EMPOWER & INSPIRE
2ND ANNUAL LEIGH SYNDROME SYMPOSIUM
Thank you to 240 attendees who joined us on September 19, 2023. Live language translation that we have provided was used to translate the symposium to 13 different languages. Read conference report HERE.
Brought to you by:
Cure Mito Foundation
&
integrative Cardiovascular Metabolism and Pathophysiology Laboratory (iCaMP) at Boston University
AGENDA
Please note that the times provided below are in Eastern Standard Time (EST). To convert these times to your local time zone, click HERE to use the time zone converter.
Time (EST)
Title
Speaker
Description
9 - 9:10 am
Introduction
Kasey Woleben
Cure Mito Co-founder
9:10 - 10:00 am
Gene Therapy for Neurological Disorders
Steven Grey, PhD
Associate Professor, University of Texas Southwestern Medical Center
Dr. Gray will describe the current state-of-the art for using AAV vectors to deliver genes to the nervous system. He’ll discuss examples where this has been used successfully, and also challenges and limitations of the technology.
Followed by 10 min Q&A
Kasey Woleben to moderate Q&A with Dr. Gray
10 - 10:15 am
Running for a Cure
Greg MacCurtain
Parent
Abby was diagnosed with Leigh’s disease at nine months of age and given a life expectancy of two years. Abby, who is now 12, and her dad Greg have become a fixture in local road races around Massachusetts including several Boston Marathons.
RESEARCH
Moderator: Jessica Fetterman, PhD
Assistant Professor, Boston University School of Medicine
10 - 10:15 am
10:15 - 10:30 am
Developing diagnostic resources for Leigh syndrome
Shamima Rahman, FRCP FRCPCH PhD
Professor of Pediatric Metabolic Medicine
Mitochondrial Research Group
Genetics and Genomic Medicine
UCL Great Ormond Street Institute of Child Health
10 - 10:15 am
10:30 - 10:45 am
Pluripotent stem cells and brain organoids for Leigh syndrome
Alessandro Prigione, MD, PhD
Professor, University of Düsseldorf, Germany
In this talk, Dr. Prigione will summarize his work to apply pluripotent stem cells and brain organoids as a platform for studying Leigh syndrome and for identifying potential treatment strategies.
10 - 10:15 am
10:45 - 11:00 am
Towards therapeutic editing of mitochondrial genome
Michal Minczuk, PhD
Group Leader, University of Cambridge
Co-founder, Pretzel Therapeutics
In this talk, Professor Minczuk will discuss his team’s work on the genetic modification of mammalian mtDNA, in particular, the progress towards using genome editing technologies, such as programmable nucleases and base editors, for the treatment of hereditary mitochondrial disease.
10 - 10:15 am
11 - 11:15 am
The immunological origins of Leigh syndrome
Allison Hanaford, PhD
Postdoctoral Research Fellow | Center for Integrative Brain Research
Seattle Children's Research Institute
This talk presents laboratory findings on the immune system's role in Leigh syndrome using an Ndufs4 mouse model. Researchers discovered that treating mice with a drug that removes specific immune cells called macrophages (which are a type of white blood cell), prevents the disease. They also discovered that some of these immune cells travel to the brain, and a protein called interferon gamma plays a role in Leigh syndrome. These findings offer potential avenues for immune system-based treatments for Leigh syndrome.
10 - 10:15 am
11:15 - 11:30 am
Gene therapy development for SURF1-related Leigh syndrome
Qinglan Ling, PhD
Postdoctoral Researcher, University of Texas Southwestern Medical Center
The talk will focus on pre-clinical studies for developing a translatable AAV-based gene therapy approach for SURF1-related Leigh syndrome patients.
10 - 10:15 am
11:30 - 11:45 am
Yeast-powered Drug Repurposing for Leigh Syndrome
Ethan Perlstein, PhD
Founder/CEO Perlara PBC Cofounder/CEO Maggie's Pearl
Dr. Perlstein will talk about SURF1 yeast project, the rescue mechanisms that were discovered through our collaboration with Perlara, and the path forward toward 1-to-N clinical trials.
10 - 10:15 am
11:45 - 12:00 pm
Q&A with the speakers
Moderator: Jessica Fetterman, PhD
Assistant Professor, Boston University School of Medicine
Followed by 15 minutes break
Moderator: Jessica Fetterman, PhD
Assistant Professor, Boston University School of Medicine
clinical care
12:15 - 12:30 pm
Preventative Care and Medical Monitoring in Leigh Syndrome
Sumit Parikh, MD
Director, Mitochondrial Medicine Center, Cleveland Clinic
Associate Professor of Neurology
Mitochondrial diseases unfortunately impact many parts of the body outside of the brain. Active surveillance and routine monitoring of a person's health allows for the medical team to optimize care for individuals with Leigh Syndrome. In his talk, Dr. Sumit Parikh will discuss recommendations that are based on international guidelines developed by Mitochondrial Medicine Society in 2017 and utilized at all national Mitochondrial Care Network sites.
12:30 - 12:45 pm
Developmental and rehabilitation therapies in mitochondrial disease: PT, OT, Speech and beyond
Ibrahim Elsharkawi, MD
Icahn School of Medicine at Mt. Sinai
The goal of this talk is to share insights into how and why different therapies impact and benefit patients with mitochondrial disease, including physical therapy, occupational therapy, speech therapy, and to touch on the role of physical medicine and rehabilitation physicians.
12:45 - 1 pm
Mito and Cannabis, what's the hype all about?
Sebastien Cotte
Co Founder, Georgia's Hope
National Education Director, Flowering Hope Foundation
In this presentation, we will take a look at using medical cannabis for mito, can it really help? Should you consider it? How to get started? and more.
1 - 1:15 pm
Q&A with the speakers
Moderator: Jessica Fetterman, PhD
Assistant Professor, Boston University School of Medicine
PATIENT REGISTRY AND DATA PANEL
Moderator: Danielle Boyce, MPH, DPA
Assistant Professor, Johns Hopkins University School of Medicine
Cure Mito Scientific & Medical Advisory Member
Leigh syndrome patient registry updates
Sophia Zilber, Cure Mito Foundation
Interoperability of patient registry data with regulatory standards
Pallavi Bakare, MS, Sumptuous Data Sciences
Accelerating Mitochondrial Drug Development through a Public Private Partnership
Amanda Klein, PharmD CDCES, C-path
1:15 - 1:30 pm
Q&A with the speakers
Moderator: Danielle Boyce, MPH, DPA
Assistant Professor, Johns Hopkins University School of Medicine
Cure Mito Scientific & Medical Advisory Member
1:30 - 1:45 pm
Industry panel
Moderator: Sophia Zilber
1:45 - 2 pm
Industry Updates
Magnus Hansson, MD, PhD, Chief Medical Officer, VP Preclinical & Clinical Development, Abliva
Dave Penake, CEO, Saol Therapeutics
Zhu Tong, PhD, FCP, VP, Primary Focus Lead, Mitochondria
Astellas Pharma Global Development, Inc.
Janice Keramedjian, Executive Vice President, MytoLyfe Biotherapeutics. Inc.
Minyoung So, PhD, Principal Scientist, Standigm Inc.
2 - 2:15 pm
Q&A with the speakers
Moderator: Sophia Zilber
2:15 - 2: 30 pm
Conclusion
Kasey Woleben, Jessica Fetterman, PhD
CONFIRMED SPEAKERS
Steven Gray, PhD
Keynote Speaker
"Gene Therapy for Neurological Disorders"
Associate Professor, University of Texas Southwestern Medical Center
Shamima Rahman,
FRCP FRCPCH PhD
Professor of Pediatric Metabolic Medicine
Mitochondrial Research Group
Genetics and Genomic Medicine
UCL Great Ormond Street Institute of Child Health
Alessandro Prigione, MD, PhD
Professor, University of Düsseldorf, Germany
Michal Minczuk, PhD
Group Leader, University of Cambridge
Co-founder, Pretzel Therapeutics
Allison Hanaford, PhD
Postdoctoral Research Fellow | Center for Integrative Brain Research
Seattle Children's Research Institute
Sumit Parikh, MD
Director, Mitochondrial Medicine Center, Cleveland Clinic
Associate Professor of Neurology
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Qinglan Ling, PhD
Postdoctoral Researcher, University of Texas Southwestern Medical Center
Ethan Perlstein, PhD
Founder/CEO Perlara PBC
Cofounder/CEO Maggie's Pearl
Dave Penake
CEO, Saol Therapeutics
Sebastien Cotte
Co Founder, Georgia's Hope
National Education Director, Flowering Hope Foundation
Greg MacCurtain
Parent
Magnus Hansson, MD,
PhD
Chief Medical Officer
VP Preclinical & Clinical Development
Abliva
Kasey Woleben
Cure Mito Foundation
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Sophia Zilber
Cure Mito Foundation
Ibrahim Elsharkawi, MD
Icahn School of Medicine at Mt. Sinai
Pallavi Bakare, MS
Sumptuous Data Sciences
Amanda Klein, PharmD CDCES
Critical Path Institute
Tong Zhu, PhD, FCP
Astellas Pharma, Inc.
Janice Keramedjian
MytoLyfe Therapeutics. Inc.
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Minyoung So, PhD
Standigm Inc.
THANK YOU TO OUR SPONSORS
THANK YOU TO OUR media partners
To view conference 2022 agenda and recordings, please click HERE
The following sponsorship levels are available:
Inspiration sponsorship $1000:
Logo on the conference website
Recognition in conference communications and social media
Empowerment sponsorship $3000:
All benefits of Inspiration sponsorship
Separate thank you social media post (Facebook, LinkedIn, Instagram, Twitter)
Hope sponsorship $5000:
All benefits of Empowerment sponsorship
Sponsor's materials (such as PDF) can be downloaded from conference website
Acknowledgement during conference opening and closing sessions.
Customized sponsorship
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