Accelerated Approval pathway for our ST-920 gene therapy product candidate for the treatment of Fabry disease.

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Cell Therapy

Feasibility of manufacture of chimeric antigen receptor-regulatory T cells from patients with end-stage renal disease
July 17th, 2023
Human Leukocyte Antigen Class I Molecule A* 02-Chimeric Antigen Receptor Regulatory T Cells in Renal Transplantation
October 4th, 2022
2020 White Paper on Recent Issues in Bioanalysis: Vaccine Assay Validation, qPCR Assay Validation, QC for CAR-T Flow Cytometry, NAb Assay Harmonization and ELISpot Validation (Part 3 – Recommendations on Immunogenicity Assay Strategies, NAb Assays, Biosimilars and FDA/EMA Immunogenicity Guidance/Guideline, Gene & Cell Therapy and Vaccine Assays)
March 18th, 2021
Highly Efficient Gene Editing of Cystic Fibrosis Patient-derived Airway Basal Cells Results in Functional CFTR Correction
May 7th, 2020
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
May 7th, 2020
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.
August 24th, 2018
Functional footprinting of regulatory DNA.
August 24th, 2018
Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.
August 24th, 2018
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.
August 24th, 2018
Off-the-shelf, steroid-resistant, IL13Rα2-specific CAR T cells for treatment of glioblastoma
August 24th, 2018