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.

Researchers and Clinicians

Monkol Lek, PhD
Yale Medical School
  • Monkol received an undergraduate degree in Engineering (Computer Engineering) in 2000 at the University of New South Wales (UNSW) and then worked for IBM for 3.5 years. He returned to UNSW and completed undergraduate degrees in Science (Physiology) and Engineering (Bioinformatics) and received the University Medal in 2007. He completed his PhD (Medicine) at the University of Sydney in 2012 with the thesis topic: Functional differences between alpha-actinin-2 and alpha-actinin-3. Monkol did his post-doctoral training in Daniel MacArthur’s lab based at Massachusetts General Hospital, Harvard Medical School and the Broad Institute.
  • ​He was the lead author/analyst for the Exome Aggregation Consortium (ExAC) project that was published in Nature 2016. He went on to lead the NIH funded Broad Center for Mendelian Genomics (CMG) analysis team. As lead analyst, he oversaw the analysis strategy for all major CMG collaborations and organized monthly meetings to foster sharing of new methods and analysis amongst the rare disease community. He also coordinated the data processing and preliminary analysis of NIH Gabriella Miller Kids First (GMKF) cohorts sequenced or reprocessed at the Broad Institute.
  • ​Monkol has a strong passion for rare muscle disease research as a patient with Limb Girdle Muscular dystrophy (LGMD2G). During his time in the Broad Institute, he led the exome/genome analysis of MYOSEQ (European cohort of >1000 LGMD patients) and SeqNMD (an international consortium of undiagnosed rare muscle diseases) which has resulted in novel disease gene discovery.
Chris Dowdy, PhD
Charles River Labs

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.

Diane Balderson, PhD
Regulatory Innovation LLC

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.

Lauren E. Black, PhD
Charles River Labs

An ex-FDA reviewer, Lauren Black is now a Distinguished Scientist at Charles River with more than 25 years of experience in drug development, focusing on accelerated translation to Phase I/II clinical trials.

Kaiyue Ma, MPhil
Yale Medical School
  • Kaiyue received a Bachelor of Science degree in Biological Sciences in 2018 at the University of Science and Technology of China (USTC). He completed his undergraduate thesis study at Ludwig Institute for Cancer Research at the University of California, San Diego (UCSD). He is currently a PhD student in Dr. Monkol Lek’s lab in the Genetics Department at Yale University. He has been admitted to candidacy and awarded a Master of Philosophy degree.
  • Kaiyue’s PhD thesis topic is Improving the Interpretation of Rare Variants in the Context of the Dystrophin Glycoprotein Complex. In addition, Kaiyue has a great interest in applying novel techniques to developing treatments for diseases.
Marjorie Ferguson
Charles River Labs
Shushu Huang, MD
Yale Medical School‍
  • Shushu is currently an Associate Research Scientist in the Genetics Department at Yale School of Medicine. She received her MD at Nanjing Medical University, China, and then worked as a physician for 4 years in China. During her clinical work, she developed a strong passion for rare disease research, especially discovery of novel disease genes. 
  • Since joining Dr. Monkol Lek’s lab in August 2018, Shushu has mastered skills in whole exome sequencing (WES) analysis and helped lots of patients harboring rare genetic mutations to find their pathogenic variants. 
  • In addition to her work with NGS analysis, Shushu also dedicates herself to individualized mutation correction using CRISPR/Cas9 on Duchenne Muscular Dystrophy (DMD). She hopes there would be one day she can use the techniques she has developed to not only diagnose but also cure DMD patients.
Keith Sutton, PhD
Charles River Labs

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.

Robert H. Brown, MD
UMass Chan Medical School

Robert H. Brown Jr., MD, DPhil, is director of the Program in Neurotherapeutics at the University of Massachusetts Medical School. Prior to joining the Medical School in May 2008, Dr. Brown was professor of neurology at Harvard Medical School and a neurologist at Massachusetts General Hospital. 

Dr. Brown is an internationally known researcher and clinician leading the quest to cure neurodegenerative and neuromuscular diseases like amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Dr. Brown founded the Day Neuromuscular Laboratory in 1984 to investigate neuromuscular diseases and continues his work with the Day Lab today at the University of Massachusetts Medical School. In 1993, a team of researchers led by Dr. Brown discovered the first gene linked to the inherited form of ALS, a protein antioxidant known as superoxide dismutase, or SOD1.

In addition to his work with ALS, Dr. Brown has also identified disease genes in other inherited neurodegenerative and neuromuscular diseases such as hyperkalemic periodic paralysis, Miyoshi muscular dystrophy and hereditary sensory neuropathy.

Dr. Brown is well known for his work with patient advocacy groups and charitable organizations, such as Project ALS and ALS Therapy. He is also a founding member of the Northeast ALS Study Consortium and is president of the ALS Therapy Alliance's Board of Directors. 

Board Of Directors

Rich Horgan, MBA

Richard Horgan is the Founder and President of Cure Rare Disease. He has a deep passion for reimagining how rare and ultra-rare diseases are treated. With a younger sibling impacted by a rare disease, Rich has a strong interest in accelerating promising treatments for rare diseases. He formed an interdisciplinary collaboration of world-class researchers and clinicians to pioneer the rapid development of customized therapies for 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 also launched a successful car washing business in New York. 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 Business 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.

Melissa Hunter-Ensor, PhD, JD

Dr. Melissa Hunter-Ensor is a board member of Cure Rare Disease. She is also the Co-chair of Greenberg Traurig’s Global Patent Prosecution Group. Melissa focuses her practice on the intellectual property and commercial needs of clients in the life science industry, encompassing pharmaceutical, biotechnology, chemical agricultural, diagnostics and medical device companies. Melissa has experience representing clients throughout the business cycle, from start-ups and universities to global pharmaceutical companies, with patent portfolio management and strategic alliances, as well as litigation and pre-litigation strategy. She also assists venture capital firms and other investors in assessing the IP risks of target investments and conducts IP patent and licensing due diligence in connection with life science technology transactions.

Before becoming a lawyer, Melissa earned a Ph.D. in neuroscience from the University of Pennsylvania and her thesis research resulted in a first author paper in Cell. She followed this accomplishment with a four-year post-doctoral fellowship at the Massachusetts Institute of Technology laboratory of Nobel laureate H. Robert Horvitz where she was a Jane Coffin Childs fellow.

Mark Smith, MBA

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.

Kwesi Acquay, MBA

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.

Stephanie Herzog

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.

Karen Morales

Karen Morales spent over 20 years in nationally recognized agencies solving Fortune 500 marketing and advertising challenges before she founded her own company, Marketing Magnet, in 2017. Marketing Magnet provides in-depth agency services focused on efficiency, fun, and fast results for businesses of all sizes.

Karen was also diagnosed with LGMD2b when she was a junior in college. Two decades later, she is a mom, survivor and advocate for those with health challenges. She is eager to bring hope and change to the disease community after spending so much of her time and personal resources on navigating the unpaved medical road of a rare disease.


Sara Cole, MBA

Sara Cole currently serves as a board member for Cure Rare Disease. She is a Business Operations Manager at Cambrian Biopharma. Prior to that, she worked as a Life Sciences Strategy Consultant at IQVIA, a large Healthcare Data Science Company and contract research organization. There she provided strategy and advisory services to guide clients in making high-level decisions in the life sciences field. She has experience working across market landscaping, opportunity assessments, R&D strategy, clinical outcomes assessments, and pricing & market access. While at IQVIA, she led major project workstreams and leveraged business experience and acumen to identify strategic alternatives and project approaches for her clients. She completed her MBA in the Health Sector Management Program at Boston University and her BS in Medicinal Chemistry from the University at Buffalo.

Jessica Curran, MA

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. 


Liz Duffy, MPA

Liz Duffy is the Director of Alumni Relations at Greenwich Country Day School (GCDS), her alma mater. She received her Bachelor’s degree from Hobart and William Smith Colleges and her Master of Public Administration from NYU. As a Director in the GCDS Development office, Liz manages fundraising efforts across more than 4,000 constituents and implements programming and events for the alumni community. Prior to GCDS, Liz worked at Tiger Foundation in New York City. As Program Officer, she conducted comprehensive research for grant renewals and new grant requests for funding areas including education, employment, youth and families, and criminal justice. She serves on the Associate Board of Waterside School, a PK-5th Grade independent school serving children and families regardless of limitations in income or circumstance. Liz lives in Greenwich, CT with her husband and two children, Quinn (6) and Ned (2). Ned was diagnosed with DMD in July 2019, three weeks before his second birthday. Liz, together with Tim, hope to bring development knowledge to Cure Rare Disease.

Greg Klassen, MS

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.
All Mr. Klassen’s three (married) children (architects and engineers) and sister (public speaker)have Spinocerebellar Ataxia, though Mr. Klassen is the only one symptomatic at this time. Mr.Klassen is pursuing a medical cure through CRD; leading the way for a cure for his family and the world.

Keven Lin

Keven Lin is the founder of Lin Capital Management, a technology-focused partnership.  Prior to that, he has launched and worked for various internet startups, and as a computational biologist at BC Genome Science Centre.  He attended University of Waterloo with a BMath in Computer Science.

Keven lives in Vancouver, BC with his wife Patricia and three children.  His son Damian was diagnosed with DMD in 2017 after the family spent six months travelling across Asia.  Keven believes in equality through education and technology, and volunteers to teach coding to a local homeschooling community.

Ali Urman, MPA

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.

Cody Tranbarger

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.

Staff

Alan H. Beggs, PhD
Scientific Director

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.

Rachel Duncan
Chief of Staff

Rachel Duncan previously served as the director of social media and digital marketing and is now Chief of Staff at Cure Rare Disease. In addition to overseeing all of Cure Rare Disease’s staff and communications, Rachel also works on general development in furthering the organization’s efforts. Rachel graduated magna cum laude from the College of Communication at Boston University. She joined the CRD team in May of 2019 and says she has been consistently inspired by the incredible work it does every day. She’s both honored and proud to be part of something so special to her. Say hi to Rachel by DMing Cure Rare Disease on any of our social media accounts!

Sarah Krieger
Director of Community Engagement

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 Kuhl
Development Specialist

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.