Around the Helix: Cell and Gene Therapy Company Updates – April 15, 2026

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. International Pompe Day 2026: Top News and Expert Insights

Over the past year, our sister site NeurologyLive® has covered news and clinical trial data updates related to the various therapies, including gene therapies, currently in development for Pompe disease and conducted interviews with experts to discern the potential for new research and treatments to affect the care paradigm. For International Pompe Day, observed annually on April 15 by the patient and clinician communities, the NeurologyLive team gathered some key news stories and expert insights on Pompe disease to offer an overview of the progress being made for this rare disease.

2. FDA Draft Guidance Outlines NGS-Based Safety Assessment for Genome Editing Therapies

The FDA has issued draft guidance on the safety assessment of human gene therapy products that use genome editing technologies. The document focuses on next-generation sequencing (NGS)–based methods to evaluate off-target editing and genomic integrity for both ex vivo and in vivo products. Recommendations address sequencing strategies, sample selection, analysis parameters, and reporting for nonclinical studies supporting investigational new drug applications (INDs) and biologics license applications. The guidance aligns with the agency’s broader framework to facilitate development of individualized therapies for ultrarare diseases and is open for public comment for 90 days.

3. DMD Treatment Changes: Combination Therapy, Adherence, and Gene Therapy Access

In a panel discussion on NeurologyLive, John F. Brandsema, MD, and Aravindhan Veerapandiyan, MD, discussed how clinicians approach adding or modifying therapies over time and how families navigate the growing complexity of treatment decisions. They spoke on safety, dosing, and combo therapies in Duchenne, balancing infusion burden, ethics, and gene-transfer risks before decline.

4. Grace Science Snags RMAT Designation for Gene Therapy for NGLY1 Deficiency

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to GS-100, an investigational AAV9 gene replacement therapy for children with NGLY1 deficiency, an ultrarare neurogenetic disorder with no approved treatments. The designation is supported by preliminary data from an open-label, single-arm phase 1/2/3 clinical trial (NCT06199531) in 10 patients, in which treated participants followed for at least 52 weeks have shown gains in motor function and cognitive skills. GS-100 also holds orphan drug, rare pediatric disease, and fast track designations.

5. Andelyn to Manufacture Evolyra’s AAVMYO2 Programs for LGMDR3 and LGMDR5

Andelyn Biosciences will manufacture clinical‑grade adeno-associated virus (AAV) vector-based gene therapies for Evolyra Therapeutics’ programs targeting limb-girdle muscular dystrophies (LGMDs) LGMDR3 and LGMDR5, using its AAV Curator platform. Evolyra’s AAVMYO2 vector reportedly improved skeletal muscle expression and reduced liver exposure in preclinical models; an IND submission is planned in the second half of 2026 to support phase 1/2 trials.