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Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from chronic debilitating diseases.
Making gene therapy a reality
Since the foundation of Freeline in 2015, the aim has been to advance curative gene therapy treatments for chronic systemic diseases which have not to date been treatable. An entrepreneurial spirit and a vision to challenge the standard of care to create better lives has been the driving force behind Freeline.
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7
AUG
BTIG Virtual Biotechnology Conference
10:00 – 10:25 (UTC-05:00) Eastern Time (US & Canada)
Chief Executive Officer Michael Parini will participate in a fireside chat. To join the conference, email info@btig.com.
Gene Therapy
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Gene Therapy
The evaluation demonstrated preclinical proof-of-concept of FLT190 for the treatment of Fabry disease. In vivo studies demonstrated several features of liver-directed gene transfer for Fabry disease that may have considerable therapeutic value, including α-Gal A over-expression that drives cellular uptake; stable long-term α-Gal A expression; normalization of Lyso-Gb3 storage material in plasma and key disease tissues; normalization of Gb3 storage material in plasma, heart, urine; and significant clearance of Gb3 storage material from kidney.
The Journal of Gene Medicine
Evaluation of life cycle defective adenovirus mutants for production of adeno‐associated virus vectors
The Journal of Gene Medicine
The study indicates the advantages of using a ∆pTP mutant adenovirus rather than adenovirus wild‐type as a helper virus for AAV2 production and also indicates that temperature shifts to lower temperatures may improve AAV2 vector production rates.
Molecular Genetics and Metabolism
Liver-directed gene therapy corrects Fabry disease in mice
Elsevier
We examined the therapeutic efficacy of GLA gene therapy in Fabry mice using recombinant adeno-associated virus (rAAV) vectors, directed by the liver-specific promoter (FRE1). The data resulting from this study provides strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease.