Cell therapy weekly: first iPSC therapy holding duo-combo recognition
This week: Genethon (Paris, France) and AskBio (NC, USA) announced an exclusive licensing agreement for an adeno-associated virus (AAV) gene therapy, while ImmunityBio, Inc. (CA, USA) reported on the latest trial update for its CAR-NK cell therapy for Waldenström Non-Hodgkin’s lymphoma. Plus, iRegene Therapeutics (Wuhan, China) announced that a Regenerative Medicine Advanced Therapy designation was granted to its allogeneic induced pluripotent stem cell (iPSC)-derived therapy, adding to the therapy’s regulatory recognitions.
The news highlights:
- Licensing agreement for AAV gene therapy
- Lymphoma CAR-NK cell therapy trial update
- iPSC therapy holding duo-combo recognition
Licensing agreement for AAV gene therapy
Genethon has entered into an exclusive, worldwide licensing agreement with AskBio, a subsidiary of Bayer AG (Leverkusen, Germany), granting rights to a patented component of the gene therapy AB-1009. AB-1009 is an AAV gene therapy under development for the treatment of late-onset Pompe disease, a rare genetic disorder caused by a deficiency in the GAA enzyme. Pompe disease, which affects an estimated 5,000 to 10,000 people globally, leads to severe muscle weakness and wasting, loss of mobility, and, in many cases, premature death.
The approach of AB-1009 involves the use of a transgene encoding a truncated form of the GAA enzyme, which has demonstrated preclinical efficacy. This critical component, protected by patents held by Genethon and other French institutions, is now licensed to AskBio for the continued development of a gene therapy product aimed at treating Pompe disease. Askbio anticipates recruiting its first patient for clinical trial in early 2026.
“Genethon is delighted with this agreement with AskBio, which enables the development of a gene therapy for patients suffering from this particularly severe condition,” said Frédéric Revah, CEO of Genethon. “This agreement demonstrates the quality of Genethon’s research and its ability to design innovative treatments, driven by its mission to provide therapeutic solutions to patients with rare diseases.”
Lymphoma CAR-NK cell therapy trial update
Updated follow-up data from the ongoing QUILT-106 clinical study (NCT06334991) evaluating an off-the-shelf allogeneic CD19 chimeric antigen receptor natural killer (CAR-NK) cell therapy, indicated for Waldenstrom Lymphoma, has demonstrated encouraging results. Waldenström Non-Hodgkin’s lymphoma is a slow-growing B-cell lymphoma characterized by abnormal production of monoclonal immunoglobulin M antibodies. Developed by ImmunityBio, Inc., the CAR-NK therapy has been developed to express a CD19-specific CAR and high-affinity CD16 receptor, enabling dual anti-tumor mechanism of direct CAR-mediated cytotoxicity and enhanced antibody-dependent cellular cytotoxicity when paired with anti-CD20 monoclonal antibody rituximab.
To date, four patients with Waldenström Non-Hodgkin’s lymphoma have been enrolled in the trial, all of whom remain in clinical disease control. Patients treated so far have sustained complete responses for at least 15 months, and ongoing, with 100% disease control observed to date. Notably, patients who had failed prior standard-of-care treatments achieved disease control after receiving a total of eight doses without lymphodepletion and chemotherapy. This marks the first chemotherapy-free, lymphodepletion-free CAR-NK cell therapy to achieve 100% disease control, with all treatments administered entirely in an outpatient setting.
“These data highlight a favorable safety and efficacy profile that is particularly important for patients with indolent yet incurable lymphomas,” said Lennie Sender, Chief Medical Officer, Liquid Tumors and Cell Therapy at ImmunityBio. “To date, all patients have been treated as outpatients with no serious adverse events, demonstrating the feasibility of delivering potent cellular immunotherapy without the morbidity traditionally associated with cell-based treatments.”
iPSC therapy holding duo-combo recognition
iRegene Therapeutics’ lead product NouvNeu001, an allogeneic iPSC-derived cell therapy, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA; MD, USA) for the treatment of Parkinson’s disease. NouvNeu001 now holds both FDA Fast Track Designation, which was granted in August 2025 and now RMAT.
“The RMAT designation is a pivotal regulatory advancement for NouvNeu001 and a strong endorsement of its clinical value. This designation reflects the FDA’s recognition of NouvNeu001’s potential to address a serious unmet medical need in Parkinson’s disease and provides a structured framework for deep, efficient collaboration with the agency. We are committed to leveraging this opportunity to accelerate our global clinical development program and bring this transformative therapy to patients as early as possible,” said iRegene’s Chief Medical Officer Meng Cai.
The therapy is designed to replace lost dopaminergic neurons, restore disrupted neural circuits and enable patients to regain endogenous dopamine production, slowing or preventing disease progression.
Data from a Phase I trial show significant clinical efficacy, with improvements in MDS-UPDRS Part III motor scores, the gold standard for evaluating motor function in Parkinson’s disease. At 12 months, the low-dose cohort demonstrated improvements of 30.6 points These improvements continued through 15 months post-treatment
