Around the Helix: Cell and Gene Therapy Company Updates – April 22, 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. Reimagining SMA Treatment: Salanersen and the Push for Durable, Low-Frequency Therapies

In a conversation with NeurologyLive, a sister brand of CGTLive®, Diana Castro, MD, founder and director of the Neurology and Neuromuscular Care Center, discussed the scientific rationale behind salanersen, the implications of emerging biomarker data, and how this therapy may fit into the broader evolution of SMA care.

2. Why One Target Isn't Enough: The Future of Neuroblastoma Therapy

Kristopher R. Bosse, MD, and the ImmunoLogic cohosts discussed the importance of targeting multiple antigens with CAR-T approaches in high-risk neuroblastoma.

3. Early Biomarker Signals From SGT-003 Highlight Ongoing Progress in Duchenne Gene Therapy

In this Q&A with NeurologyLive, Aravindhan Veerapandiyan, MD, a pediatric neuromuscular neurologist at Arkansas Children’s Hospital, and Gabriel Brooks, MD, chief medical officer at Solid Biosciences, discuss the evolving biomarker profile of SGT-003, early safety observations, and how these findings may inform the broader trajectory of gene therapy development in Duchenne muscular dystrophy.

4. Evaluating Gene Therapy BB-301 for OPMD-Related Dysphagia

CGTLive interviewed Jerel A. Banks, MD, PhD, the chief executive officer and chairman of the board of Benitec Biopharma, about data from a phase 1b/2a clinical trial (NCT06185673) evaluating BB-301, the company’s investigational adeno-associated virus vector-based silence and replace gene therapy for oculopharyngeal muscular dystrophy (OPMD)-related dysphagia.

5. Andelyn Biosciences and ENCell Enter Strategic CDMO Partnership to Link US and Asia-Pacific Gene Therapy Manufacturing

Andelyn Biosciences, a Columbus, Ohio–based cell and gene therapy (CGT) contract development and manufacturing organization (CDMO), and ENCell Co., Ltd., a Seoul-based CGT CDMO, have signed a collaboration agreement to establish a cross-regional manufacturing framework spanning the United States and Asia-Pacific. The partnership is intended to support gene therapy developers seeking to conduct clinical trials across both regions by enabling local in-country manufacturing, with the goal of reducing regulatory complexity and supply chain lead times for global programs.