Around the Helix: Cell and Gene Therapy Company Updates – May 6, 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.

Have a cell and gene therapy news update you’d like to share with our editorial team? Tag us on social (@CGT_Live on X, or CGTLive on LinkedIn) and use #AroundTheHelix!

1. What are some of the misconceptions your colleagues might have about the use of CAR-T in neurology?

At the 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18 to 22 in Chicago, Illinois, Amanda Piquet, MD, FAAN, discussed how the potential use of CAR-T therapy in neurological and autoimmune diseases may differ from expectations based on its historical use in oncology.

2. Srikanth Muppidi, MD, on Evaluating Miv-Cel in Myasthenia Gravis

Srikanth Muppidi, MD, a clinical professor of adult neurology at Stanford University, presented findings from the phase 2 KYSA-6 clinical trial (NCT06193889) at the 2026 AAN Annual Meeting. The trial is evaluating mivocabtagene autoleucel (miv-cel; KYV-101), an investigational fully human antiCD19 chimeric antigen receptor T-cell (CAR-T) therapy, in patients with myasthenia gravis (MG). CGTLive®’s sister site NeurologyLive® sat down with Muppidi on the conference floor to discuss the results in more detail.

3. The Challenge of T-Cell Therapy Timing

In a discussion with ImmunoLogic cohosts Janna Minehart, MD, and Joseph Fraietta, PhD, Kristopher R. Bosse, MD, shared his thoughts on T-cell collection for patients being treated for neuroblastoma.

4. Bill Nye on Genetic Testing, Awareness, and the Future of Friedreich Ataxia Care

At the 2026 AAN Annual Meeting, a unique educational session brought together science communicator Bill Nye, neurologist Dr. Kuo, and a patient-researcher living with friedreich ataxia (FA) to examine real-world case studies of FA and explore FA disease recognition, progression, and emerging therapeutic approaches. The session aimed to bridge gaps between scientific understanding, clinical practice, and patient experience, while highlighting the evolving landscape of FA care, including the emergence of disease-modifying therapies and gene-targeted strategies. Shortly after the meeting, NeurologyLive^® spoke with Nye to gain his perspective on the importance of awareness, the role of genetic testing, and the responsibility of clinicians and scientists in communicating complex neurologic diseases.

5. J&J Discontinues CD20 and CD19-CD20 CAR-T Programs in LBCL

Johnson & Johnson has announced the discontinuation of its investigational CD20 monospecific CAR-T cell therapy and CD19-CD20 bispecific CAR-T programs for large B-cell lymphoma (LBCL), citing portfolio prioritization and the evolving LBCL treatment landscape rather than safety or efficacy concerns. Patients currently enrolled in ongoing trials will continue to receive support per study protocols. No clinical data were cited as contributing to the decision.

6. Apertura Gene Therapy, TSC Alliance Join Forces on IV-Delivered AAV Gene Therapy for Tuberous Sclerosis Complex

Apertura Gene Therapy and the TSC Alliance have announced a preclinical collaboration to develop gene therapy for tuberous sclerosis complex (TSC), a rare genetic disorder in which approximately 85% of patients experience seizures and two-thirds develop refractory epilepsy. Programs will utilize Apertura's TfR1 CapX™, an investigational adeno-associated virus (AAV) capsid designed for intravenous delivery with blood-brain barrier crossing capability, intended to deliver functional copies of TSC1 or TSC2. Work will be conducted through the TSC Alliance's Preclinical Consortium.