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Interview with Dr Allyson Berent, Chief Scientific Officer at the Foundation for Angelman Syndrome Therapeutics

20 August 2020   |  
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Following on from our recent announcement that we would be working with the Foundation for Angelman Syndrome Therapeutics (FAST) to find new treatments for Angelman Syndrome, we interviewed Dr Allyson Berent, Chief Scientific Officer at FAST, to learn more about the organisation and the ways in which AI is being used to speed up drug discovery for rare diseases. 

Hey Allyson! Thanks for chatting. Can you tell us a bit more about FAST and its work?

FAST (Foundation for Angelman Syndrome Therapeutics) is a Section 501(c)(3) nonprofit research organization singularly focused on funding research that holds the greatest promise of treating the symptoms of Angelman syndrome and allowing those living with the condition to realize their greatest potential and quality of life. FAST is the largest, non-governmental funder of Angelman-specific research in the world.

That’s a great mission. And how does your role fit into that?

I’m the Chief Science Officer for FAST. I am responsible for evaluating and exploring all of the potential therapeutic treatment options that could benefit those living with Angelman syndrome and ensure that FAST is supporting the best and brightest minds to guarantee all options are thoroughly and carefully considered. This is in collaboration with a world renowned Scientific Advisory Board and numerous Scientific Advisors.  In addition, I help to focus the vision for FAST to ensure that all needs for clinical trial readiness, and engagement with all pharmaceutical sponsors, are optimized.

For those who don’t know much about Angelman Syndrome, what would you say are the biggest challenges facing patients and families living with the condition? 

One of the biggest challenges is the fact that individuals living with Angelman Syndrome are universally non-verbal, making it very difficult to be heard and understood. This creates so many hardships in their lives. In addition, individuals living with the condition can have a significant seizure disorder, sleep disturbances, mobility issues and developmental delay. This is all tremendously challenging for both the individual and their family.  

What’s the most important thing you’re working on right now?

The most important thing we are working on currently is to ensure that all therapeutic platforms are supported as thoroughly as possible so that all individuals living with Angelman Syndrome, regardless of age or genotype, will have the opportunity to see meaningful benefit for various different therapeutic modalities in their future. 

That’s great. Increasingly, one of the ways therapies are being developed is through the use of artificial intelligence. Why does drug discovery, particularly in rare diseases, need AI’s help? 

There has been so much work done from a basic science perspective in rare diseases and many disorders have generated a tremendous amount of information that is not easily accessible to others. By using AI, we can gather an exponential amount of information from not only Angelman Syndrome research, but also that of other similar, or mechanistically similar, disorders. This can easily broaden our understanding of drugs that have already taken a long regulatory pathway, and where they might benefit children living with Angelman Syndrome, and further de-risk therapeutic failures before wasting excessive amounts of time and money. Because we have a large infrastructure of cell lines and animal models that has been funded directly by FAST, we can assist Healx in taking their predictions based on AI and testing it in models, to further de-risk potential drug candidates. 

On that, what are you most excited about regarding the new partnership with Healx?

FAST is so excited to partner with such a thoughtful company that is able to see the complexities of not only drug development, but also Angelman syndrome, and can use scientific discoveries to predict and de-risk potentially beneficial therapeutic compounds for Angelman syndrome. Having Healx, with its incredible technology, scientific and clinical expertise, in this space, is a monumental step forward for the Angelman community. FAST is looking forward to working together on pre-clinical and clinical exploration for the benefit of those living with Angelman syndrome. This holistic approach will really fill so many scientific gaps in Angelman syndrome.

And how does FAST work with other organisations and groups?

FAST is collaborative with all organizations and pharmaceutical companies in the space. FAST has funded numerous outcome measures and biomarkers, all of which have been 100% pre-competitive and shared with everyone in the Angelman syndrome community – both academic or industry partners – to ensure that the best treatments go forward. In addition, FAST has grown globally to include a FAST Australia, FAST Canada, FAST UK, FAST Italy and FAST France. All organizations are working collaboratively in unison to meet the common goal of bringing meaningful therapeutics to all of those individuals living with Angelman Syndrome.

What’s been your proudest moment with FAST?

My proudest moment with FAST was at the 2019 FAST Scientific Summit when we had 12 pharmaceutical companies at the table supporting various future clinical trials for the potential treatment of Angelman Syndrome – 4 of which announced they would be entering clinical trials in 2020.  This was a dream come true for me. When I joined this community in late 2014 – when my daughter was initially diagnosed at only 6 months of age – there was only 1 company at the table considering a possible trial for Angelman Syndrome. We have grown so much and prepared our community, including our families, clinicians, and scientists, to be ready for this. This has been an astonishing journey to be a part of. 

That’s amazing – and great to see such progress. Where can people go if they want to get involved in your work? 

Head over to www.CureAngelman and if you want to learn more about Healx’s partnership with FAST, please click here.

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