Around the Helix: Cell and Gene Therapy Company Updates – August 6, 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. Roche Slammed With Negative EMA CHMP Opinion for Sarepta’s DMD Gene Therapy Elevidys

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has issued an opinion recommending against conditional marketing authorization for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys) for patients who are ambulatory, according to Roche, which has commercial rights for the gene therapy product in markets outside the United States.

2. Patient Dies After Receiving Monoclonal Antibody Lymphodepletion Regimen in LBCL Trial for Allogene’s Cema-Cel

A patient treated in Allogene Therapeutics’ phase 2 ALPHA3 clinical trial (NCT06500273), which is evaluating investigational CD19-directed allogeneic chimeric antigen receptor T-cell (CAR-T) therapy cemacabtagene ansegedleucel (cema-cel) as a first-line consolidation treatment for large B-cell lymphoma (LBCL), has died. Notably, the patient was treated in an arm of the trial evaluating a lymphodepletion regimen consisting of fludarabine and cyclophosphamide and antiCD52 monoclonal antibody ALLO-647, and the patient’s death was attributed to this lymphodepletion regimen rather than to cema-cel itself.

3. FDA Gives Priority Review to BMS’s Supplemental BLA for Liso-Cel in R/R Marginal Zone Lymphoma

Bristol Myers Squibb’s supplemental biologics license application (sBLA) for marketed autologous CD19-directed CAR T-cell therapy lisocabtagene maraleucel (liso-cel, marketed as Breyanzi) for marginal zone lymphoma (MZL) has been accepted by the FDA with priority review. The Prescription Drug User Fee Act (PDUFA) goal date for the sBLA has been set for December 5, 2025.

4. BioCardia Reveals Plans to Request Meeting With FDA Regarding CardiAMP Heart Failure Cell Therapy

BioCardia has stated that it plans to request a meeting with the FDA to discuss the pathway to approval for its CardiAMP cell therapy system, an investigational autologous cell therapy for the treatment of ischemic heart failure.

5. Galapagos Snags FDA RMAT Designation for R/R Mantle Cell Lymphoma CAR-T

Galapagos' GLPG5101, an investigational CD19-directed CAR-T therapy product, has received regenerative medicine advanced therapy (RMAT) designation from the FDA for the treatment of relapsed/refractory (r/r) mantle cell lymphoma (MCL). The RMAT designation was granted based on data from a subset of patients with r/r MCL treated in the phase 1/2 ATALANTA-1 clinical trial (NCT06561425).

6. Adicet to Leverage MaxCyte's Flow Electroporation Technology and ExPERT Platform in New Collaboration

Adicet Bio and MaxCyte have signed a platform license agreement that will enable the former company to utilize MaxCyte’s Flow Electroporation technology and ExPERT platform for research, clinical, and commercial purposes. “We are pleased to support Adicet Bio as they expand their allogeneic gamma delta T-cell manufacturing capabilities to include nonviral gene editing delivery,” Maher Masoud, JD, the president and CEO of MaxCyte, said in a statement.

7. INmune Bio's INKmune Hits End Points in mCR Prostate Cancer Trial

INmune Bio's phase 1/2 CaRe PC clinical trial (NCT06056791), which is evaluating its cell therapy INKmune for the treatment of metastatic castration-resistant prostate cancer (mCRPC), has met its primary and secondary end points. The company additionally noted that the trial is no longer enrolling new patients.