Andelyn Biosciences and Evolyra Therapeutics Join Forces to Advance Gene Therapies for Limb-Girdle Muscular Dystrophy

Andelyn Biosciences, a Columbus, Ohio-based cell and gene therapy contract development and manufacturing organisation (CDMO), has announced a manufacturing partnership with Evolyra Therapeutics to produce clinical-grade adeno-associated virus (AAV) gene therapies for two forms of limb-girdle muscular dystrophy (LGMD): types 2C and 2D, also designated LGMDR5 and LGMDR3. The deal brings together Evolyra's proprietary next-generation viral vector with Andelyn's established manufacturing infrastructure, and represents another step forward in a field that is steadily gaining momentum.

Limb-girdle muscular dystrophies are a group of around 32 distinct genetic disorders characterised by progressive weakness and wasting of the muscles surrounding the hips and shoulders. There are no approved disease-modifying treatments for any LGMD subtype, and the conditions can significantly reduce quality of life, mobility, and life expectancy. With more than 580,000 patients affected worldwide and a global market estimated to exceed $100 billion, the commercial and clinical case for effective therapies is considerable. Evolyra's programmes target the LGMDR3 and LGMDR5 subtypes specifically, a segment representing roughly 20,000 patients and an $11 billion market opportunity.

At the core of Evolyra's approach is AAVMYO2, a proprietary muscle-targeting AAV vector that the company says substantially improves skeletal muscle expression while reducing liver toxicity relative to first-generation vectors. Liver toxicity has been one of the more persistent concerns in systemic AAV gene therapy, and addressing it at the vector level rather than through post-treatment management is a meaningful engineering priority.

Evolyra reports complete protein expression and muscle restoration in preclinical animal models, though as with all such results, the translation to human trials will be the real test.

Evolyra's approach is designed to deliver a complete, functional gene rather than a partial replacement, which the company considers essential for effective treatment Evolyra Therapeutics of conditions like LGMDR3 and LGMDR5. The company is building what it describes as a scalable, modular gene therapy platform, drawing on a consortium of over 500 patients to provide biomarker data, disease progression tools, and trial readiness support Evolyra Therapeutics.

Under the partnership, Andelyn will apply its AAV Curator® Platform to support IND-enabling studies and Good Manufacturing Practice (GMP) production for Evolyra's clinical trials. The platform is designed to support the full arc of rare disease therapy development, from preclinical stages through to clinical manufacturing, without requiring companies to rebuild their supply chain at each phase transition.

"Evolyra exemplifies the kind of transformative science we are eager to support," said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences. "Their innovative approach to treating LGMDs with an AAV gene therapy aligns with our mission to accelerate access to life-changing treatments for patients with rare and ultra-rare diseases. We are very honored to help bring this breakthrough therapy to the patients who need it most."

Nicholas Johnson, MD, CEO of Evolyra Therapeutics, pointed to the manufacturing continuity as a particular advantage: "We appreciate the partnership and commitment from Andelyn Biosciences. Their platform will allow a seamless transition between the different phases of our therapeutic development, ultimately accelerating our ability to bring these therapies to individuals living with LGMD as quickly as possible."

Evolyra is targeting an IND submission in the second half of 2026, with Phase I/II clinical trials to follow. The company recently closed a $5 million seed round and is currently in the process of raising a $20 million Series A to fund programme advancement. That funding trajectory is not unusual for a company at this stage, though completing the Series A in the current biotech financing environment will matter considerably for whether the 2026 timeline holds.

The partnership arrives at a time of genuine, if cautious, progress in LGMD gene therapy more broadly. In March 2026, Atamyo Therapeutics shared the first clinical results for its ATA-200 gene therapy in LGMD-2C/R5, from a Phase Ib/II trial at the University of Florida's Powell Gene Therapy Center. Ddw-online Meanwhile, Sarepta Therapeutics has multiple LGMD programmes in clinical development, with its SRP-9003 therapy meeting the primary endpoint of its Phase 3 EMERGENE trial, demonstrating robust beta-sarcoglycan protein expression in treated patients. CGTLive®

These parallel programmes in overlapping LGMD subtypes underscore both the scale of unmet need and the competitive dynamics emerging in this space. Evolyra will need to differentiate on both the clinical profile of AAVMYO2 and its speed to trials. The Andelyn partnership is designed to help on the latter count, and the combination of an experienced CDMO with a next-generation vector and a patient-connected clinical network gives Evolyra a credible, if still early-stage, development platform.

For patients with LGMDR3 and LGMDR5, who currently have no therapeutic options beyond symptom management, the prospect of multiple well-resourced gene therapy programmes entering clinical testing is the most tangible reason for optimism. Whether any of them ultimately translate into approved treatments remains to be seen.