Accelerating gene therapy development

The potential of gene therapies is finally being realized—therapies that can deliver life-changing treatments for a broad range of disorders.

At Atuka we have decades of experience when it comes to understanding complex biological issues and working with our collaborators to find bespoke solutions to their problems.

More recently, we have leveraged our expertise in rodent and NHP neuroanatomy and neurosurgery to become experts at delivering pharmacodynamic and biodistribution studies in the gene therapy area. Our access to a plentiful supply of NHPs also allows us to be your partner in evaluating the tropism of newly developed vectors.

The potential of gene therapies is finally being realized and these therapies can deliver life-changing treatments for a broad range of disorders.

Atuka’s Chief Innovation Officer Dr Patrick A. Howson explores the past, present, and future of gene therapy.

Gene therapy is a fast-developing field, with new technologies, such as direct genome editing and non-viral delivery systems, constantly evolving and being improved upon. At Atuka, we understand that all gene therapies are unique and that different gene therapies require different solutions for them to progress smoothly into the clinic. We pride ourselves on creating individual solutions that meet the unique gene therapy needs of our partners.

+ 20 biodistribution studies performed supporting regulatory submissions

+ Rodent and non-human primate studies

+ Access to large numbers of NHPs of both sexes and various ages

+ Wide range of routes of administration – PO, IV, IM, SC, IT, ICM

+ Direct injection into parenchyma—surgical delivery to discrete brain regions

+ Neurosurgical expertise

+ In-life assessments and biofluid collections

+ In-life imaging

+ Histological services – non-GLP toxicology assessments

+ Tissue collection including subbrain regions

+ Behavioural and postmortem endpoints to assess pharmacodynamics

+ Neutralizing antibody analysis