Around the Helix: Cell and Gene Therapy Company Updates – May 27, 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. Evaluating Miv-Cel for Stiff Person Syndrome

Mivocabtagene autoleucel (miv-cel; KYV-101; Kyverna Therapeutics) is an autologous, fully humanized anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy currently under clinical investigation in several antibody-mediated autoimmune neurological diseases, among them stiff person syndrome (SPS). CGTLive®’s sister site NeurologyLive® spoke with Amanda Piquet, MD, director of the Autoimmune Neurology Program and holder of the Celine Dion Foundation Endowed Chair in Autoimmune Neurology at the University of Colorado, about data from a phase 2 clinical trial evaluating miv-cel in SPS that she presented at the 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18 to 22 in Chicago, Illinois.

2. Dyne Submits BLA for Z-Rostudirsen in Exon 51 Skipping Duchenne Muscular Dystrophy

Dyne Therapeutics has submitted a biologics license application (BLA) to the FDA seeking accelerated approval of zeleciment rostudirsen (z-rostudirsen; formerly DYNE-251) for patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, marking a major regulatory milestone for the investigational exon-skipping therapy. The submission is supported by findings from the phase 1/2 DELIVER trial (NCT05524883), in which treatment with z-rostudirsen led to statistically significant increases in dystrophin production alongside improvements across multiple functional end points and a favorable safety profile.

3. Liminatus Pharma Enters Merger Agreement With InnocsAI to Acquire CAR-T and Antibody-Based Oncology Pipeline

Liminatus Pharma has announced a definitive merger agreement with InnocsAI LLC, a biotechnology company developing CAR-T and antibody-based therapies for hematologic malignancies and solid tumors. Under the terms of the agreement, InnocsAI will merge into a newly formed wholly owned subsidiary of Liminatus. “This proposed transaction represents a transformational opportunity for Liminatus to expand into next-generation oncology cell therapies,” Chris Kim, the CEO of Liminatus Pharma, said in a statement. “InnocsAI’s portfolio includes differentiated CAR-T technologies designed to address major limitations in current cancer therapies, including antigen escape, tumor heterogeneity, and the immunosuppressive tumor microenvironment.

4. Genethon and Ampersand Biomedicines Partner to Engineer Tissue-Specific AAV Capsids Aimed at Improving Gene Therapy Efficacy and Tolerability

Genethon and Ampersand Biomedicines have announced an exclusive research collaboration to develop next-generation adeno-associated virus (AAV) capsids with enhanced tissue specificity, with the goal of reducing the high vector doses currently required to achieve therapeutic effect in nonhepatic target tissues—a key driver of safety, tolerability, and cost concerns in AAV-based gene therapy. The partnership will combine Ampersand's Address, Navigate, Determine (AND) platform for cell-specific ligand identification with Genethon's AAV capsid engineering abilities, with an initial focus on skeletal muscle before planned expansion to additional tissues and organs.

5. Sartorius Opens €140 Million Competence Center in Germany to Expand CGT Component Manufacturing Capacity

Sartorius has opened a new cell and gene therapy competence center in Freiburg, Germany, representing an investment of more than €140 million and more than doubling the company's manufacturing capacity for quality-critical cell and gene therapy (CGT) materials, including cytokines, growth factors, and cell culture media. The 18,000-square-meter facility, which operates entirely without fossil fuels and has received Gold precertification from the German Sustainable Building Council, is fully good manufacturing practice (GMP)-compliant and can employ a workforce of up to 180.