Our Team
Cure Rare Disease is realizing the potential of life-saving therapeutics through a collaboration of the world's leading academics, clinicians, regulatory experts, translational experts and manufacturing experts.
Cure Rare Disease is realizing the potential of life-saving therapeutics through a collaboration of the world's leading academics, clinicians, regulatory experts, translational experts and manufacturing experts.
Regulatory professional providing consulting services to small and large pharmaceutical companies in strategic global pharmaceutical development. Over 30 years of experience in multiple therapeutic areas and all stages of development with a background in regulatory, clinical and nonclinical disciplines.
Diverse and extensive regulatory and clinical experience including ultra rare disease/cell and gene therapy, and involving various regulatory designations.
Alan H. Beggs PhD. joins Cure Rare Disease as our Scientific Director and is excited about the mission of developing customized therapeutics. Alan is also the Director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital and Sir Edwin & Lady Manton Professor of Pediatrics at Harvard Medical School. Following undergraduate studies at Cornell University, Dr. Beggs obtained his PhD in Human Genetics at Johns Hopkins, with subsequent postdoctoral fellowship training in medical and molecular genetics at Johns Hopkins and Boston Children’s hospitals. Throughout his career, he has used the toolset of human molecular genetics to study normal biology and disease processes in a variety of diseases with a particular focus on congenital myopathies, muscular dystrophies, and related neuromuscular diseases. In this role, and as founding director of The Manton Center, he has led the discovery of numerous new disease genes and has pioneered the development of gene therapy for one such condition, myotubular myopathy. The Manton Center provides resources and a training environment to support research on understudied “orphan diseases” through support of junior faculty with multiyear career development awards, pilot project grants, and access to the Center’s Gene Discovery Core (GDC), which provides genetic and genomic analysis services, and a rich collaborative environment for interactions between clinicians, researchers and bioinformaticians. Dr. Beggs has been a standing and ad hoc member of numerous National Institutes of Health grant committees and reviewer for the Muscular Dystrophy Association, March of Dimes and other national and international medical research foundations. He is a member of multiple scientific advisory boards, and boards of directors for both nonprofit and commercial entities.
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Gerald F. Cox, MD, PhD
Chief Medical Officer
Owner, Gerald Cox Rare Care Consulting, LLC
Staff Physician in Genetics, Part-time, Boston Children’s Hospital
Instructor in Pediatrics, Harvard Medical School
Dr. Gerry Cox is a 20-plus year veteran of the biotechnology industry, and he has been a practicing clinical geneticist at Boston Children’s Hospital for over 25 years. Dr. Cox’s introduction to drug development began at Genzyme in 2000, where as the company’s first clinical geneticist, he rose to Vice President of Clinical Development for Rare Diseases. During his 16 years at Genzyme and later at Sanofi Genzyme, he led the clinical development programs of several enzyme replacement and substrate reduction therapies for lysosomal storage disorders. He played a key role in the approvals of treatments for MPS I (Aldurazyme), MPS II (Elaprase, Japan), Gaucher disease type 1
(Cerdelga), and Gaucher disease type 3 (Cerezyme, China). He also led the early clinical trials of treatments for infantile Pompe disease (Myozyme) and Niemann-Pick disease type B (Xenpozyme), which were subsequently approved. From 2016-2018, Dr. Cox was Chief Medical Officer at Editas Medicine, a CRISPR-based genome editing company. He led the first IND for the in vivo delivery of a CRISPR-based medicine, EDIT-101, to treat Leber congenital amaurosis type 10, the
most common cause of genetic blindness in children.
Dr. Cox is a board-certified medical geneticist (clinical, biochemical, and molecular genetics) who continues to teach fellows and see patients on a part-time basis at Boston Children’s Hospital, where he was previously on the full-time staff and completed his pediatrics and genetics training. He is also an Instructor in Pediatrics at Harvard Medical School. Dr. Cox received his MD and PhD from the University of California at San Diego in 1989, and a BA in biology magna cum laude from Harvard College in 1980. He has authored over 90 peer-reviewed publications and written several book chapters and reviews. In 2019, Dr. Cox joined the board of directors of the National Tay-Sachs and Allied Diseases patient advocacy organization, and in 2023, he joined the Scientific and Medical Advisory Boards of NORD, the Barth Syndrome Foundation, and CTNNB1 Connect and Cure.
Dr. Grimm is a part of the Medical Faculty at the University of Heidelberg, teaching about viral vector technologies. He leads the Grimm Lab at the university and has also received the Outstanding Achievement Award from the Dutch Society of Gene and Cell Therapy (2020) and a Research Award from the German Duchenne Foundation (2017). Dr. Grimm has notable research interests in the creation and use of synthetic AAV vectors for human gene therapy and CRISPR/Cas systems and zinc finger nucleases.
Dr. Evgueni (Zhenya) Ivakine is a scientist working to apply genome engineering tools and strategies to therapy development for genetic conditions, with a focus on childhood neurodegenerative and neurodevelopmental disorders. While his laboratory is a recent addition to the research institute, Dr. Ivakine has been leading cutting-edge genetics research at Sickkids in the laboratory of Dr. Ronald Cohn for nearly 7 years prior to starting his own laboratory. Originally from Russia, Dr. Ivakine has completed his B.Sc. there before coming to Canada for graduate and post-doctoral studies. Dr. Ivakine has wide-ranging scientific expertise and interests and is passionate about mentorship of trainees and students of all levels. Dr. Ivakine has co-authored over 40 peer-reviewed publications and is generously funded by a number of sources including CIHR, Niemann-Pick Canada and the Blu Genes Foundation and Cure Rare Disease.
Dr. Johnson is an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University with a focus in inherited neuromuscular disorders. He received his undergraduate degree in molecular and cellular biology and psychology at the University of Arizona. He then obtained his medical degree at the University of Arizona. He completed his neurology residency and combined fellowship in neuromuscular medicine and experimental therapeutics at the University of Rochester. His laboratory is focused on identifying the pathogenesis of myotonic dystrophy, the limb girdle muscular dystrophies, and facioscapulohumeral muscular dystrophy and identifying appropriate clinical endpoints for these conditions. Johnson conducts therapeutic trials in many other inherited nerve and muscle disorders.
Marc leads Cure Rare Disease’s ASO Chemistry, Manufacturing and Controls (CMC) efforts. Marc holds a first degree in Organic Chemistry and a PhD in Biochemistry (specialising in DNA repair and cancer) from the University of Liège, Belgium, and since 1980 the “fil rouge” of Marc’s professional interests has been the study of the Nucleic Acids.
After pioneering work on Antisense and oligo delivery in Montpellier, France, followed by 2 years in the lab of Nobel Prize winner Dr. Luc Montagnier at the Pasteur Institute in Paris, Marc held positions of increasing seniority in R&D, operational management, business development, and general management within CMO’s, Pharma and Biotech companies.
He moved to the USA in 2006 in a leadership role with Glen Research, before being recruited in 2009 as CEO of Girindus America, a CMO with a focus on the cGMP manufacture of oligonucleotides for therapeutic applications. In this role, Marc also held the legal responsibility of “Sprecher des Vorstands” (President of the management board) of the German listed company Girindus AG, adding invaluable experience of leadership of a public company.
Having negotiated the merger between Nitto-Denko Avecia and the US operations of Girindus, Marc agreed to stay for a year to facilitate the integration of the newly merged entities. With that project now complete, he is working now as an independent consultant helping companies on various strategic and/or technical projects.
Willeke van Roon-Mom’s work is highly translational in nature, working in close collaboration with clinical departments and industry. The main topic of her research is autosomal dominant neurodegenerative diseases that have aberrant protein aggregation as a pathological hallmark. She studies molecular disease mechanisms, identifies biomarkers and then uses this knowledge to develop novel therapies with a focus on RNA targeting antisense oligonucleotide therapies. Her research is part of the LUMC research themes Neuroscience, Medical Genomics, Cell Tissue and Organs as well as Academic Pharma. The applicable NWA routes are regenerative medicine, personalized medicine, NeuroLabNL and health research, prevention and treatment.
Keith is a scientific advisor at our site in Edinburgh, UK. He has over 20 years of experience working in the fields of molecular biology, physiology, and immunology. Dr. Sutton also has broad experience with cell and gene therapies and designing PK and PD assessments for cell and gene therapies, mAbs, and ASOs. He has worked in commercial and academic environments across the UK, USA, and Australia.
Tracy has over 28 years of experience in Pharmaceutical Quality, Manufacturing and Regulatory. Most recently she has performed multi-product facility audits with additional pre and post-viral clearance requirements, global commercialization and submission readiness assessments, and prepared several U.S. and Europe Chemistry, Manufacturing, and Control sections in the CTD format for an adjuvanted vaccine, combination product, and biosimilar. This responsibility included the preparation of documents that spanned the course of process development, clinical and commercial-scale manufacturing for both the Drug Substance and Drug Product. This experience enabled a full understanding of the expectations for development, manufacturing, and analytical characterization of the product to obtain global product approval.
Ms. TreDenick previously directed the validation and pre-approval readiness programs for biopharmaceutical products within Pfizer (formerly Pharmacia Corporation), including the process validation, registration, and commercialization of Somavert®, a well-characterized recombinant protein product.
From 2000 to 2002, Ms. TreDenick was Manager – Global Quality and Compliance for G.D. Searle, a division of Monsanto. During this time she was involved in the quality and regulatory review of women’s health care products. Prior to joining G.D. Searle, Ms. TreDenick had roles with increasing Quality Assurance responsibility for SoloPak Pharmaceuticals and Baxter Health Care that supported drug product Aseptic filling operations.
Ms. TreDenick received her B.A. in Biology/Pre-Med from Indiana Wesleyan University. She also completed graduate study courses in Business Management at Keller Graduate School of Management.
Alan H. Beggs PhD. joins Cure Rare Disease as our Scientific Director and is excited about the mission of developing customized therapeutics. Alan is also the Director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital and Sir Edwin & Lady Manton Professor of Pediatrics at Harvard Medical School. Following undergraduate studies at Cornell University, Dr. Beggs obtained his PhD in Human Genetics at Johns Hopkins, with subsequent postdoctoral fellowship training in medical and molecular genetics at Johns Hopkins and Boston Children’s hospitals. Throughout his career, he has used the toolset of human molecular genetics to study normal biology and disease processes in a variety of diseases with a particular focus on congenital myopathies, muscular dystrophies, and related neuromuscular diseases. In this role, and as founding director of The Manton Center, he has led the discovery of numerous new disease genes and has pioneered the development of gene therapy for one such condition, myotubular myopathy. The Manton Center provides resources and a training environment to support research on understudied “orphan diseases” through support of junior faculty with multiyear career development awards, pilot project grants, and access to the Center’s Gene Discovery Core (GDC), which provides genetic and genomic analysis services, and a rich collaborative environment for interactions between clinicians, researchers and bioinformaticians. Dr. Beggs has been a standing and ad hoc member of numerous National Institutes of Health grant committees and reviewer for the Muscular Dystrophy Association, March of Dimes and other national and international medical research foundations. He is a member of multiple scientific advisory boards, and boards of directors for both nonprofit and commercial entities.
Dr. Bonnemann received his MD from Freiburg University, Germany and then completed residency in pediatric neurology at MGH/Harvard, followed by postdoctoral work with Dr. Louis Kunkel at Children's Hospital Boston working on the molecular genetics of muscular dystrophy. In 2002 he joined the Children's Hospital of Philadelphia/University of Pennsylvania as Assistant Professor, Co-Director of the Neuromuscular Program, and Director of the Neurogenetics Clinic. He joined NINDS in 2010 as Senior Investigator and Chief of the Neuromuscular and Neurogenetic Disorders of Childhood Section. Research in Dr. Bonnemann's laboratory revolves around molecular mechanisms underlying early onset muscle disease (congenital muscular dystrophies, congenital myopathies, and reducing body myopathy). The laboratory's goal is to identify the genetic and cellular mechanisms in these conditions in order to develop strategies for molecular-based treatments.
Dr. Russell Butterfield is a clinician scientist focused on translational research in neuromuscular disorders. He received his PhD in genetics, and medical degree from the University of Illinois. He completed his residency training in pediatric neurology and fellowship in neuromuscular disorders at the University of Utah. He is currently an Associate Professor in the Departments of Pediatrics and Neurology at the University of Utah where he directs the Muscular Dystrophy Clinic at the University of Utah and Neuromuscular Clinic at Primary Children’s Hospital. Dr Butterfield leads a translational research program through the Utah Program in Inherited Neuromuscular Disorders (UPIN) that includes clinical and basic research studies. Dr. Butterfield’s research is focused on implementation of genetic therapies and understanding genetic modifiers of severity in various types of muscular dystrophy including Duchenne muscular dystrophy, FSHD, and collagen VI-related muscular dystrophies.
Gerald F. Cox, MD, PhD
Chief Medical Officer
Owner, Gerald Cox Rare Care Consulting, LLC
Staff Physician in Genetics, Part-time, Boston Children’s Hospital
Instructor in Pediatrics, Harvard Medical School
Dr. Gerry Cox is a 20-plus year veteran of the biotechnology industry, and he has been a practicing clinical geneticist at Boston Children’s Hospital for over 25 years. Dr. Cox’s introduction to drug development began at Genzyme in 2000, where as the company’s first clinical geneticist, he rose to Vice President of Clinical Development for Rare Diseases. During his 16 years at Genzyme and later at Sanofi Genzyme, he led the clinical development programs of several enzyme replacement and substrate reduction therapies for lysosomal storage disorders. He played a key role in the approvals of treatments for MPS I (Aldurazyme), MPS II (Elaprase, Japan), Gaucher disease type 1
(Cerdelga), and Gaucher disease type 3 (Cerezyme, China). He also led the early clinical trials of treatments for infantile Pompe disease (Myozyme) and Niemann-Pick disease type B (Xenpozyme), which were subsequently approved. From 2016-2018, Dr. Cox was Chief Medical Officer at Editas Medicine, a CRISPR-based genome editing company. He led the first IND for the in vivo delivery of a CRISPR-based medicine, EDIT-101, to treat Leber congenital amaurosis type 10, the
most common cause of genetic blindness in children.
Dr. Cox is a board-certified medical geneticist (clinical, biochemical, and molecular genetics) who continues to teach fellows and see patients on a part-time basis at Boston Children’s Hospital, where he was previously on the full-time staff and completed his pediatrics and genetics training. He is also an Instructor in Pediatrics at Harvard Medical School. Dr. Cox received his MD and PhD from the University of California at San Diego in 1989, and a BA in biology magna cum laude from Harvard College in 1980. He has authored over 90 peer-reviewed publications and written several book chapters and reviews. In 2019, Dr. Cox joined the board of directors of the National Tay-Sachs and Allied Diseases patient advocacy organization, and in 2023, he joined the Scientific and Medical Advisory Boards of NORD, the Barth Syndrome Foundation, and CTNNB1 Connect and Cure.
Dr. Johnson is an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University with a focus in inherited neuromuscular disorders. He received his undergraduate degree in molecular and cellular biology and psychology at the University of Arizona. He then obtained his medical degree at the University of Arizona. He completed his neurology residency and combined fellowship in neuromuscular medicine and experimental therapeutics at the University of Rochester. His laboratory is focused on identifying the pathogenesis of myotonic dystrophy, the limb girdle muscular dystrophies, and facioscapulohumeral muscular dystrophy and identifying appropriate clinical endpoints for these conditions. Johnson conducts therapeutic trials in many other inherited nerve and muscle disorders.
• Chief Executive Officer; Diverge Translational Science Laboratory (Milwaukee, WI)
• Tenured Professor, Pathology; Children’s Hospital of Wisconsin/The Medical College of Wisconsin (Milwaukee, WI)
Dr Lawlor received his medical degree and doctorate from Loyola University School of Medicine in Maywood, Illinois (2004). Since completing his postdoctoral research training at Boston Children's Hospital in Massachusetts in 2008, Dr Lawlor has established clinical and research neuromuscular pathology laboratories at the Medical College of Wisconsin with a focus on the diagnosis and treatment of pediatric muscle disease. His research interests includepathological analyses for preclinical trials in animal models of X-linked myotubular myopathy, nemaline myopathy, Duchenne muscular dystrophy, and a variety of other skeletal muscle and cardiac disorders. Dr Lawlor is currently working with multiple industry partners in support of their gene therapy programs for genetic disorders involving skeletal muscle, heart, liver, lung, and other organ systems. He is a board-certified anatomic pathologist and neuropathologist, andhas directed two clinical laboratories that perform diagnostic services for skeletal muscle and nerve biopsies. Dr Lawlor has authored 6 book chapters and over 100 publications in peer-reviewed scientific journals. In the summer of 2022, Dr. Lawlor spun out his academic research laboratory to establish a new commercial research laboratory outside of MCW that is called Diverge Translational Science Laboratory, which is focused on continuing their work in supporting the translation of promising therapies from the preclinical through the clinical trial stages.
Dr. McNally is a physician and scientist who is keenly interested in bringing the benefits of research discovery to the practice of medicine. As a human geneticist and cardiologist, her clinical and research interests are in the genetics of cardiovascular and neuromuscular disorders. In addition to directing the Center for Genetic Medicine at Northwestern Medicine, she also leads the Cardiovascular Genetics program at the Bluhm Cardiovascular Institute. The Center for Genetic Medicine studies genetic mechanisms responsible for inherited human diseases including heart failure, cardiomyopathy, muscular dystrophy, arrhythmias, aortic aneurysms. Working with individuals and families, they are defining the genetic mutations that cause these disorders. By establishing models for these disorders, development and testing can begin for new therapies, including genetic correction and gene editing.
Francesco Muntoni is a Professor of Paediatric Neurology and the Director of the Dubowitz Neuromuscular Centre, at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. In the Institute and hospital he led the Novel Therapies Programme of the Biomedical Research Centre and between 2008 and 2022 and also the Developmental Neuroscience Programme between 2008 and 2018.
Since 2022 Muntoni is also director of the Genetic Therapy Accelerator Centre, a new gene therapy translational research partnership , based at Queen Square Institute of Neurology and in close collaboration with the UCL Great Ormond Street Institute of Child Health. This cross-faculty collaboration within UCL will drive forward an exciting evolving area with direct therapeutic benefits for patients with disabling neurological conditions.
Muntoni has an interest in pathogenesis, deep phenotyping, gene identification for rare neuromuscular conditions and translational research in Duchenne muscular dystrophy, spinal muscular atrophy and congenital myopathies. He is involved in several natural history studies and clinical trials. His research funded by the Department of Health, MRC and the European commission lead to the development and early clinical trials of 2 morpholino antisense
oligonucleotides, now approved by FDA, that induce partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy. He is the UK chief investigator for multiple clinical trials on DMD and SMA, including genetic therapies involving antisense oligonucleotides and AAV delivered transgenes.
In the last few years he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award and the 2024 Muscular Dystrophy UK President Award.
Web sites:
https://www.ucl.ac.uk/child-health/research/developmental-neurosciences/molecular-neurosciences/dubowitz-neuromuscular-centre
https://www.northstardmd.com/
http://www.smareachuk.org/
www.bindproject.eu
https://www.ucl.ac.uk/child-health/news/2022/sep/new-gene-therapy-translational-research-partnership-between-institutes-child-health
Kanneboyina Nagaraju is the Dean of the School of Pharmacy and Pharmaceutical
Sciences at Binghamton University and a SUNY Distinguished Professor of
Pharmaceutical Sciences. He joined as the founding chair of the Department of
Pharmaceutical Sciences and was one of the initial leadership members to start a new
pharmacy school in 2016. His over 28 years of academic and research tenure spans the
prestigious National Institutes of Health, Johns Hopkins University School of Medicine in
Baltimore, George Washington University School of Medicine and Health Sciences, and
Children’s National Hospital in Washington, DC. He and his colleagues are credited with
discovering and developing AGAMREE (Vamorolone), a first-in-class dissociated
glucocorticoid with a significantly reduced side effect profile. The Food and Drug
Administration and European Medicines Agency have recently approved AGAMREE.
He is an academic entrepreneur who co-founded two successful biotechnology
companies, ReveraGen BioPharma Inc and Agada Biosciences and is engaged in
discovering and developing therapies for neuromuscular diseases. Dr. Nagaraju is an
internationally recognized leader in neuromuscular diseases (NMD). He has led
international efforts to improve rigor and reproducibility by developing reliable and
sensitive outcome measures to test drug efficacy in NMD models in collaboration with
TREAT-NMD. He is described as an exceptional mentor and empathetic leader with
outstanding interpersonal skills.
Dr. Perry Shieh, MD is a Neurology Specialist in Los Angeles, CA and has over 23 years of experience in the medical field. He graduated from Johns Hopkins University School Of Medicine in 2000. He is affiliated with Ronald Reagan UCLA Medical Center and specializes in Neurology and Physical Medicine & Rehabilitation. Dr. Shieh has a research focus on neuromuscular diseases, specifically spinal muscular atrophy, limb-girdle muscular dystrophy, and the muscular dystrophies as a whole
Nicolas Wein, is a PhD who received his doctorate degree in Molecular Biology in the laboratory of Nicolas Levy, MD, PhD, at the Marseille Medical School in France. He worked on new diagnosis tools and therapy for dysferlinopathies. In 2011, he did a postdoctoral position at the Center for Gene Therapy, in columbus, Ohio, USA, where he developed a therapy for a subpopulation of Duchenne Muscular Dystrophy patients (DMD; NCT04240314). In 2016, he opened his own laboratory as an independent investigator and became an associate professor in 2023. His laboratory studies the mechanisms of neuromuscular disorders and the development of treatment for these diseases with a primary focus on DMD. Dr. Wein recently joined Cure Rare Disease organization independently as a consultant to provide scientific guidance to help developing treatment for patient affected by neuromuscular disorders. His experiences in exon skipping and gene therapy will help CRD missions and goals.
Dr. Kuo is an Associate Professor of Neurology at Columbia University, and he is also a physician-scientist specializing in Movement Disorders. He is also the H. Houston Merritt endowed Associate Professor, a title held by Dr. Stanley Fahn for many years, and he succeeded the legacy. There is also an interesting story about this endowed position, as Dr. Houston Merritt is the funding father of modern neurology who discovered phenytoin as an anticonvulsant. Dr. Kuo has published extensively in the field of ataxia and tremor, including over 170 papers, 5 book chapters, and 1 book. He serves as the Chair for the Movement Disorders Section at the American Neurological Association and also a Scientific Advisor for the National Ataxia Foundation and International Essential Tremor Foundation. He is the Director of the Initiative for Columbia Ataxia and Tremor, an organization that brings together physicians, scientists, and engineers to advance therapies for cerebellar disorders. His lab uses multi-disciplinary approaches, ranging from the postmortem human brain, animal models, optogenetics, in vivo recording and human physiological recordings, to comprehensively
study the cerebellar circuit with the goal to finding ways to restore circuit dysfunction to reduce disabling movement disorders symptoms.
Huw Morris is Professor of Neurology and Neurogenetics, at UCL Queen Square Institute of Neurology, and Honorary Consultant Neurologist at the Royal Free Hospital and the National Hospital, Queen Square, London. His clinical and research training took place at the National Hospita Queen Squarel, the Mayo Clinic and the Western Pacific island of Guam. He began research in PSP and related disorders as a PSP Association and MRC Clinical Research Fellow in 1997. His main interests are Neurogenetics, Movement Disorders and Dementia. He leads a UK-wide research network in PSP, Corticobasal syndrome and MSA (PROSPECT and EXPRESS) and a UK-wide research project in genetic Parkinson’s (Parkinson’s Families Project). He leads the cohort group of the Global Parkinson’s Genetics Program (gp2.org). He also studies genetic determinants of progression and variation in Parkinsonian disorders using clinical and brain bank analysis to help to define new treatments.
Bart van de Warrenburg is professor of Neurology and medical lead of the expert center for rare and genetic movement disorders at Radboudumc, Nijmegen, the Netherlands. He did a PhD on autosomal dominant cerebellar ataxias and, after his neurology residency, was a movement disorders fellow at Queen Square, London, UK. He is a former visiting professor at UKM Medical Center, Kuala Lumpur, Malaysia. As research group leader, Van de Warrenburg does translational research into rare movement disorders, with a strong focus on ataxias, and current efforts center around trial-readiness and the development and testing of therapies for these disorders. He is currently chair of the MDS Ataxia Study Group and of the Ataxia-ACT (Advisory Committee Therapeutics) for the Ataxia Global Initiative, and member of various national and international ataxia research consortia.
Kwesi Acquay is a principal at Redpoint Ventures, focused on investing in AI and enterprise software startups. Prior to Redpoint, Kwesi spent time at J.P. Morgan's tech investment banking practice where he worked on tech IPOs and M&A deals. Kwesi earned an MBA from Harvard Business School and graduated magna cum laude from Cornell University with a B.S. in Applied Economics and Management.
Jessica Curran is a first-grade teacher at Matthew Paterson Elementary School in Patterson, NY. In her twenty years of experience as an elementary school teacher Jessica has held positions on multiple elementary committees.
Jessica received her Bachelor of Arts in Elementary Education (K-6) and a Bachelor of Science in Psychology from Keene State College in Keene, NH. She also received her master’s degree in Literacy from Manhattanville College in Purchase, NY.
Jessica’s son Conner was diagnosed with Duchenne Muscular Dystrophy in 2015. From that point on she was committed to raising money and awareness for all the patients with DMD. Jessica and her husband, Christopher, founded their organization, Kindness Over Muscular Dystrophy or KOMD. They host charitable events that have raised over 1 million dollars to date. KOMD funds research, awareness and benevolence assistance to Duchenne families in need.
Jessica has been active in advocacy in her home state of Connecticut, Capitol Hill and the FDA. Working directly with Governor Ned Lamont, Jessica spearheaded a campaign to have the Governor sign a proclamation proclaiming that the state of Connecticut recognizes September 7th as World Duchenne Awareness Day. With that proclamation, Connecticut, became the second state in the nation to recognize World Duchenne Day.
On Capitol Hill, Jessica has advocated for Duchenne appropriations with her respective congressional leaders and Senators. Additionally, Jessica has testified before the FDA in a patient-focused compass meeting as part of the FDA’s Patient-focused Drug Development initiative with members of the CDC, SSA and CMS in attendance.
Jessica has been asked to speak at events led by Parent Project Muscular Dystrophy, Springboard Enterprises, Ask Bio and Pfizer. She participated in a 2020 ACA press conference with Senator Richard Blumenthal where she gave an impact statement on how the protections under the Affordable Care Act are vital for people with complex medical issues. Jessica has also been interviewed by NPR, Pix 11NY, Fox CT, News 12 CT, NBC CT, The Hartford Courant, The Ridgefield Press and The Lewisboro Ledger about her experiences raising a son with Duchenne, participating in a Duchenne gene therapy clinical trial and how the ACA impacts her family.
Stephanie Herzog currently serves as a board member for Cure Rare Disease. Stephanie is the CEO/Founder/Owner of a boutique Home & Commercial Interior Design Firm in Connecticut with proven specialties ranging from interior furnishings to complete custom space builds.
Stephanie has over 15 years experience in designing, team leading, project execution, and completion in the robust space she serves.
Stephanie’s passion for Cure Rare Disease and the research strategies for curing Genetic Disorders was born from the recent DMD diagnosis of her four year old son, Max. Throughout Stephanie’s journey she was fortunate enough to meet Rich Horgan and be introduced to the amazing ground-breaking research and work his team is doing for Muscular Dystrophy and similar rare genetic diseases.
Stephanie also serves on the Branford Arts and Culture Alliance Board in Branford, CT as well as the Board of Governors for the Pine Orchard Yacht & Country Club, chairing the Entertainment Committee.
Richard Horgan is the Founder, President and Chief Executive Officer of Cure Rare Disease. He has a deep passion for creating systems to enable and accelerate the development of promising science into new therapeutics, in honor of his late brother, Terry, who lived with an ultra-rare form of Duchenne muscular dystrophy. He formed an interdisciplinary collaboration of world-class researchers and clinicians to pioneer a novel framework to enable the development and financing of therapies for ultra-rare, genetic diseases. Prior to making his foray into biotech, Rich had extensive experience working in new business development at Corning Incorporated, where he led the successful launch of a new product line. He holds a BS from Cornell University, where he graduated summa cum laude, and an MBA from Harvard Business School, where he was awarded the Blavatnik Fellowship for Life Science Entrepreneurship. Rich was recognized by Insider as one of ‘30 leaders under 40 transforming healthcare in 2020’ and, most recently, was named on the 2021 Forbes 30 under 30 list.
Mr.Klassen is a serial entrepreneur. He started, ran and sold four companies: (three software development companies) Genesis Engineering, Applied Signal Processing,, SigInt Technologies, and (areal estate company), Global Property Holdings), before “retiring” in 2021. He has served on numerous non profit boards.
Mr. Klassen has a BS and MS from VA Tech, as well as graduate work toward an MBA at Maryland’s Smith School.. Mr. Klassen enjoys mentoring, recumbent cycling (tours and day rides) and bodybuilding.
Having received a diagnosis of Spinocerebellar Ataxia that not only impacts him, but several members of his family, Mr.Klassen is pursuing a medical cure for the disease through CRD; leading the way for a cure for his family and the world.
Mark is a highly accomplished senior executive with demonstrated performance in all aspects of the energy industry, including operations, capital allocation, strategic planning, business development, corporate finance, capital markets, M&A, IPOs, turnarounds and restructuring. Mark has served on multiple executive committees and boards, and he is a Governance Fellow, National Association of Corporate Directors (NACD). Mark is recognized for his leadership in corporate strategy and development and has a strong operational track record combined with business development and corporate finance expertise. He has extensive experience in transformation and optimization of organizational design and processes. Mark is known for leading and developing high performing teams focused on creatively and collaboratively solving complex business issues.
Currently, Mark is Chief Financial Officer and Corporate Secretary of Guidon Energy, Blackstone’s largest energy - focused investment. His duties include CFO and Corporate Secretary for Blackstone affiliates, Swallowtail Royalties and Waterfield Midstream. He directs corporate finance, accounting, financial reporting, treasury, tax, internal audit, and risk management functions. Mark is a strategic partner with the CEO, executive team and Blackstone on growth and operational strategy, capital allocation, M&A, and portfolio optimization. He is an integral point of contact for Blackstone on financial matters for Guidon and Blackstone affiliates.
Prior to Guidon he served as Senior Executive Vice President and Chief Financial Officer at California Resources Corporation and served on the Executive Committee, Compliance Committee and Reserves Committee. He performed an essential role advising the CEO, executive team and Board through a challenging bankruptcy proceeding in response to dramatic drop in commodity prices and significantly lower demand.
Previously, Mark was Senior Executive Vice President and Chief Financial Officer, California Resources at Occidental Petroleum Corporation, where he was selected by this $75B enterprise value NYSE listed, international corporation to serve as “second in command” for spin-off/IPO of its California businessin a tax-free distribution to shareholders.
As Senior Vice President and Chief Financial Officer for Ultra Petroleum Corporation and chairman of its international finance subsidiary, Mark served as strategic partner with the CEO and COO for the $6B enterprise value NYSE listed company. He partnered directly with the COO on identification, evaluation, and execution of strategic M&A opportunities, and was responsible for negotiating, closing, and funding strategic acquisitions and divestitures.
Prior to that, Mark was Vice President, Business Development at J.M. Huber Energy. He was retained by this $500M asset base company to develop and lead a transactional M&A effort. Before that, Mark was Chief Financial Officer at Gulf Liquids, Inc., where he was retained as a key member of the executive management team and managed the finance function in face of the critical financial and contractual issues. Earlier in his career, Mark served as Managing Director, Investment Banking at Nesbitt Burns Securities Inc. (now BMO Capital Markets), and was appointed to the board of Nesbitt Burns Securities. He managed business development and advisory assignments in M&A, capital markets transactions and corporate finance for Texaco, Union Pacific Resources, Oryx, Pennzoil, and Anadarko among others. Prior to that, he held various positions, including Director, Energy Group at Bank of Montreal.
Mark holds an MBA, Finance (summa cum laude) from Oklahoma City University and a BS in Petroleum Engineering (Distinguished Scholar) from University of Oklahoma. He has completed leadership coursework at Harvard Business School and attended the Director’s College at Stanford Law. Mark has been recognized as CFO of the Year for a Large Public Company, San Fernando Valley Business Journal, and CFO of the Year for a Medium Public Company, Houston Business Journal. He is member and past chairman, Advisory Board, University of Oklahoma Mewbourne School of Petroleum Engineering and a member of numerous boards, including the Muscular Dystrophy Association, where he serves on the Executive Committee and is chairman of the Audit Committee.
Jeremy Sternberg is a partner in the Boston office of the global law firm Holland & Knight LLP. Jeremy’s practice focuses on working with health care companies, including life sciences companies, on complex disputes, compliance matters, and government facing issues. Prior to joining Holland & Knight, Jeremy was a federal prosecutor in the Boston U.S. Attorney’s Office. Jeremy graduated from Tufts University with a B.A. in Economics and History and from the George Washington University Law School.
Cody Tranbarger is biotech entrepreneur and investor. Cody is currently the Co-Founder of Revision Bio, a next-generating gene writing company, and holds a Consultant / Entrepreneur-in-Residence role at Atlas Venture, a biotech venture creation firm. Previously, Cody served as Analyst and Head of Genetic Medicines at Casdin Capital, where he led the firm’s investing effort focused on next-generation therapeutic technologies, such as cell and gene therapies. Cody began his career as an Investment Banking Analyst at Goldman Sachs, where he focused on the evaluation and execution of M&A and financing transactions for clients across the biotech sector. Cody graduated magna cum laude from Amherst College with a B.A. in Biology and Economics, and is an MS/MBA Biotechnology: Life Sciences candidate at Harvard Business School and Harvard Kenneth C. Griffin Graduate School of Arts and Sciences.
Ali Urman is a Genomics Analyst and investor, passionate about immunotherapy, gene editing, stem cell therapy, AI, space medicine and public health.
Ali joined ARK as an Analyst on the Genomic Revolution strategy in February 2020. She focuses on gene editing, stem cell and novel immunotherapy technologies.
Prior to ARK, Ali conducted clinical cancer research at the Memorial Sloan Kettering Cancer Center and the Montefiore Medical Center; and was a Senior Research Program Manager at IBM Watson Health. Ali incorporated her own consulting firm in 2018.
Ali holds numerous board and advisory positions, including a Board Member of Cure Rare Disease, an Industry Advisor for the Sanford School of Medicine’s SPARK program, No Patient Left Behind, Sortium, and The Innovation Foundation.
Ali has published over 25 articles spanning cancer research, AI and space medicine. She has three filed patents and received the Manager’s Choice Award in 2017 and 2018, and the Rookie of the Year Award in 2017 from IBM Watson Health.
Ali has a Bachelor of Arts from McGill University and a Masters in Epidemiology and Community Health from New York Medical College. She has additional training from the MIT Labs in Human-Computer Interaction for User Experience Design.
Ali has appeared on CNBC, Bloomberg and TD Ameritrade. She has been quoted and her research has been featured in Fortune, Business Insider, Nasdaq.com, Advisor Analyst, OPTO, ETF Trends, among other publications. She has been a featured speaker at the Forbes and APSG conferences and guest lecturer at the University of Maryland and New York University.
Klaus is a Venture Partner with OrbiMed Advisors and is focused on venture investments in the therapeutic space. Since joining OrbiMed, he has served or currently serves on the boards of numerous public and private OrbiMed portfolio companies. Previously, Klaus was the Chief Executive Officer of Schwarz Pharma Inc. with responsibility for the U.S. and Asia businesses culminating in the ultimate sale of the Schwarz Group. For seven years he was a Director of PhRMA. He received his medical degree and his doctorate (Ph.D.) from the University of Heidelberg and earned his M.B.A. at INSEAD in France.
Mr. Warner is currently President, Gene Therapy at Poseida Therapeutics leading advancements across rare diseases. Mr. Warner brings significant biopharmaceutical experience in rare diseases and gene therapy. Prior to Poseida, he was with Novartis as Vice President, Gene Therapy and Rare Disease, he was responsible for leading efforts to commercialize multiple gene therapies. His prior roles include serving as a U.S. commercial leader in Hemophilia A at BioMarin Pharmaceuticals, in a variety of strategic and operational roles at Biogen, and in commercial launch roles in blood disorders at Baxalta, now a wholly owned subsidiary of Takeda. Mr. Warner has an MBA with an emphasis in marketing and finance and a Bachelor of Business Administration degree, both from Northwood University in Michigan.
Mike Williams is the owner of Longhorn Liquor, a family-owned and operated retail chain of liquor and fine spirits stores with a focus on providing premium experiences and selections for customers. Since opening the first location in 2012, operations have grown to include 12 locations across Southeast Texas and the Greater Houston area.
In 2022, Mike and his wife, Katherine, received the diagnosis of Duchenne muscular dystrophy for their son. Since developing a partnership with Cure Rare Disease, they have launched a number of initiatives to support the organization’s work, including founding the Southeast Texas Whiskey Festival, in which 100% of the proceeds benefit Cure Rare Disease. In just two years, the annual event has become the largest whiskey and bourbon event in Texas.
Brittany Stineman is a fierce advocate for rare disease research, and a powerhouse in fundraising bringing years of expertise, passion, and visionary leadership to her role as Chief Advancement Officer at Cure Rare Disease. Known for her strategic acumen and relentless drive, Brittany has spearheaded transformative initiatives in rare disease advocacy, securing millions in funding, building high-profile partnerships, and engaging global media to rally support and awareness.
Prior to joining Cure Rare Disease, Brittany founded and led smashSMARD, a nonprofit dedicated to advancing research for Spinal Muscular Atrophy with Respiratory Distress (SMARD), a rare and devastating neuromuscular disease. After her son Nash was diagnosed, Brittany turned her pain into her purpose and her mission was clear. In this role, she catalyzed a revolutionary gene therapy project from concept to clinical trial, managing everything from high-stakes fundraising to navigating complex regulatory processes with the FDA. Her tenacity and strategic approach to advocacy led to a historic FDA approval reversal, securing emergency access for patients and pushing boundaries in ultra-rare disease research. To date, 7 patients have been treated with this gene therapy that is still in clinical trial.
Throughout her career, Brittany has cultivated key relationships with major pharmaceutical companies, academic institutions, and biotech firms to advance groundbreaking therapies. Her ability to mobilize donors, execute large-scale fundraising events, and engage with the media has earned her accolades, including being named one of Chicago's “Top 6 Most Influential Women You Need to Know” by Chicago Parent and a nomination for Global Genes’ “Rare Champions of Hope” award.
As Chief Advancement Officer, Brittany will leverage her unparalleled fundraising skills and deep commitment to rare disease patients to drive Cure Rare Disease’s mission forward. Her work will continue to focus on expanding Cure Rare Disease’s reach, securing funding for critical research, and building a robust network of supporters, ensuring life-saving advancements for rare disease patients around the world.
We are the Krieger Family from Phoenix AZ. My name is Sarah, my husband is Bryant, and we have 4 kiddos; Hans (7), Otto (6), Fritz (4), Ingrid (18mo). I am a former elementary teacher, who now homeschools our kiddos. Bryant works as a Data Analyst at Truckstop.com.
Our family loves traveling and exploring new places, even if that means our own backyard. In our spare time you can find us outdoors! We love hiking, bike riding, playing all sports, swimming, skateboarding, planting and gardening, and ultimately anything we can do together as a family.
In 2016 our son Fritz was diagnosed with Duchenne at just 10 months old. In the midst of wrestling with such news, and taking time to grieve the harsh reality that our life was going to look different from anything we could have ever imagined, our family started to realize that despite a muscle wasting disease; we can be strong, and Fritz can be strong, because STRENGTH IS MORE THAN MUSCLE. That phrase has become our battle cry, and we desire to share that message with the world.
One way we share our perspective of strength is through our small non-profit, Fritz&Friends. Over the past 2 years we have partnered with a variety of people and businesses to help raise funds for Duchenne Research, and we are honored to now do the same with Cure Rare Disease. We also own a Barbershop, called Fritz’s Barbershop that partners with our nonprofit to help give strength to Duchenne!
Before Duchenne entered our life, we had always preached to our kiddos; ‘teamwork makes the dream work.’ Years later, we are still finding truth in that idea. We could not be more excited to be an ambassador for Cure Rare Disease and join a team with our same dream. A dream that does not involve making a name for our self but collaborating with others. A dream that does not settle for the current process, but instead pushes to try new things. We dream of nothing short of a cure, and we are excited to a join a team that desires simply that! Strength is more than muscle, Strength is teamwork, strength is Cure Rare Disease.
Katie served as a development intern when she started with CRD in January of 2021 and is now the Development Specialist at Cure Rare Disease. In addition to overseeing the intern team at CRD, Katie also works on development and operational efforts to further the organization's mission. Katie is studying in the Department of Allied Health Sciences at the University of Connecticut.
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.
© CURE RARE DISEASE. ALL RIGHTS RESERVED.
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© CURE RARE DISEASE.ALL RIGHTS RESERVED.