Research
Gene Therapy for BPAN
Beat BPAN has prioritized Gene Therapy and genetic medicines as the best path forward for a medical Treatment. We are excited to announce an exciting new research project at Children’s Hospital of Philadelphia and we need your help! ​
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Rare Diseases don’t cure themselves and developing genetic therapies are a very complicated and expensive effort. Our Goal is to raise $10 Million in 2025 to fund this exciting research at CHOP and other research projects.
This research project at Chop will establish the pre-clinical research necessary to position the Clinic for BPAN and WDR45-related Disorders at Children's Hospital of Philadelphia (CHOP) for clinical trial readiness to investigate gene therapy as a therapeutic treatment for beta-propeller proteinassociated neurodegeneration (BPAN). The proposed work aims to determine whether this avenue of research might someday lead to the therapies so eagerly awaited by families affected by this condition. The work outlines a series of studies targeting three aims: development of a BPAN clinical trial protocol with biomarker validation, establishment of a gene therapy approach for BPAN, and characterization of the role of microglia in BPAN as a potential therapeutic alternative. Dr. Adang and her colleagues hope this work will be the foundation for a future clinical trial.
Laura Adang, MD, PhD
Laura Adang, MD, PhD, MSTR is an attending physician in the Division of Neurology at Children's Hospital of Philadelphia, specializing in the care of children with leukodystrophies.
Rebecca Ahrens-Nicklas, MD, PhD
Rebecca Ahrens-Nicklas, MD, PhD, is an attending physician with the Metabolic Disease Program and the Division of Human Genetics at Children’s Hospital of Philadelphia.
Beverly L. Davidson, PhD
Beverly L. Davidson, PhD, is Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at Children’s Hospital of Philadelphia.
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Areas of expertise: Neurodegenerative disease, RNA biology, Gene therapy, Animal models
CHOP Research
Gene Therapy Basics
Your Questions Answered
Research
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Beat BPAN was created by the Don’t Forget Morgan Foundation to facilitate a unique, cohesive strategy that incorporates independent scientific laboratories, Academia, and collaborations with related funding organizations to direct research for BPAN
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We are dedicated to collaboratively developing, funding, and managing basic science, animal models, preclinical, IND-enabling, translational and clinical research to improve the lives of children with BPAN. To inform our research initiatives, DFM gathers information from one-on-one meetings, broad group discussions, scientific peer-reviewed publications, conference proceedings, drug pipelines, and tailored merit reviews into the foundation’s decision-making processes. Input comes from across the Research and Development spectrum including academia, biotechnology, the pharmaceutical sector, and clinicians, caregivers, and patients.
Our research funding decisions are based on these research objectives:
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Supporting basic science and animal models to discover new mechanisms of disease, targets, and treatments
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Supporting projects which facilitate execution of clinical trials such as expanding patient natural history studies and outcome measures
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Identifying biomarkers and other innovative clinical endpoints
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Gene Therapy
Scientific Research Team
Laura Adang, MD, PhD
Laura Adang, MD, PhD, MSTR is an attending physician in the Division of Neurology at Children's Hospital of Philadelphia, specializing in the care of children with leukodystrophies.
Sami Barmada, M.D, Ph.D
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Michigan Neuroscience Institute Affiliate
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Angela Dobson Welch and Lyndon Welch Research Professor
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Associate Professor of Neurology
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Director, Michigan Brain Bank
Jason Paul Carreon Chua, M.D, PhD
Johns Hopkins Medicine
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Assistant Professor in the Department of Neurology and Division of Movement Disorders
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Primary research interests are in neurodegenerative disease with the special focus on autophagy
Send H. Cheng, PhD
Senior Vice President, Research and Product Development at Alexion, AstraZeneca RareDisease
Beverly L. Davidson, PhD
Beverly L. Davidson, PhD, is Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at Children’s Hospital of Philadelphia.
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Areas of expertise: Neurodegenerative disease, RNA biology, Gene therapy, Animal models
Yulia Grishchuk, PhD
Investigator, Asst Prof (M)
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Center for Genomic Medicine, Mass General Research Institute
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Assistant Professor of Neurology Harvard Medical School
Kathrin Meyer, PhD
Chief Scientific Officer & Head of Research and Development
Alcyone Therapeutics
Linda Narhi, PhD
Consultant with BioPhia, Adjunct Prof at UCSB
Industry expert with over 30 years at Amgen, industrial advisory board for UCLA and industry consultant
Louis-Jan Pilaz, PhD
Sanford Research
Pilaz Lab research focusing on BPAN mouse models
Afrooz Rashnonejad, MSc, PhD
Assistant Professor & Principal Investigator, Center for Gene Therapy
Abigail Wexner Research Institute at Nationwide Children’s Hospital
Department of Pediatrics, The Ohio State University College of Medicine
Jill Weimer, PhD
Chief Science Officer at Amicus Therapeutics & Senior Director of Therapeutic Development at Sanford Research
Network
Published Papers
AAV-mediated gene transfer of WDR45 corrects neurologic deficits in the mouse model of beta-propeller protein-associated neurodegeneration
Psychometric outcome measures in beta-propeller protein-associated neurodegeneration (BPAN)
Mutation in Wdr45 leads to early motor dysfunction and widespread aberrant axon terminals in a beta-propeller protein associated neurodegeneration (BPAN) patient-inspired mouse model
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