Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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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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).
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.
Tami John, MD, a clinical associate professor at Stanford Medicine, discussed the importance of building community for patients with transfusion-dependent β-thalassemia.
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.
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.
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.
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.
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.
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