Research
Symposium Agenda

July 22 - 23

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Agenda is subject to change and will be updated regularly.
Remote Conferencing Available: Register Here
Agenda is subject to change and will be updated regularly.

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for a collection of recorded sessions post-event.

Time

Title

Location

Presenter

7:00am
7:00am

 - 

5:00pm

Symposium Registration Desk Open

7:00am

 - 

5:00pm
600 minutes

Registration Booth A

Registration Booth A

8:15am
8:15am

 - 

9:00am

Breakfast On Your Own

8:15am

 - 

9:00am
45 minutes

Various

Various

9:00am
9:00am

 - 

11:00am

LADDER Learning Network Meeting

9:00am

 - 

11:00am
120 minutes

Kilimanjaro 1

Kilimanjaro 1

Hosted by
Angelman Syndrome Foundation & Dup15q Alliance

11:00am
11:00am

 - 

12:00pm

Break

11:00am

 - 

12:00pm
60 minutes

Various

Various

SESSION #1: Genomics of Angelman Syndrome/Duplication 15q Syndrome

11:45am
11:45am

 - 

12:05pm

Lunch & Opening Remarks

11:45am

 - 

12:05pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

12:10pm
12:10pm

 - 

12:20pm

NIH Address by Walter Koroshetz, M.D., Director of NINDS

12:10pm

 - 

12:20pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

12:25pm
12:25pm

 - 

12:45pm

The Role of UBE3A and the Additional 15q11-13 Genes in Angelman Syndrome and Duplication 15q Syndrome

12:25pm

 - 

12:45pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

12:50pm
12:50pm

 - 

1:10pm

Contributions of Non-Imprinted Genes to Angelman Syndrome and Duplication 15q Syndrome Pathophysiology

12:50pm

 - 

1:10pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

1:15pm
1:15pm

 - 

1:35pm

UBE3A Mouse Models

1:15pm

 - 

1:35pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

1:40pm
1:40pm

 - 

1:50pm

Integration of CTCF loops, Methylome and amd Transcriptome in Differentiating LUHMES

1:40pm

 - 

1:50pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

1:55pm
1:55pm

 - 

2:15pm

Empowering research through genetic disorder online portals

1:55pm

 - 

2:15pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

SESSION #2: Clinical Assessments

2:20pm
2:20pm

 - 

2:30pm

Climbing the LADDER to treatment: The impact of data sharing.

2:20pm

 - 

2:30pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

2:35pm
2:35pm

 - 

2:55pm

Early Findings from the First Deep Phenotypes Study of Infants with Angelman Syndrome

2:35pm

 - 

2:55pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

3:00pm
3:00pm

 - 

3:20pm

The Relationship Between Cortical Visual Impairment (CVI) and Communication: Preliminary Data from Two Studies

3:00pm

 - 

3:20pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

3:20pm
3:20pm

 - 

3:35pm

BREAK

3:20pm

 - 

3:35pm
15 minutes

Various

Various

SESSION #3: Therapeutic Approaches

3:35pm
3:35pm

 - 

3:55pm

Angelman Syndrome Therapeutics

3:35pm

 - 

3:55pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

4:00pm
4:00pm

 - 

4:20pm

UBE3A-Centric Approaches to Treating Angelman Syndrome and Duplication 15q syndrome

4:00pm

 - 

4:20pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

4:25pm
4:25pm

 - 

4:45pm

shRNA-Mediated Gene Therapy for the Treatment of Angelman Syndrome

4:25pm

 - 

4:45pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

4:50pm
4:50pm

 - 

5:05pm

Multi-Target AAV-Cas9 Vectors for Angelman syndrome Efficiently Disrupt Ube3a-ATS

4:50pm

 - 

5:05pm
15 minutes

Kilimanjaro 1

Kilimanjaro 1

5:10pm
5:10pm

 - 

5:20pm

A Vectorized miRNA-based Approach to Unsilence UBE3A in Angelman Syndrome

5:10pm

 - 

5:20pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

5:25pm
5:25pm

 - 

5:40pm

Defining UBE3A Pathways in Neuronal Development

5:25pm

 - 

5:40pm
15 minutes

Kilimanjaro 1

Kilimanjaro 1

5:45pm
5:45pm

 - 

5:55pm

Final Questions & Wrap Up

5:45pm

 - 

5:55pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

5:55pm
5:55pm

 - 

6:15pm

BREAK

5:55pm

 - 

6:15pm
20 minutes

Various

Various

6:15pm
6:15pm

 - 

8:00pm

Poster Session/ Scientific Reception

6:15pm

 - 

8:00pm
105 minutes

Merakesh Market

Merakesh Market

Hosted by
Angelman Syndrome Foundation & Dup15q Alliance

Time

Title

Location

Presenter

7:00am
7:00am

 - 

1:00pm

Symposium Registration Desk Open

7:00am

 - 

1:00pm
360 minutes

Registration Booth A

Registration Booth A

7:00am
7:00am

 - 

8:00am

Breakfast On Your Own

7:00am

 - 

8:00am
60 minutes

Various

Various

SESSION #4: UBE3A Function

8:00am
8:00am

 - 

8:20am

Epilepsy iPSC models

8:00am

 - 

8:20am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

8:25am
8:25am

 - 

8:35am

Inhibitory Neural Circuitry Driving Seizure Phenotypes in a Mouse Model of Angelman Syndrome

8:25am

 - 

8:35am
10 minutes

Zambezi

Zambezi

8:40am
8:40am

 - 

8:50am

White Matter Deficit and Delayed Myelination in Angelman syndrome

8:40am

 - 

8:50am
10 minutes

Kilimanjaro 1

Kilimanjaro 1

8:55am
8:55am

 - 

9:15am

Linking UBE3A Misexpression to Altered Circuit Dynamics Controlling Skilled Movements

8:55am

 - 

9:15am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

9:20am
9:20am

 - 

9:40am

Understanding the Molecular Mechanisms that Goven Synaptic Plasticity and Neuronal Function

9:20am

 - 

9:40am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

9:45am
9:45am

 - 

10:05am

UBE3A and Gut Function in Angelman syndrome Mice

9:45am

 - 

10:05am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

10:10am
10:10am

 - 

10:20am

BREAK

10:10am

 - 

10:20am
10 minutes

Various

Various

SESSION #5: ASOs & Clinical Trial Updates

10:20am
10:20am

 - 

10:40am

Kicho: A Parent-led Drug Discovery Program

10:20am

 - 

10:40am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

10:45am
10:45am

 - 

10:55am

Boosting UBE3A Expression with Antisense Oligonucleotides Targeting 5’UTR Splicing

10:45am

 - 

10:55am
10 minutes

Kilimanjaro 1

Kilimanjaro 1

11:00am
11:00am

 - 

11:15am

PACIFIC Trial Update

11:00am

 - 

11:15am
15 minutes

Kilimanjaro 1

Kilimanjaro 1

11:20am
11:20am

 - 

11:40am

Results from TANGELO, a Phase 1 Open Label Multicenter Study of Rugonersen

11:20am

 - 

11:40am
20 minutes

Kilimanjaro 1

Kilimanjaro 1

11:45am
11:45am

 - 

11:55am

Results from the prematurely terminated Phase 2 trial of basmisanil in children with Dup15q syndrome (Quindecim study)

11:45am

 - 

11:55am
10 minutes

Kilimanjaro 1

Kilimanjaro 1

11:55am
11:55am

 - 

12:20pm

Break for a Boxed Lunch

11:55am

 - 

12:20pm
25 minutes

Kilimanjaro 1

Kilimanjaro 1

SESSION #6: Newborn Screening and Clinical Features

12:20pm
12:20pm

 - 

12:40pm

Long Read Sequencing for Angelman Syndrome in Newborn Screening

12:20pm

 - 

12:40pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

12:45pm
12:45pm

 - 

1:05pm

In Utero Antisense Oligonucleotide Administration in Mice

12:45pm

 - 

1:05pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

1:10pm
1:10pm

 - 

1:20pm

Assessment of a Screening Assay for NBS for Angelman syndrome, Prader-Willi Syndrome, and Duplication 15q syndrome

1:10pm

 - 

1:20pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

1:25pm
1:25pm

 - 

1:35pm

High Frequency Ultrasonic Vocalizations are Disrupted in a Mouse Model of Angelman Syndrome

1:25pm

 - 

1:35pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

1:40pm
1:40pm

 - 

1:50pm

Duplication 15q Syndrome: A Parental Genetic Education Needs Assessment

1:40pm

 - 

1:50pm
10 minutes

Kilimanjaro 1

Kilimanjaro 1

1:55pm
1:55pm

 - 

2:15pm

The Tale of two Therapies for Angelman syndrome

1:55pm

 - 

2:15pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

2:15pm
2:15pm

 - 

2:25pm

BREAK

2:15pm

 - 

2:25pm
10 minutes

Various

Various

SESSION #7: Outcome Measures

2:25pm
2:25pm

 - 

2:45pm

Improving Administration of Critical Outcome Measures in Clinical Trials

2:25pm

 - 

2:45pm
20 minutes

Kilimanjaro 1

Kilimanjaro 1

2:50pm
2:50pm

 - 

3:00pm

C-BiLLT in Angelman Syndrome