Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”), a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases, today presents six posters detailing research data from its proprietary gene therapy platform at the American Society of Gene and Cell Therapy (“ASGCT”) Annual Meeting 2021, taking place May 11-14, 2021.
"These data presented today at ASGCT highlight the strong scientific foundation that underlies our gene therapy programs and broader technology platform,” said Theresa Heggie, Chief Executive Officer of Freeline. “Collectively, these posters demonstrate our potential to innovate across capsid, protein engineering, transduction efficiency, analytics and manufacturing, with the goal of delivering better gene therapy products for patients. These presentations also are a harbinger of things to come as our platform capabilities have the potential to unlock new diseases for which gene therapy could provide transformative benefits for patients."
Presentation Highlights
- Established a 96-well high-throughput suspension platform to facilitate both rAAV manufacturing platform optimization and candidate screening and selection to enable the continued growth of the Company’s gene therapy pipeline and manufacturing processes.
- Developed and completed scale up of suspension cell-based AAV manufacturing which reduces process variability, eliminates animal-derived components and reduces overall cost of goods, thereby maintaining the high quality and potency of vectors manufactured with the Freeline iCELLis® commercial scale process.
- Developed a dosing assay strategy for AAV-based gene therapies that enables the accurate and reproducible quantification of vector genome titer, which is required across the product lifecycle of AAV-based gene therapies. Utilization of the dosing assay is critical to ensure consistent potency of vector across in vitro and in vivo disease model studies and patient dosing.
- Developed a universally applicable transducing titer assay for AAV-based gene therapies that quantifies the percentage of liver cells functionally expressing a gene of interest at a given dose. This assay is complementary to traditional in vitro potency assays and provides an orthogonal tool to improve the understanding and quantification of functional AAV transduction for liver-directed AAV gene therapies.
- FLT190, the Company’s AAV gene therapy candidate for Fabry disease, demonstrated GLA uptake and metabolic cross correction in Fabry disease relevant cell lines, with a significant proportion of GLA enzyme effectively delivered to lysosomal compartments, where it exerted its degradative function as measured by Gb3 clearance in Fabry cells. These results are supportive of the Company’s ongoing development of FLT190, which is being evaluated in an ongoing Phase 1/2 clinical trial.
- FLT201, the Company’s AAV gene therapy candidate for Gaucher disease Type 1, administered as a single infusion in animal models, produced steady-state expression of a novel GCase variant (GCasevar85) in plasma to drive uptake of GCase in target cells and tissues. Importantly, restoration of tissue GCase activity achieved with FLT201 resulted in clearance of lipid storage cells and normalization of lyso-Gb1 biomarker levels in all tissues examined.
Presentations Details
Development of a 96-Well Plate-Based High-Throughput System for rAAV Manufacturing Platform Optimization and Candidate Selection
Development and Scale Up of a Suspension Cell-Based AAV Manufacturing Process
Defining a Reliable Quantification Assay Strategy for Adeno-Associated Virus (AAV)-Based Gene Therapies
Development of an Assay to Measure Transduction Efficiency of Adeno-Associated Virus (AAV)-Based Gene Therapies
GLA Uptake and Metabolic Cross Correction in Fabry Disease Relevant Cell Lines: A Rationale for Liver-Directed AAV Gene Therapy
FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1
These poster presentations are available on the Investors section of the Freeline website.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (“AAV”) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing protein into the patient’s bloodstream. The Company’s integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in Hemophilia B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.
Forward-Looking Statements
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