Technology Platform

  • THE BENEFITS

Technology Overview

AlveoGene InGenuiTy™ technology includes a proprietary and validated next-generation lentiviral delivery platform originally developed by the GTC specifically targeted for the treatment of respiratory diseases with high unmet need.1

Unlike may current gene therapy approaches, which require administration via IV bolus, respiratory gene therapies developed using the InGenuiTy™ platform can be delivered through a nebuliser, directly targeting the lung.  This facilitates the transduction of the lung epithelial cells with high efficiency, providing a long duration of action and sustaining these effects following repeated administration, if required.

AlveoGene also benefits from a comprehensive and interlinked IP estate with more than 10 patent families and reach through 2042.  It is further underpinned by significant know-how, particularly in the scalable manufacturing processes for its viral vectors.

1 Excluding the use of the CFTR gene, which is mutated in cystic fibrosis

Manufacturing Overview

  • rSIV.F/HN vector production is scalable
  • Vector has large packaging capacity (>10 kb) and allows the facile introduction of other transgenes for other indications
  • State-of-the-art innate safety features being replication incompetent
  • Animal component free
  • Scalable cGMP manufacture already developed for an upcoming cystic fibrosis clinical trial1
  • Manufacturing AATD product at scale expected to follow a similar course

REF 1: North American Cystic Fibrosis Conference 2022: NACFC 2022 | S01: Design of Clinical Trials to Evaluate Nucleotide-Based Interventions – YouTube: Talk 4 (EW Alton)