Dear Roger, 

We hope this message finds you well. 

As members of the Australian CTNNB1 Community, whether family, friends, or affected people, we are reaching out to urge you to consider our plea for equitable access to the gene augmentation therapy developed by CMRI. 

We understand that CMRI is in the process of granting an exclusive licence to the Slovenian CTNNB1 foundation for a clinical trial in Slovenia. This decision, if unchanged, would limit treatment access for non-European affected individuals, including those within the Australian community who currently have no alternative for their incurable condition, as you well know. 

While we appreciate the work done by the CTNNB1 Foundation in Slovenia to progress the program, we would like to underscore that the therapeutic approach is inspired by Leszek and Andrea's ground-breaking work, which has been supported by the Australian Government and Australian people via CMRI.

We do not wish to see the proposed clinical trial be limited in effect to patients in Europe. We also would like to underscore that we have no intention of preventing the current agreement to take place; we simply wish to ensure additional doses may be funded and distributed within Australia, in any shape you feel is adequate. 

As you are no doubt aware, the founder of Little Miracles for Kids non-for-profit charity, has taken proactive steps to collaborate with CMRI and the Slovenian foundation. Thanks to Little Miracles for Kids we have the sufficient funds to produce an additional batch of doses equivalent in number to those planned for use in Slovenia. We believe the whole CTNNB1 community would benefit from this approach, as it would double the number of patients accessing the proposed therapy, thus allowing a more robust investigation of the therapy’s effects on the condition both from a medical but also a scientific standpoint.  

While we initially hoped to use these within the European trial, it is becoming obvious that the logistics makes it impossible for Australian patients to receive treatment there. With the support of CMRI, it should be possible to develop and distribute these additional doses within Australia.

In light of the urgency and the potential brighter future this treatment could offer CTNNB1 patients, we kindly urge CMRI to reconsider the licensing agreement. We once again acknowledge and appreciate the dedication of your researchers in Australia.  

We, the undersigned, believe in the collaborative efforts with the CTNNB1 foundation in Slovenia and request that access to the potential treatment be not limited by the current agreement. 

Regards, 
Auatralian CTNNB1 families, friends, and patients