FULL PROGRAM TO COME....

Join us for a conversation about treatments for Angelman syndrome. There are a number of exciting and different gene therapy approaches being undertaken to treat Angelman syndrome as well as current and proposed clinical trials aimed at alleviating symptoms (downstream therapeutics). With Agilis Biotherpauetics announcing gene therapy clinical trials as early as next year, now is the perfect time to empower yourself and become fully aware on different avenues for treatements and potential trials as they become available.

We will also talk about Australian specific logistics for Clinical trials, drug approval timelines, PBS and how what is being done to advocate for rare disease policy in Australia.

Gene therapy approaches -

**Antisense oligonucleotides (ASO) - Roche Pharma Research and Early Development – Meghan Millar PhD, Discovery Project Leader, Rare Diseases.

**Artificial Transcription Factors/Zinc Fingers (ATF) - Professor David Segal from UC Davis/FAST FIRE team

**Adeno-associated virus (AAV) - Agilis Biotherapeutics, Dr Jennifer Daily, the Director of Research

**Professor Edwin Weeber, University of South Florida & the head of the FAST FIRE team 
FAST Australia Chief Science Officer Chloe Simons

 

Downstream therapies -

** Donna Herber PhD, Disruptive Nutrition to talk about the clinical trial they announced in December for a potential downstream therapeutic using ketones and also about some of the new outcome measures being adapted specifically for Angelman syndrome.

 

Legislation and logistics - 

 

Tina Soulis from Neuroscience Trials Australia will talk about legislation in Australia including the Therapeutic goods administration (TGA), timelines and where to once trials have been held in other countries.

 

More speakers and full program to be announced.

 


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