Around the Helix: Cell and Gene Therapy Company Updates – July 9, 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. ProKidney’s Diabetes and CKD Cell Therapy Rilparencel Improves eGFR Slope Annual Decline in Phase 2 Trial

ProKidney’s rilparencel, a renal autologous cell therapy intended to treat diabetes and chronic kidney disease (CKD), has improved annual decline in estimated glomerular filtration rate (eGFR) slope in patients treated in the phase 2 REGEN-007 clinical trial (NCT05018416).

2. Cell Therapy’s Lot in Solid Tumors: Challenges and Opportunities

With the aim of getting a cross-section of the current state of progress in the field, CGTLive reached out to several experts to get their insight into several of the notable approaches currently being assessed.

3. β-Thalassemia Gene Therapy in the Context of the Broader World

Tami John, MD, a clinical associate professor at Stanford Medicine, discussed the importance of building community for patients with transfusion-dependent β-thalassemia.

4. China's NMPA Accepts Satri-Cel NDA for Filing

CARsgen's new drug application (NDA) for Claudin18.2-directed chimeric antigen receptor T-cell (CAR-T) satricabtagene autoleucel (satri-cel), which is intended to treat gastric/gastroesophageal junction adenocarcinoma (G/GEJA), has been accepted for review by China's National Medical Products Administration (NMPA) for Claudin18.2-positive advanced G/GEJA in patients who have received at least 2 prior lines of unsuccessful therapy.

5. Concentra Biosciences to Bring CARGO Therapeutics Into the Fold

CARGO Therapeutics, which is developing CAR-T products for oncology indications, has entered an agreement to be purchased by Concentra Biosciences, following a strategic review that determined it would be in the best interests of CARGO stockholders. According to FirstWord Pharma, CARGO was in a difficult spot financially, having shut down its research and development operations, let go of 90% of its staff, and terminated its lead program earlier in 2025.

6. Solid Biosciences' CPVT Gene Therapy SGT-501 Cleared for Phase 1b Trial

Solid Biosciences' SGT-501, an investigational AAV vector-based gene therapy intended to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), has received clearance of an investigational new drug application by the FDA and a clinical trial application by Health Canada. In light of the clearances, the company plans to launch a phase 1b clinical trial in the final quarter of this year.

7. Orchard's Registrational Trial for MPS-IH Gene Therapy Doses Final Participant

Orchard Therapeutics has dosed the last patient in the phase 3 HURCULES clinical trial (NCT06149403), which is evaluating OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy intended to treat the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). “The completion of study enrollment nearly a year ahead of schedule underscores the urgent medical need that still exists in MPS-IH, and the hopefulness of physicians and patients to contribute to the development of new treatment options,” Leslie Meltzer, PhD, the chief medical officer of Orchard Therapeutics, said in a statement.

8. Mustang Bio Snags Orphan Drug Designation for IL13Ra2-Directed CAR-T MB-101

Mustang Bio's MB-101, an investigational IL13Ra2-directed CAR-T therapy, has received orphan drug designation from the FDA for the treatment of recurrent diffuse and anaplastic astrocytoma (astrocytomas) and glioblastoma (GBM). Mustang is evaluating the potential of MB-101 in combination with MB-108, an oncolytic virus also being developed by the company, to treat recurrent GBM and high-grade astrocytomas. Together the regimen is referred to as MB-109.