Around the Helix: Cell and Gene Therapy Company Updates – June 11, 2025

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. CD19/20-Targeted KITE-363 Elicits Strong Response in LBCL

KITE-363 elicited responses in nearly all patients with relapsed/refractory large B-cell lymphoma (LBCL) who were treated with the dual CD19 and CD20-directed chimeric antigen receptor (CAR) T-cell therapy, according to data from a phase 1 study (NCT04989803) presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 30 to June 3, in Chicago, Illinois.

2. Lowering Barriers to Access for Liso-Cel Based on New Data

At ASCO Annual Meeting, Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, presented data from an analysis of more than 1,500 patients treated with liso-cel in clinical trials and the real-world setting. At the conference, CGTLive®'s sister site OncLive® interviewed Kamdar to learn more about the key takeaways from the presentation.

3. Kristina Jordahl, PhD, on Arlo-Cel's Potential to Destroy Multiple Myeloma Cells While Preserving Immune Function

Bristol Myers Squibb's GPRC5D-targeted chimeric antigen receptor (CAR) T-cell therapy arlocabtagene autoleucel (arlo-cel) is currently being evaluated for the treatment of multiple myeloma (MM) in a phase 2 clinical trial (NCT06297226) and in a phase 1 clinical trial (NCT04674813). In an interview with CGTLive®, Kristina Jordahl, PhD, a principal scientist at Bristol Myers Squibb, discussed a biomarker analysis of data from the phase 1 trial, comparing it to data from the phase 2 KarMMa clinical trial (NCT03361748) evaluating BCMA-directed CAR-T idecabtagene vicleucel (ide-cel) for the treatment of MM.

4. The Potential to Apply Immunotherapy DOC1021 to a Broad Array of Tumor Types

Diakonos Oncology is currently evaluating dubodencel (also known as DOC1021), an investigational autologous dendritic cell immunotherapy made from patients’ own tumor cells and peripheral blood mononuclear cells, for the treatment of gliobastoma in a phase 1 clinical trial. CGTLive® spoke with Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology, about the development plan for dubodencel, which is being assessed for other cancer types as well.

5. FDA Holds Roundtable Discussion on Cell and Gene Therapy

On June 5, 2025, the FDA held a roundtable discussion focused on cell and gene therapy, the recording of which can now be viewed on the agency's website. According to BiopharmaDive, speakers at the roundtable noted some recent success in the field of cell and gene therapy, but mainly focused on the industry and financial challenges holding the field back from reaching its full potential. “We estimate that over 100 rare disease gene therapy products that had reached clinical stage have been discontinued since 2023—not because of treatment failure, but because of the risk of market failure,” Terence Flotte, MD, the dean of the University of Massachusetts’ T.H. Chan School of Medicine and president of the American Society of Cell and Gene Therapy, said during the roundtable.

6. Nuevocor Snags IND Clearance for Gene Therapy Trial in LMNA DCM

Nuevocor has received clearance from the FDA for an investigational new drug (IND) application for NVC-001, an adeno-associated virus (AAV) vector-based gene therapy intended to treat LMNA-related dilated cardiomyopathy (LMNA DCM). In light of the IND clearance, the company intends to go forward with plans for a phase 1/2 ascending-dose clinical trial.

7. Capricor Gets AdComm Date for Deramiocel

Capricor Therapeutics announced that the FDA will hold an advisory committee (AdComm) meeting regarding the biologics license application (BLA) for deramiocel (also known as CAP-1002), on July 30, 2025. Deramiocel is an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. The BLA continues to have a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025.

8. FDA Confirms That Accelerated Approval Pathway is Open for Avidity's Del-Brax

The FDA has given Avidity Biosciences confirmation that an accelerated approval pathway is open for the company's investigational antibody oligonucleotide conjugate delpacibart braxlosiran (del-brax), which is in development for the treatment of facioscapulohumeral muscular dystrophy. Avidity plans to submit a BLA for del-brax to the agency in the second half of next year.