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ASGCT Conference

ESGCT Presentation

ASGCT conference

Investigating the oxygen control before and after fixed bed in the iCELLis® Nano bioreactor to create a more robust scale-down model for the iCELLis® 500+ bioprocess
Development of a high-throughput, miniaturized, semiautomated rapid transduction inhibition assay (TIA) for the characterization of anti-AAV antibodies in gene therapy
Lysis and clarification strategies for AAV suspension processes
Host-cell DNA impurity sizing in rAAV by an 18S rRNA gene-based ddPCR approach
Engineering AAV capsid variants to overcome pre-existing immunity and improve gene delivery to human liver

WORLDSymposium

  • Safety and efficacy of FLT190 for the treatment of patients with Fabry disease: Results from the MARVEL-1 phase 1/2 clinical trial

  • Design of GALILEO-1, a phase 1/2 safety and efficacy study of FLT201 in adult patients with Gaucher disease type 1

  • Safety and efficacy of FLT190 for the treatment of patients with Fabry disease: Results from the MARVEL-1 phase 1/2 clinical trial

Factor IX Expression within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients with Severe Hemophilia B: Long-term Follow-up Study of the B-AMAZE Program

Factor IX Expression within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients with Severe Hemophilia B: Long-term Follow-up Study of the B-AMAZE Program

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

Freeline Research Platform: Gaucher disease case study

Freeline Research Platform: Gaucher disease case study

Development of a GLA NAb Assay with a Fully-human, Neutralising IgG4 Positive Control to Characterise Antibody Response in Fabry Disease Patients

FLT201: AN AAV-MEDIATED GENE THERAPY FOR TYPE 1 GAUCHER DISEASE DESIGNED TO TARGET DIFFICULT TO REACH TISSUES

Generation of β-Glucocerebrosidase Variants with Increased Half-life in Human Plasma for Liver Directed AAV Gene Therapy for the Treatment of Type 1 Gaucher Disease

Follow-up on a Novel Adeno-Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Haemophilia B (HB) Patients (B-AMAZE Study)

Insight into the persistent clinical burden underlying problem joints, pain, and bleeding in adults from across Europe with haemophilia A and B: The CHESS II Study

Presentation – Data update B-AMAZE Phase 1/2 Study of FLT180a in Haemophilia B – 14th December 2020

Data update B-AMAZE Phase 1/2 Study of FLT180a in Haemophilia B

Multi-Centre Field Study of One-Stage and Chromogenic Factor IX Assays in Samples Containing the Factor IX Padua Variant

Mechanistic Evaluation of Factor IX-Padua Activity in Chromogenic FIX and Thrombin Generation Assays

Feasibility of Using Hospital Episode Statistics in England to Assess the Resource Use and Outcomes in Haemophilia

The Relationship between Societal Costs Associated with Haemophilia and Disease Severity: A Regression Analysis Using CHESS II Data

Modelling the Cost of Equivalent trough Level across Gene Therapy and Factor IX Replacement Therapy in Haemophilia B

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