Around the Helix: Cell and Gene Therapy Company Updates – May 13, 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. ETX101 Gene Therapy Shows Early Developmental, Seizure Benefits in Dravet Syndrome

Interim findings from the ongoing POLARIS phase 1/2 program suggest that Encoded Therapeutics’ investigational gene therapy ETX101 may produce substantial seizure reductions and early neurodevelopmental improvements in young children with SCN1A-positive Dravet syndrome, supporting continued advancement of the program as it enters pivotal-stage development.

2. Thomas Crawford, MD, on High-Dose Nusinersen and the Future of SMA Treatment

The FDA approved an updated higher-dose formulation of the antisense oligonucleotide therapy nusinersen (Spinraza; Biogen) for spinal muscular atrophy (SMA) on March 30, 2026. In the wake of the decision, CGTLive’s sister site NeurologyLive® spoke with Thomas Crawford, MD, a pediatric neurologist at Johns Hopkins with more than 3 decades of experience in SMA, to gather his perspective on the decision and the evolving landscape of SMA management.

3. Beacon Therapeutics Reports Sustained 12-Month Visual Gains With Laru-Zova in XLRP

Beacon Therapeutics has reported positive 12-month safety and efficacy findings from the phase 2 DAWN trial evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP). According to the company, the latest results demonstrated sustained improvements in visual function through 1 year, building on the 9-month interim findings previously shared at EURETINA 2025 and further supporting the therapy’s advancement ahead of topline results from the pivotal phase 2/3 VISTA trial expected later this year.

4. More on Misconceptions About CAR-T in Neurology

At the 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18 to 22 in Chicago, Illinois, Srikanth Muppidi, MD, discussed how the safety profile of CAR-T therapy in neurological autoimmune conditions may be different from expectations based on the modality’s use in oncology.

5. NINDS Awards $3.2 Million Grant to UMass Chan Investigator to Develop AAV Gene Therapy for UBA5 Disorder

The National Institute of Neurological Disorders and Stroke (NINDS) has awarded Toloo Taghian, PhD, an assistant professor of genetic and cellular medicine and radiology at UMass Chan Medical School, a 5-year, $3.2 million grant to advance gene therapy development for UBA5 disorder (developmental and epileptic encephalopathy 44; DEE44), an ultrarare progressive neurological disease with approximately 40 diagnosed cases worldwide and no approved treatments. The grant will support investigation of disease mechanisms, biomarker identification, and preclinical development of an adeno-associated virus (AAV) AAV-based gene therapy approach targeting the UBA5 gene, which encodes a protein essential for the UFMylation pathway in cellular homeostasis.

6. Fractyl Health Receives Clinical Trial Authorization in Netherlands for Type 2 Diabetes Gene Therapy

Fractyl Health has received Clinical Trial Application authorization in the Netherlands to initiate a phase 1/2 first-in-human study of RJVA-001, an investigational AAV vector-based gene therapy designed to enable nutrient-responsive GLP-1 expression within pancreatic beta cells via a one-time endoscopic ultrasound-guided intrapancreatic infusion. The open-label, single-ascending-dose trial will enroll adults aged 35 to 70 years with inadequately controlled type 2 diabetes on stable background therapy including GLP-1 receptor agonists. Notably, RJVA-001 is the first AAV vector-based gene therapy to enter clinical-stage development for type 2 diabetes.