Hi Cure,

We’re still thinking back fondly to last year’s Global Science Summit & Gala, which was a HUGE success.

Now it’s time to get ready for this year’s celebration! Keep reading for more details below…

If you have updates you would like to be included in next week’s newsletter, please email us at info@cureangelman.org.

More good news! This month we welcome a new board member to FAST: Mike Hanrahan. Mike and his wife, Jo, have been vital and beloved members of the AS community for years. We are thrilled and privileged that Mike will be bringing to FAST his peerless experience in the world of business and entrepreneurship, as well as energy for and optimism about the future for AS therapeutics. For a bit more about Mike, please read below, and help me give him a very warm welcome!

--Alana

Q: Welcome to the board, Mike! This is a new role, but you're not new to the AS community and its work. Can you tell us a bit about when you first got involved, and what forms that engagement has taken?

A: Thanks, Alana. I am very excited to be joining the board at such an exciting time for the AS community. My first, very minor involvement goes back more than 10 years at this point, when I was an enthusiastic participant in the Vivint competition on Facebook. I spent weeks cajoling family members, friends and co workers to get onto Facebook everyday, and ultimately we were successful -- which was a huge win for the community at the time. More recently, I feel very privileged to have been able to serve on the board of the FAST majority-owned Genetx Biotherapeutics company that did such amazing work in getting the GTX-102 ASO to human trials.

You can learn more about Mike by reading the full Q&A on our website:

This week we are going to continue with the focus on FAST’s 4 Pillars in the Roadmap to a Cure 2.0, highlighting Pillar 2 – Fix Dad’s Gene.

To quickly reiterate, everyone has both a mom (maternal) and dad (paternal) copy of UBE3A in the brain and the paternal copy is silenced due to an antisense transcript that binds to the dad’s copy and prevents the process required to produce UBE3A protein.

This is fine when the maternal copy can produce functional UBE3A, but in the case of Angelman syndrome where maternal UBE3A does not, a promising approach towards treating AS lies in turning on, or reactivating, paternal UBE3A. 

This Pillar 2 – Fix Dad’s Gene encompasses all work being done to reactivate the paternal UBE3A copy via antisense knockdown in the brain. 

We want to feature some of the exciting work Dr. Yong-Hui Jiang presented at the 2022 FAST Global Science Summit.

In his talk, Dr. Jiang discussed a novel CRISPR editing approach packaged in chemically engineered ribonucleoproteins to knockdown the paternal antisense transcript and reinstate paternal expression, using a mouse model of AS.

Click here to read more about Dr. Jiang’s research and watch his presentation from the Science Summit:

As mentioned above, Pillar 2 of the FAST Roadmap to a Cure 2.0 consists of therapeutic programs that focus on “turning on dad’s gene” by activating the silent paternal copy of the UBE3A gene in neurons.

Currently, we have 4 programs in the pipeline under Pillar 2:

• Antisense Oligonucleotides/Spherical Nucleic Acids (ASO/SNA)
• Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
• Artificial Transcription Factors / Zinc Fingers (AFT)
• Short-hairpin RNA/micro-RNA (shRNA)

Click here to learn more about each of these programs:

SAVE THE DATE: November 10-11, 2023 for the 16th annual FAST Global Science Summit & Gala at The Diplomat Beach Resort in Florida.

We are taking another bite of the Florida orange! We are working out the details to provide the community with a variety of ways to join the event in person and virtually. Stay tuned!

Make sure to put the dates in your calendar now!

Want to help us make this year's event even better? Email us at events@cureangelman.org.

Next month’s fireside chat is also a special episode of Genes & Juice!

Join Alana for a conversation with Nycole Copping, PhD and Dr. Allyson Berent about the latest pre-clinical work being done in gene and cell therapy advancing potential therapeutics for Angelman syndrome.

Register for the chat on February 12th at 3pm ET now:

We’re already looking forward to the next Global Science Summit & Gala in Florida this November, and we hope you’ll join us! 🌴

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