FDA Activity Recap: December 2025 Features Multiple Approvals, RMAT Designation, and More

Last month, December 2025, the CGTLive^® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem. Last month proved no different, with the FDA approving lisocabtagene maraleucel (liso-cel, marketed as Breyanzi) for relapsed/refractory (r/r) marginal zone lymphoma (MZL) and approving etuvetidigene autotemcel (Waskyra; Fondazione Telethon ETS) as the first cell-based gene therapy approved for the treatment of Wiskott-Aldrich syndrome (WAS). Our team has highlighted these, and several other important actions, below.

Click the read more buttons for more details and information about each update.

FDA Approves BMS’s Liso-Cel for R/R Marginal Zone Lymphoma

December 4, 2025 — The FDA has approved Bristol Myers Squibb’s autologous CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy liso-cel for the treatment of r/r MZL in adults who have previously been treated with at least 2 lines of systemic therapy.

The agency's decision was based on results from an r/r MZL cohort in the phase 2 TRANSCEND FL clinical trial (NCT04245839), a multicenter, open-label study. The cohort included adults with r/r MZL who had previously been treated with 2 or more previous lines of systemic therapy or who had relapsed following hematopoietic stem cell transplant (HSCT) and was open to patients with an Eastern Cooperative Oncology Group performance status of 1 or lower.

Data from the r/r MZL cohort were presented earlier this year at the 2025 International Conference on Malignant Lymphoma (ICML), held June 17 to June 21 in Lugano, Switzerland. Among the 66 efficacy evaluable patients with R/R MZL who were treated with liso-cel, the overall response rate (ORR) was 95.5% (95% CI: 87.3%-99.1%; one-sided P <.0001) and the complete response rate was 62.1% (95% CI: 49.3%-73.8%; one-sided P < .0001). The 24-month rate for duration of response was 88.6% (median follow-up, 21.6 months), the 24-month rate for progression-free survival was 85.7% (median follow-up, 23.8 months), and the 24-month rate for overall survival was 90.4% (median follow-up, 24.5 months).

FDA Approves Waskyra as First Gene Therapy for Wiskott-Aldrich Syndrome

December 10, 2025 — The FDA has approved Waskyra as the first cell-based gene therapy approved for the treatment of WAS. Its indication is specifically for pediatric patients aged 6 months and older and adults with a mutation in the WAS gene for whom HSCT is appropriate but a suitable HLA-matched related donor is unavailable.

The approval marks a milestone for the treatment landscape of WAS, a rare, life-threatening, X-linked primary immunodeficiency characterized by thrombocytopenia, recurrent infections, eczema, autoimmunity, and elevated risk for hematologic malignancies. Historically, therapeutic options have been limited to supportive care and allogeneic HSCT, the latter of which is most successful when performed early in life and with a fully matched sibling donor—a scenario unfortunately unavailable to many patients.

“Today’s approval is a transformative milestone for patients with Wiskott-Aldrich syndrome, offering the first FDA-approved gene therapy that uses the patient's own genetically corrected hematopoietic stem cells to treat the disease,” Vinay Prasad, MD, MPH, the chief medical and scientific officer and director of the FDA Center for Biologics Evaluation and Research, said in a statement.

Latus Bio's CLN2 Disease Gene Therapy Cleared for US Trial

December 3, 2025 — Latus Bio has received clearance of an investigational new drug (IND) application from the FDA for LTS-101, an adeno-associated virus (AAV) vector-based gene therapy candidate for the treatment of central nervous system manifestations of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Alongside the IND clearance, LTS-101 also received a slew of designations from the FDA, including orphan drug designation, rare pediatric disease designation, and fast track designation.

Senti Bio's SENTI-202 Snags RMAT Designation

December 10, 2025 — Senti Bio has received a regenerative medicine advanced therapy (RMAT) designation from the FDA for SENTI-202, an investigational allogeneic chimeric antigen receptor natural killer (CAR-NK) cell therapy candidate intended to treat r/r acute myeloid leukemia (AML) and myelodysplastic syndrome. The RMAT designation specifically applies to treating adults with r/r AML. Notably, SENTI-202 received orphan drug designation from the FDA earlier in 2025.

“Leveraging our Logic Gate technology, SENTI-202 has continued to demonstrate its ability to aggressively kill cancer cells while protecting normal cells for hard-to-treat cancers such as AML, a central challenge in oncology,” Kanya Rajangam, MD, PhD, the chief Medical Officer of Senti Biosciences, said in a statement. “We view the FDA’s decision to grant RMAT and orphan drug designations to SENTI-202 as major milestones for the AML patient community and we look forward to working with regulators to develop this potentially first-in-class treatment as quickly as possible and to accelerate a paradigm shift in how we treat other difficult cancers.”

FDA Lifts Clinical Hold on Tenaya's MYBPC3-Associated Hypertrophic Cardiomyopathy Gene Therapy Trial

December 17, 2025 — The FDA has lifted a clinical hold that it had previously placed on the phase 1b/2a MyPeak-1 clinical trial (NCT05836259), which is evaluating TN-201, an investigational AAV-vector based gene therapy, for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). The hold was originally place by the FDA in November 2025 when Tenaya reached out to the agency explaining its intent to make a protocol amendment to MyPeak-1. Tenaya now states that all concerns leading to the clinical hold have been addressed and that it will resume dosing in the trial once it implements the protocol amendments.