Around the Helix: Cell and Gene Therapy Company Updates – April 8, 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. ImmunoLogic, Episode 9: "Rewiring Immunotherapy for High-Risk Neuroblastoma" With Kristopher R. Bosse, MD

In this episode, the cohosts speak with Kristopher R. Bosse, MD, an attending physician in the Division of Oncology at Children's Hospital of Philadelphia, about how immunotherapy is reshaping treatment for high-risk neuroblastoma. Bosse, Fraietta, and Minehart cover topics including why high-risk neuroblastoma remains so hard to cure; the state of immune-based therapies in neuroblastoma; the place of GD2-directed therapy in the treatment course; tumor heterogeneity, GPC2 biology, and antigen loss under chimeric antigen receptor (CAR) pressure; and more.

2. Context, Evidence, and Ongoing Questions Regarding the FDA's Approval of High-Dose Nusinersen in SMA: Angela Lek, PhD

On March 30, 2026, the FDA has approved a new higher dose strength for nusinersen (Spinraza; Biogen) as a treatment for patients with spinal muscular atrophy (SMA), almost 10 years after the therapy’s original approval. Following the approval, NeurologyLive® sat down with Angela Lek, PhD, MDA's chief research officer, to hear her perspective on the FDA’s decision.

3. PDUFA for Orca Bio’s Orca-T Pushed Back

The FDA’s Prescription Drug User Fee Act (PDUFA) target action date for Orca Bio’s biologics license application (BLA) for Orca-T, an investigational allogeneic cell therapy product intended to treat various hematologic malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndromes (MDS), has been pushed back to July 6, 2026. The FDA extended the review timeline after the company submitted updated chemistry, manufacturing and controls (CMC) information to accommodate FDA requests, considering the CMC information submission a Major Amendment. The PDUFA action date for the BLA had originally been set at April 6, 2026, when the FDA first accepted the BLA.

4. FDA Accepts Ultragenyx’s New BLA Submission for MPS IIIA Gene Therapy UX111

The FDA has accepted Ultragenyx’s resubmission of a BLA for UX111 (rebisufligene etisparvovec), an adeno-associated virus serotype 9 vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome). The new PDUFA action date for the BLA has been set at September 19, 2026. Notably, a previous BLA submission for UX111 was met with a complete response letter from the FDA in July 2025.

5. Wugen’s Allogeneic CAR-T Soficabtagene Geleucel Enters FDA CMC Readiness Pilot

Wugen’s investigational allogeneic antiCD7 CAR-T therapy soficabtagene geleucel (sofi-cel), in pivotal evaluation (NCT06514794) for relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma (R/R T-ALL/T-LBL), has been selected for the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program. The selection is intended to facilitate early, structured FDA–sponsor interactions on CMC strategy and manufacturing readiness in advance of a potential BLA, and complements sofi-cel’s existing breakthrough therapy, regenerative medicine advanced therapy, fast track, orphan drug, and rare pediatric disease designations.

6. Virica–FUJIFILM Biosciences Collaboration Targets More Efficient AAV Vector Manufacturing

Virica Biotech and FUJIFILM Biosciences have entered a Canada–Japan Co‑Innovation Program–supported collaboration to codevelop an off-the-shelf enhancer–media combination aimed at improving AAV vector production. The effort will optimize Virica’s Viral Sensitizer (VSE) formulations for use with FUJIFILM Biosciences’ BalanCD HEK293 suspension system, with the goal of increasing AAV yields and process robustness for academic and commercial producers facing scalability and cost-of-goods constraints in in vivo gene therapy manufacturing.