TRANSFORMATIVE GENE TECHNOLOGY FOR LIFE CHANGING CURES

Zegen Our service Platform technology | Zegen Therapeutics

Gene therapy offers potential life-long cures by correcting for the missing or mutated genes that cause genetic diseases. Safe and efficacious gene therapy treatments are relatively new in translational medicine, however gene therapy has been investigated and pursued by scientists for the past five decades.
One of the breakthrough of gene therapy in brain diseases came from our founder Professor Paul Carney in 2013. Professor Carney has developed and tested a somatostatin gene therapy treatment a epilepsy rat model that produced transformative results. The techniques used in this treatment approach have been transferable, creating a new wave of gene therapy products which ZeGen is developing to treat a variety of genetic diseases. The basic components of our approach are based on recent success in gene therapy development and commercialization: an AAV-capsid technology, intrathecal delivery and an efficient manufacturing process.

Transformative Gene Technology for Life Changing Cures
Zegen Our service Platform technology | Zegen Therapeutics

Gene therapy offers potential life-long cures by correcting for the missing or mutated genes that cause genetic diseases. Safe and efficacious gene therapy treatments are relatively new in translational medicine, however gene therapy has been investigated and pursued by scientists for the past five decades.
One of the breakthrough of gene therapy in brain diseases came from our founder Professor Paul Carney in 2013. Professor Carney has developed and tested a somatostatin gene therapy treatment a epilepsy rat model that produced transformative results. The techniques used in this treatment approach have been transferable, creating a new wave of gene therapy products which ZeGen is developing to treat a variety of genetic diseases. The basic components of our approach are based on recent success in gene therapy development and commercialization: an AAV-capsid technology, intrathecal delivery and an efficient manufacturing process.

Next-Generation Platform Technologies

We are developing next-generation technologies to optimize key components of our AAV-based gene therapies, including redosing, transgene regulation and capsid development.

Novel Capsid Packaging

Our novel AAV capsid platform allows us to:

  • Utilize machine learning, capsid shuffling and directed evolution to improve targeted delivery
  • Deliver targeted gene therapies at lower doses
  • Exhibit selective tropism for neurons, astrocytes and/or oligodendrocytes

  • We believe that our approach will allow us to rapidly identify new capsids to drive new product candidates for CNS disorders with novel biodistribution and transduction profiles into our development pipeline.

Zegen Platform Technology | Zegen Therapeutics

Manufacturing Collaborators

Our high-performing manufacturing collaborators, focuses on quality at scale. Our team’s in-depth experience in AAV biology, clinical expertise, process development and associated analytics has allowed us to establish our high performing manufacturing platform rapidly to support the pipeline. We have an ongoing and ambitious programs of development to continuously improve quality and productivity. This includes extensive pilot-scale laboratories, covering all aspects of process and analytical development, and our own GMP manufacturing facility, which we are establishing alongside our R&D labs to ensure we can supply clinical-grade products rapidly.