November 2026– date –
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Kyverna’s miv-cel: Phase 1 data for progressive MS and RMAT status
BioSpace USA Overview Kyverna Therapeutics reported that a single dose of miv-cel achieved robust CNS penetration and improved disability scores in patients with progressive multiple sclerosis (PMS). The Phase 1 trial showed sustained cl... -
FDA Gene Editing Guidance: BLA Process and IFS Framework 2026
Pharmaceutical Technology USA Overview The FDA has issued draft guidance allowing Biologics License Applications (BLAs) for individualized genome editing products to add patient-specific variants without new clinical trials. By utilizing... -
Sumitomo Pharma: First iPS-derived Parkinson’s drug transplant
Nikkei Japan Overview Sumitomo Pharma has performed the first clinical transplant of its iPS cell-derived Parkinson's disease treatment, "Amshepri," following its conditional and time-limited approval in March. The procedure was conducte... -
Osimertinib Cardiotoxicity: AMPK–LKB1 Signaling and iPSC Models
BIOENGINEER.ORG USA Overview Patient-derived iPSC cardiomyocytes reveal that susceptibility to osimertinib-induced cardiac dysfunction is linked to impaired AMPK–LKB1 signaling. Metformin cotreatment mitigated the decline in contraction ... -
CD73-Targeting CAR-NK Cells: Strategy for Lung Adenocarcinoma
BIOENGINEER.ORG USA Overview Engineered NK-92 cells targeting CD73 demonstrate the ability to destroy relapsed or refractory lung adenocarcinoma cells in preclinical models. By combining direct tumor lysis with the neutralization of aden... -
CAR-T-bridged allo-HSCT: KO-DLI rescue for mixed chimerism
BIOENGINEER.ORG Overview Residual CAR transgene levels at the time of transplantation may signal a risk of persistent CAR-T donor cells causing mixed chimerism with cytopenia. In three cases, CAR-target-knockout donor lymphocyte infusion... -
Zimislecel: Islet Cell Therapy’s Impact on Type 1 Diabetes Distress
Healio USA Overview Zimislecel, a stem cell-derived islet cell therapy from Vertex Pharmaceuticals, reduced HbA1c to below 7% and eliminated severe hypoglycemia in 12 adults with type 1 diabetes over one year. The therapy significantly l... -
SOD1 Gene Editing: Prime and Base Correction in Human iPSCs
BIOENGINEER.ORG USA Overview Researchers have successfully corrected two ALS-causing SOD1 mutations in human induced pluripotent stem cells (iPSCs) using prime and base editing. The study achieved correction efficiencies of approximately... -
FDA Personalized Gene Therapy: Draft Guidance for Rare Diseases
BioPharma Dive USA Overview The FDA has proposed a new regulatory framework for personalized gene therapies targeting different mutations within the same disease-causing gene. This "plug and play" approach allows for subsequent mutation ... -
Tetraploid iPS Cells: Enhancing Cardiac Function via Cell Fusion
Mirage News Japan Overview Researchers have established tetraploid human induced pluripotent stem (iPS) cells via cell fusion to create cardiomyocytes with enhanced functional capabilities. By replicating the natural chromosome doubling ...
