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Empower and Inspire: Leigh syndrome symposium

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.

Brought to you by:

Cure Mito Foundation

&

integrative Cardiovascular Metabolism and Pathophysiology Laboratory (iCaMP) at Boston University

AGEND

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.

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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

Dr. Steven Gray Empower and Inspire: Leigh syndrome symposium

Steven Gray, PhD

Keynote Speaker

"Gene Therapy for Neurological Disorders"

Associate Professor, University of Texas Southwestern Medical Center

Dr. Shamima Rahman Empower and Inspire: Leigh syndrome symposium

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

Dr. Alessandro Prigione Empower and Inspire: Leigh syndrome symposium

Alessandro Prigione, MD, PhD

 

Professor, University of Düsseldorf, Germany

Michal Minczuk, PhD  Empower and Inspire: Leigh syndrome symposium

Michal Minczuk, PhD

 

Group Leader, University of Cambridge

Co-founder, Pretzel Therapeutics

Allison, Hanaford, PhD  Empower and Inspire: Leigh syndrome symposium

Allison Hanaford, PhD

Postdoctoral Research Fellow | Center for Integrative Brain Research

Seattle Children's Research Institute

Sumit Parikh, MD  Empower and Inspire: Leigh syndrome symposium

Sumit Parikh, MD

Director, Mitochondrial Medicine Center, Cleveland Clinic

Associate Professor of Neurology

 

Qinglan Ling, PhD  Empower and Inspire: Leigh syndrome symposium

Qinglan Ling, PhD

Postdoctoral Researcher, University of Texas Southwestern Medical Center

 

ethan.jpg

Ethan Perlstein, PhD

Founder/CEO Perlara PBC

Cofounder/CEO Maggie's Pearl 

Dave Penake, Saol therapeutics  Empower and Inspire: Leigh syndrome symposium

Dave Penake

CEO, Saol Therapeutics

Sebastien Cotte  Empower and Inspire: Leigh syndrome symposium

Sebastien Cotte

Co Founder, Georgia's Hope

National Education Director, Flowering Hope Foundation

 

Greg MacCurtain  Empower and Inspire: Leigh syndrome symposium

Greg MacCurtain

Parent

 

Magnus Hansson, PhD Abliva Empower and Inspire: Leigh syndrome symposium

Magnus Hansson, MD,

PhD

Chief Medical Officer

VP Preclinical & Clinical Development

Abliva

 

kasey headshot.jpg

Kasey Woleben

Cure Mito Foundation

Sophia  Empower and Inspire: Leigh syndrome symposium

Sophia Zilber

Cure Mito Foundation

Ibrahim Elsharkawi, MD  Empower and Inspire: Leigh syndrome symposium

Ibrahim Elsharkawi, MD

Icahn School of Medicine at Mt. Sinai

Pallavi Bakare  Empower and Inspire: Leigh syndrome symposium

Pallavi Bakare, MS

Sumptuous Data Sciences

Amanda Klein, C-path Empower and Inspire: Leigh syndrome symposium

Amanda Klein, PharmD CDCES

Critical Path Institute

Tong Zhu, PhD  Empower and Inspire: Leigh syndrome symposium

Tong Zhu, PhD, FCP

Astellas Pharma, Inc.

janice.jpg

Janice Keramedjian

MytoLyfe Therapeutics. Inc.

Minyoung So_Profile Photo (1).jpeg

Minyoung So, PhD

Standigm Inc.

PTC therapeutics  Empower and Inspire: Leigh syndrome symposium

THANK YOU TO OUR SPONSORS

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SSIEM  Empower and Inspire: Leigh syndrome symposium

THANK YOU TO OUR media partners

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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

Please contact us at info@curemito.org

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