Around the Helix: Cell and Gene Therapy Company Updates – October 29, 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. AskBio’s Heart Failure Gene Therapy AB-1002 Shows Ability to Improve NYHA Class in Phase 1 Trial

AskBio’s AB-1002, an investigational adeno-associated virus (AAV) vector-based gene therapy, has demonstrated the ability to improve New York Heart Association (NYHA) class in patients with NYHA class III nonischemic heart failure with reduced ejection fraction (HFrEF) who were treated in the phase 1 NAN-CS101 clinical trial (NCT04179643).

2. CARsgen’s CAR-T Satri-Cel Significantly Reduces CA19-9 Levels in Patients With Pancreatic Ductal Adenocarcinoma

In a phase 1b clinical trial (CT041-ST-05, NCT05911217) for pancreatic ductal adenocarcinoma (PDAC), 5 of 6 patients (83.3%) treated with CARsgen’s Claudin18.2-directed chimeric antigen receptor T-cell (CAR-T) satricabtagene autoleucel (satri-cel) as an adjuvant therapy showed significant posttreatment decreases in carbohydrate antigen 19-9 (CA19-9).

3. Quarter Century Update: How can Partnerships Between Academia, Biotech, and Pharma be Better Structured?

In this episode, Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, and Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, discussed the evolving, but still imperfect relationship between academia and industry in advancing cell and gene therapies.

4. Intellia Puts Phase 3 Trials for Transthyretin Amyloidosis Gene Editing Therapy Nex-Z on Hold Following Grade 4 Liver AE

Intellia has temporarily paused dosing and screening of patients for both the phase 3 MAGNITUDE clinical trial (NCT06128629) and the phase 3 MAGNITUDE-2 clinical trial (NCT06672237), which are respectively evaluating the gene editing therapy nexiguran ziclumeran (nex-z, also known as NTLA-2001) for transthyretin amyloidosis with cardiomyopathy and polyneuropathy, after a patient in MAGNITUDE experienced a grade 4 liver adverse event (AE).

5. BioMarin to Leave Roctavian by the Wayside

BioMarin has announced that it will be divesting from valoctocogene roxaparvovec (val-rox, marketed as Roctavian), its gene therapy product for the treatment of adults with severe hemophilia A, and removing it from its portfolio. Although BioMarin maintains that the gene therapy product has "an important role to play" in hemophilia treatment, the company cited strategic priorities as the reason for the move. BioMarin noted that it is looking into out-licensing options for the product.

6. Secretome Brings Dilated Cardiomyopathy Cell Therapy to Clinical Trial

Secretome Therapeutics has launched a phase 1 clinical trial (NCT06560762), funded by a grant from The Marcus Foundation, that is evaluating STM-01, a neonatal cardiac progenitor cell (nCPC) therapy, for the treatment of heart failure in young adult patients with dilated cardiomyopathy. “Our preclinical published results in animals suggest that, compared to mesenchymal stem cells from older donors, STM-01 is uniquely potent at improving ejection fraction and treating heart failure,” Michael Davis, PhD, the director of the Emory and Children’s Heart Research and Outcomes Center and a professor of cardiology and biomedical engineering at the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University, said in a statement.