorders characterized by progressive weakness in the hip and shoulder muscles. With more than 25 active pipeline candidates targeting various LGMD subtypes, the field is witnessing a shift toward personalized gene therapies and precision treatments.

Recent advances have focused on gene replacement, exon skipping, and RNA-targeted therapies, particularly for subtypes such as LGMD2I/R9 (FKRP-related), LGMD2B/R2 (dysferlinopathy), and LGMD2A/R1 (calpainopathy). Prominent candidates in development include SRP-9004 (Sarepta Therapeutics), BBP-418 (ML Bio Solutions), and LION-101 (Asklepios BioPharmaceutical), each aimed at correcting or compensating for specific genetic mutations associated with LGMD progression.

Leading companies such as Sarepta Therapeutics, AskBio, ML Bio Solutions (a BridgeBio company), Astellas Gene Therapies, and Armatus Bio are advancing gene and cell therapies, supported by orphan drug and rare pediatric disease designations to accelerate development and regulatory review. Many of these investigational assets are in preclinical to early phase I/II stages, with promising results in animal models and first-in-human trials.

The report also highlights ongoing research into biomarkers, natural history studies, and emerging delivery technologies such as AAV vectors and intramuscular injections. These developments are key to optimizing efficacy, safety, and durability in long-term disease management.

With increasing investment, regulatory momentum, and scientific innovation, the LGMD therapeutic pipeline is poised to deliver transformative treatments for a patient population historically underserved, marking a hopeful new era in rare neuromuscular care.

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

Key Takeaways from the Limb Girdle Muscular Dystrophy Pipeline Report
• DelveInsight's limb girdle muscular dystrophy pipeline analysis depicts a strong space with 20+ active players working to develop 25+ pipeline drugs for limb girdle muscular dystrophy treatment.
• The leading limb girdle muscular dystrophy companies include ML Bio Solutions, Asklepios BioPharmaceutical, Sarepta Therapeutics, Inc., Atamyo Therapeutics, Edgewise Therapeutics, Vita Therapeutics, BridgeBio Pharma, Hansa Biopharma, and others are evaluating their lead assets to improve the limb girdle muscular dystrophy treatment landscape.
• Key limb girdle muscular dystrophy pipeline therapies in various stages of development include AB-1003, SRP-9003, BBP-418, LION-101, and others.
• In June 2025, Sarepta Therapeutics (NASDAQ: SRPT) announced that the FDA granted platform technology designation to its rAAVrh74 viral vector, used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for treating limb-girdle muscular dystrophy type 2E/R4.
• In June 2025, Cure Rare Disease (CRD) announced the successful completion of a pre-IND meeting with the FDA for its investigational gene therapy targeting Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9).
• In November 2024, Atamyo Therapeutics announced FDA clearance of its IND application for ATA-200, a one-time gene therapy for γ-sarcoglycan-related LGMD2C/R5, allowing it to proceed to a Phase 1b/2b clinical trial.

Request a sample and discover the recent breakthroughs happening in the limb girdle muscular dystrophy pipeline landscape at https://www.delveinsight.com/report-store/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Limb Girdle Muscular Dystrophy Overview
Limb-girdle muscular dystrophies (LGMD) are a group of rare, progressive genetic disorders characterized by muscle weakness and wasting, primarily affecting the hip and shoulder areas, known as the limb-girdle region. Over time, this weakness can spread to other muscles throughout the body. LGMD includes a range of subtypes, each caused by specific genetic mutations. The age of onset, disease severity, and rate of progression can vary widely, even among members of the same family. Some individuals experience a mild, slow-progressing form of LGMD, while others may develop a rapidly progressing version that leads to significant disability.

LGMD is classified into two main types based on inheritance patterns: autosomal dominant (LGMD1) and autosomal recessive (LGMD2). LGMD1 includes eight subtypes (LGMD1A-1H), while LGMD2, now often referred to using a letter-based system (LGMDA-Q), includes 17 subtypes. Advances in genetic and protein analysis have made it possible to more accurately diagnose and distinguish between these various forms.

Find out more about limb girdle muscular dystrophy medication at https://www.delveinsight.com/report-store/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Limb Girdle Muscular Dystrophy Treatment Analysis: Drug Profile
BBP-418: ML Bio Solutions
BBP-418 is a naturally occurring sugar in the body that serves as a substrate essential for the proper functioning of alpha-dystroglycan, a key protein involved in muscle integrity. Research has shown that BBP-418 is a critical component in enabling alpha-dystroglycan to perform its "shock absorber" role effectively. ML Bio Solutions is currently developing BBP-418 as a potential treatment for patients with FKRP gene mutations, a known cause of certain forms of limb-girdle muscular dystrophy (LGMD). The therapy is presently in Phase II clinical trials for LGMD.

LION-101: Asklepios BioPharmaceutical
LION-101 is an investigational gene therapy designed as a one-time intravenous infusion, using a recombinant adeno-associated virus (rAAV) vector. It is being developed to treat patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Currently, LION-101 is being evaluated in a Phase I/II clinical trial to assess its safety and efficacy in individuals with LGMD.

Learn more about the novel and emerging limb girdle muscular dystrophy pipeline therapies at https://www.delveinsight.com/report-store/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Limb Girdle Muscular Dystrophy 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
• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

By Molecule Type
• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Scope of the Limb Girdle Muscular Dystrophy Pipeline Report
• Coverage: Global
• Key Limb Girdle Muscular Dystrophy Companies: ML Bio Solutions, Asklepios BioPharmaceutical, Sarepta Therapeutics, Inc., Atamyo Therapeutics, Edgewise Therapeutics, Vita Therapeutics, BridgeBio Pharma, Hansa Biopharma, and others.
• Key Limb Girdle Muscular Dystrophy Pipeline Therapies: AB-1003, SRP-9003, BBP-418, LION-101, and others.

To dive deep into rich insights for drugs used for limb girdle muscular dystrophy treatment, visit: https://www.delveinsight.com/report-store/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Limb Girdle Muscular Dystrophy Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Limb Girdle Muscular Dystrophy Pipeline Therapeutics
6. Limb Girdle Muscular Dystrophy Pipeline: Late-Stage Products (Phase III)
7. Limb Girdle Muscular Dystrophy Pipeline: Mid-Stage Products (Phase II)
8. Limb Girdle Muscular Dystrophy 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

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Jatin Vimal
jvimal@delveinsight.com
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Healthcare Consulting
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This release was published on openPR.