THE PATH TO TRIAL

1 Therapy Development

In the therapy development phase, a functional copy of the gene that causes AHC, and all of its regulatory components, are packaged into a benign virus (AAV) that has been designed to target neurons safely and efficiently. This is being done by the same leading scientists in the world specializing in AAV gene therapy vector engineering. This viral vector costs about $35,000 to produce.

2 Pre-Clinical Research

In the pre-clinical research phase, the therapy is tested in mice for safety and efficacy. Mice that carry the disease are injected with a therapeutic dose of the virus containing functional genes, and they are monitored carefully to determine the adequacy of the treatment. This is being done by the leading mouse laboratory in the world. This pre-clinical testing costs about $250,000.

4 GMP Vector Production

Good Manufacturing Practice (GMP) is a system for ensuring that products are consistently produced and controlled according to quality standards. It is designed to minimize the risks involved in any pharmaceutical production that cannot be eliminated through testing the final product. This human-grade virus vector will be produced by the same leading scientists who will develop the AAV gene therapy vector in stage 1. The cost of this production will be $1.6 million to $2.4 million.

5 Trial Design

A dedicated team of specialized AHC clinicians and a preclinical team of scientists will jointly design a clinical protocol that will stipulate clearly how the trial will be conducted, how patient follow-up will be handled, how data will be gathered, and how success will be determined.