Cell & Gene Therapy

Updated NCCN multiple myeloma guidelines establish a dedicated MRD framework and specifically name Adaptive Biotechnologies' clonoSEQ as an FDA-cleared assay.

SNIPR has dosed its first Phase 2 patient with SNIPR001, expanding the E. coli bloodstream infection trial to 66 patients across ten sites including MSK

FAYUVI becomes the first approved treatment for MPS IIIA, backed by eight years of follow-up data showing significant cognitive benefit over natural history.

Tempest secures an exclusive option on Senlang's CD7-targeted lentiviral vector platform, adding a Phase 1 multiple myeloma programme to its in vivo CAR

The Palo Alto radiosensitizer biotech said its UK-led breast cancer study is now moving toward a 2027 efficacy readout, as two further cancer trials

Tenaya Therapeutics has secured RMAT designation for TN-401, its AAV9-based gene therapy for PKP2-associated ARVC, backed by Phase 1b/2 interim data.

The Danish cell-counting specialist reported DKK 511m in revenue and a 55% EBITDA margin for 2025/26, with automation demand accelerating instrument sales.

All 15 patients have been dosed with RTX001 in the Phase I/II EMERALD trial; interim data are expected in Q4 2026.

Legend Biotech will show long-term CARTITUDE-2 and real-world cilta-cel data at the International Myeloma Society meeting in Glasgow this month.

IMUNON has partnered with the NCI's CTEP to trial its IL-12 DNA immunotherapy in additional solid tumour indications and combinations beyond ovarian cancer.

All five Cohort 1 participants showed clinically meaningful visual improvement, and the FDA has aligned on a potential pivotal endpoint for the rare retinal disease

The Seattle-based biotech will use proceeds to run its ILLUMINATE Phase 2 study of BCB-276 in diffuse intrinsic pontine glioma, a universally fatal

The CRISPR biotech drew an initial $75m at closing, with further tranches tied to milestones for its HAE gene-editing candidate lonvo-z.

Eledon Pharmaceuticals has submitted an IND for a registrational islet cell transplant study of tegoprubart, while reaffirming its Q4 2026 Phase 3 kidney trial start.

All 16 TG4050-treated HNSCC patients remained relapse-free at 41 months' median follow-up, with full Phase 1 data now published in Nature Communications.

The Alameda-based CRISPR company began a Phase 1 trial of its epigenetic LDL-C therapy and secured $25m in non-dilutive CIRM grants alongside

The North Carolina gene therapy company will share Cohort 1 topline results for its BEST1 retinal disease programme at an investor webcast and three ophthalmology

Peer-reviewed results show a 61% mean reduction in daily cornstarch intake at Week 96, with glycaemic control maintained across both treatment groups.

Ocugen has initiated its Phase 3 ArMaDa3 trial of OCU410 for geographic atrophy, backed by FDA RMAT designation and a BLA filing targeted for 2028.

IBA has signed a contract with FUSVE to bring proton therapy to Brazil for the first time, with a focus on paediatric cancer care.