Around the Helix: Cell and Gene Therapy Company Updates – May 20, 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. GS-100 Gene Therapy Demonstrates Early Activity in NGLY1 Deficiency

Interim findings from the ongoing first-in-human phase 1/2/3 trial of GS-100 (Grace Sciences), an investigational intracerebroventricular (ICV) adeno-associated virus serotype 9 (AAV9) gene therapy for NGLY1 Deficiency, showed early improvements in motor and cognitive function among treated children, alongside a dose-dependent safety profile that informed dose selection for the pivotal portion of the study. Presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting by Becky Schweighardt, PhD, and colleagues, the analysis included 7 patients treated across 4 dose cohorts in the ongoing open-label study (NCT06199531).

2. Assessing Miv-Cel for Myasthenia Gravis

Mivocabtagene autoleucel (miv-cel; KYV-101; Kyverna Therapeutics) is an investigational autologous, fully humanized anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy currently in clinical evaluation for myasthenia gravis (MG). CGTLive®’s sister site NeurologyLive® spoke with Srikanth Muppidi, MD, a clinical professor of adult neurology at Stanford University, about phase 2 findings from the KYSA-6 clinical trial (NCT06193889) evaluating miv-cel in MG.

3. Phase 3 HOPE-3 Data Reinforce Skeletal and Cardiac Benefits of Deramiocel in Duchenne Muscular Dystrophy

New phase 3 findings presented at the 2026 ASGCT Annual Meeting further strengthened the case for deramiocel, an investigational allogeneic cell therapy from Capricor Therapeutics, as a potential treatment for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy and progressive skeletal muscle decline. The data come at a pivotal regulatory moment for the therapy, with the FDA currently reviewing the biologics license application (BLA) and a Prescription Drug User Fee Act (PDUFA) date set for August 22, 2026.

4. The FDA’s Approval of High-Dose Nusinersen: What Doctors Should Know

Thomas Crawford, MD, a pediatric neurologist at Johns Hopkins with more than 30 years of experience in spinal muscular atrophy, shared his thoughts on what his colleagues should know about the FDA’s March 30, 2026, approval of a new higher-dose formulation of nusinersen (Spinraza; Biogen).

5. FDA Clears Qihan’s IND for Dual-Target CAR-T QT-019C

Qihan Biotech has received an investigational new drug (IND) application clearance from the FDA for QT-019C, an investigational allogeneic, off-the-shelf CAR-T cell therapy engineered to simultaneously target CD19 and BCMA for the treatment of autoimmune diseases. Developed using the company's proprietary QUIET multiplexed gene editing platform, QT-019C is designed to mitigate graft-versus-host disease through elimination of surface T-cell receptor expression. Another distinguishing design feature is its intended compatibility with low-intensity or potentially eliminated lymphodepleting preconditioning—an approach that, if validated clinically, could broaden patient eligibility beyond those able to tolerate conventional chemotherapy regimens.

6. First Canadian Patient Receives Hemgenix for Hemophilia B Outside of a Clinical Trial

CSL Canada has announced that the first Canadian patient has received Hemgenix (etranacogene dezaparvovec; CSL Behring), an AAV5-based in vivo gene therapy for hemophilia B, at London Health Sciences Centre in London, Ontario—marking the first administration of the therapy in Canada outside a clinical trial setting. Notably, following a pan-Canadian Pharmaceutical Alliance agreement, the therapy is also now publicly reimbursed under the Exceptional Access Program in Ontario and British Columbia. Additional provinces are also being considered for this.