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.
Chris oversees a team of project managers and provides scientific support for the Genetically Engineered Model Services Group. He also leads CRISPR-based model creation services in North America. Chris is a molecular biologist with experience ranging from generating and characterizing animal models to large-scale genotyping and sequencing. He holds BS degrees in biology and literature and received his PhD in biomedical science.
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.
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.
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. 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.
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.
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
Kwesi Acquay is an associate at J.P. Morgan in the Technology Investment Banking Group. He is the founder of the J.P. Morgan Digital Initiative: the first initiative dedicated to digital ecosystems and startup engagement across NY & Boston Investment Banking practices.
In addition to Cure Rare Disease, Kwesi serves on the Board of StEP, an education technology startup in Worcester, MA.
Kwesi graduated magna cum laude with a B.S. in Applied Economics and Management from Cornell University in 2014. He is the recipient of the Richard A. Church ’64 Senior Service Award, awarded by the CALS Alumni Association in recognition of leadership and service to Cornell University. He serves on the Advisory Council for Entrepreneurship at Cornell.
Kwesi remains passionate about innovation and how it can make a difference in the community.
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 and ran a product development company (selling to the US intel agencies)- Genesis Engineering which he sold to Hewlett Packard. He started and ran a Digital Signal Processing company (primary customer: Radio Shack’s - police scanner division - called Applied Signal Processing); started ran and sold a government services company (SigInt Technologies) which he sold to Novetta (now Accenture); then he started and ran a highly successful real estate business (called Global Property Holdings), before “retiring” in 2021.
Mr. Klassen has a BS and MS from VA Tech, as well as graduate work toward an MBA at Maryland. 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.
Cody Tranbarger is currently an Analyst at Casdin Capital, a life sciences-focused investment firm. In his role, Cody leads coverage of Genetic Medicines, an investing effort focused on next-generation therapeutic technologies, such as cell and gene therapies. Previously, Cody was 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.
Alexandra “Ali” is currently an Analyst at ARK Invest on the Genomic Revolution strategy. 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 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 published over 25 articles spanning cancer research, AI and space medicine. She has four 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 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 APSG conference, a guest lecturer at New York University and is an Industry Advisor for the Sanford School of Medicine.
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.
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.
Marino has over 15 years’ experience leading fast-growing nonprofits that focus on using coaching to decrease racial disparities in educational outcomes. Having been an Executive Director at Summer Search Boston and Chief of Growth and Strategy at Duet during times of significant growth she has deep experience with the many challenges leaders face. She values meaningful relationships, a regular practice of self-reflection, honest feedback, and leaders who are ambitious in their professional and personal lives. She graduated from Boston College and has her master’s degree in teaching.
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.