A path forward,
for one or for many.
.jpg)



































Meet the Function™️ platform
Drug discovery for thousands of genetic disorders in one unified platform.
The Function platform combines two unique data sets: large scale drug screens and genetic screens performed in neurons. The drug screen tests the effects of thousands of small molecules on gene expression. The genetic screen measures changes in expression when disease causing genes are suppressed.
The combined data sets will be used to match potentially effective drugs to rare disorder genes. Our hope is that Function provides many therapeutic candidates that can be rapidly developed for patients living with neurological diseases.
The Function platform discovers drugs that address underlying disease biology, at scale.
Prediction
Disease models
Validation
Learning

“Rarebase is pushing the limits of science and innovating a financial structure that allows patient organizations to directly support drug discovery.”

“Rarebase is stepping outside the traditional drug development pipeline and harnessing new technology and science that patient organizations like ours would never be able to utilize alone.”
We're in this together.
The latest from Rarebase
For us, it’s personal.

Onno Faber

Omid Karkouti

Chris Moxham, PhD

Hayley Brooks

Elizabeth Iorns, PhD

Nicole Perfito, PhD
.jpg)
Lynsey Chediak

Natalie Downs, MS, CGC

Amina Ann Qutub, PhD

Sabine Topka, PhD

Carrie Rich

Riley Parsons

Clayton Mellina

Arun Mahadevan, PhD

Joe Bellucci, PhD

Kayla Capper, PhD

Brittany Anderton, PhD

Devin Cavero
Alex Guin, PhD

Ursula Leone Haditsch, PhD

Mo Rahman

Michael Genin, PhD

Meg Wood

Matt Deardorff, MD PhD

Matt Might, PhD

Matt De Silva

Carolina Garcia Rizo, PhD
Onno Faber
Onno has been an entrepreneur his entire life, starting his first company in high school. After receiving his architecture degree, he started several companies while running a design-thinking company on the side. The year he moved to San Francisco with a startup in the social media space, he lost his hearing on the left due to a brain tumor. It took about a year for other medical examinations to reveal he had developed multiple tumors in his central nervous system due to a rare genetic disorder called NF2.
After being diagnosed, Onno shifted his efforts to the life science space. He dedicates his life to create new ways for rare disease R&D to take place and wants to give everyone with a rare disease the opportunity to leverage the technology for a healthy life. Watch his TedX talk here.
Omid Karkouti
Omid has spent his career as a business leader in early-stage life science companies supporting the pharmaceutical industry. Omid previously co-founded the gene therapy startup Perception Bio through the Y Combinator startup accelerator. Prior to this, Omid supported Notable Labs’s transition from cancer testing to oncology drug discovery as their VP of BD, launched the Science Exchange research outsourcing platform in six major pharma companies as VP of Sales, and led the North America go-to-market for a China-based contract research organization.
Omid’s drive to work in the rare disease field comes from his experience as a patient advocate for a family member affected by the blinding genetic disorder retinitis pigmentosa.
Chris Moxham, PhD
Chris is a drug hunter with 25 plus years of experience in large pharma and biotech. He received his PhD in molecular and cellular pharmacology and spent 20 years at Eli Lilly and Co. where he helped bring over 10 molecules into the clinic across multiple therapeutic areas and across both small and large molecule modalities. Prior to leaving Eli Lilly in 2019, Chris was Vice President of Quantitative Biology. Most recently Chris was CSO at Fulcrum Therapeutics, a clinical stage biotech focused on rare diseases, where he was responsible for the full range of drug discovery, translational science and early development activities including building and advancing the company’s preclinical portfolio through Phase 1 clinical trials. He was the scientific lead for FTX-6058 that is currently in phase 1 clinical testing for Sickle Cell Disease. Chris was instrumental in creating multiple business development partnerships and with engaging the investment community to achieve multiple rounds of financing for the company.
Hayley Brooks
Hayley has spent her career in research - coordinating clinical trials, managing operations at an IRB, leading the development of the Rare Genomes Project to scale rare disease diagnosis nationwide, supporting patient communities at 23andMe, providing capacity-building support to rare disease patient organizations at Chan Zuckerberg Initiative, and ultimately landing at Rarebase as the Associate Director of Product to help translate science into therapies for as many families as possible.
Hayley is inspired by the strength of the rare disease community and looks forward to supporting patient organizations with the same sense of urgency and determination that they bring to their quest for treatments and cures.
Elizabeth Iorns, PhD
Elizabeth started her career as a research scientist, getting a PhD in Cancer Biology at the Institute of Cancer Research in London and continuing on as a postdoctoral researcher and Assistant Professor at the University of Miami. In 2011 Elizabeth left academic research to found Science Exchange, a research outsourcing platform used by the world’s leading pharmaceutical companies. Elizabeth has since served as the CEO of Science Exchange and joined Rarebase as a Scientific Advisor in 2020.
Through her work at Science Exchange, Elizabeth has seen breakthrough technology amplify and accelerate biotech and pharmaceutical companies' development pipelines. She is most excited to help Rarebase bring these technologies to bear on therapeutic discovery for rare disorders in partnership with patient advocacy-led non-profit organizations.
Nicole Perfito, PhD
Nicole started her career as an academic scientist, getting her PhD in Biology at the University of Washington and continuing on as a postdoctoral researcher at Princeton, Berkeley, and the Max Planck Institute in Germany. She made the switch to industry about six years ago, leading a Customer Operations team at Science Exchange where she worked closely with pharma and biotech scientists and CROs to manage outsourced R&D programs.
Nicole experienced firsthand the uncertainty of diagnosis and treatment options when a family member was diagnosed with a rare disease (GPA, granulomatosis with polyangiitis). At Rarebase, she hopes to draw on her commitment to patient advocacy, her scientific training, and her operational know-how to help families and foundations generate the data and materials they need to find the fastest path to effective treatments.
Lynsey Chediak
Lynsey has spent her career at the nexus of partnership building and policy development aimed at one goal: accelerating precision medicine for people living with rare diseases. She formerly led the inaugural precision medicine portfolio at the World Economic Forum, driving forward public-private partnerships to shorten the diagnostic odyssey. She is also the former Founder and CEO of a patient advocacy non-profit where she implemented new models for increased patient support across hospital systems. Prior to that, she served as a health policy advisor in the United Kingdom's Parliament, House of Commons and California Governor's Office.
For Lynsey, fighting rare diseases is personal. She was born with a rare bone disease that initially left her with very limited physical mobility. After 15 years of exploratory treatments—the first 18 of which failed—she was able to walk upright for the first time in her life shortly before her 16th birthday. All it took was one treatment that finally worked, and her life completely transformed. Lynsey hopes every child born with a rare disease can similarly find a treatment that works to be able to live life to the fullest.
Natalie Downs, MS, CGC
Natalie is a board certified genetic counselor with specific interests in pediatric and cardiac genetics, precision medicine, and project management. After graduating from Stanford with a Master's in Human Genetics and Genetic Counseling, Natalie served the last 3 years as a clinical genetic counselor in cardiology at the University of California San Francisco. While at UCSF, she provided personalized medicine and research opportunities to patients with hereditary cardiac disorders, as well as started a pediatric cardiogenetic service.
Through her clinical work, Natalie knows all too well that even after a diagnostic odyssey ends, treatment and management of genetic conditions remain ongoing challenges for families and providers. She's excited to join Rarebase in their research efforts to help patients with rare disease thrive after diagnosis.
When she's not coordinating genetic care, Natalie is an avid kiteboarder, houseboater, and vanlifer, splitting her time between the San Francisco and Tampa Bay areas.
Amina Ann Qutub, PhD
Amina has focused her career on pioneering methods at the interface of computer science, neurovascular biology and engineering in order to understand how human cells communicate during processes of growth and repair, and use this fundamental knowledge to help eradicate hematological and neurological diseases.
Amina is an Associate Professor at the University of Texas, San Antonio (UTSA), and research thrust lead of the Artificial Intelligence MATRIX Consortium. She is also Director of the UTSA – UT Health Joint Graduate Group in Biomedical Engineering. Bridging basic science to translational impact, she directs the Quantu Project, a nationwide study to optimize brain health over a lifespan using an integration of biosensing technology, modeling and functional neurogenesis bioassays. Amina also serves as the computational lead for the international Leukemia Protein Atlases, a clinical and engineering collaboration to identify new therapeutic targets for pediatric and adult leukemias. Amina received her PhD in Bioengineering from Berkeley and UCSF, with a major in mathematical modeling and minor in neurology. She completed her postdoc as a National Institute of Health NRSA fellow in Biomedical Engineering at Johns Hopkins University, School of Medicine. Amina is an American Institute for Medical and Biological Engineering Fellow, National Academies Keck Future Initiatives Awardee and National Science Foundation CAREER Awardee.
Witnessing the effects of rare conditions on family, friends, patients and research volunteers, Amina is excited to help Rarebase accomplish their vision of applying cutting-edge computational and experimental methods to rapidly advance therapeutic discovery for rare disorders.
Sabine Topka, PhD
Sabine started her career as a research scientist, getting a PhD in Neurobiology from the University of Bonn in Germany. She moved forward with her postdoctoral studies at Memorial Sloan Kettering Cancer Center in New York. Here, at the Clinical Genetics Research lab she set up the infrastructure and pipeline to perform functional genetics research. She later took on the role of Associate Director of Laboratory Operations and Head of Functional Genomics in MSK’s Niehaus Center for Inherited Cancer Genomics where she focused on leading functional genomics studies to characterize novel candidate genes for cancer susceptibility. Following her passion for translational research, she also initiated a therapeutics discovery pipeline based on genetic biomarkers which forms the basis for a recent spin off company.
Sabine is excited to apply her scientific expertise and her dedication to helping patients through personalized medicine to support the development of novel therapies for rare genetic disorders at Rarebase.
Carrie Rich
Carrie Rich is the co-founder and CEO of The Global Good Fund, a nonprofit organization dedicated to advancing the leadership of social entrepreneurs. Carrie is also the Managing Director of the Global Impact Fund, a venture capital fund that invests in socially impactful businesses. Fundamentally, Carrie’s message is about accessibility, how everyday people can empower themselves and others. That’s why Carrie is attracted to Rarebase’s mission. Carrie’s family is also touched by rare disease; it’s personal.
Carrie serves on the Board of Directors of Trinity Health System, Cabinet Health, and the Atlas Health Foundation. She is on the Dean’s Advisory Council for the College of Health at Lehigh University. Carrie is the author of books, including Health Entrepreneurship: A Practical Guide, and two editions of Sustainability for Healthcare Management: A Leadership Imperative, which became a top 5 business book in Handelsblatt (Germany). Carrie serves as an adjunct faculty instructor and is the recipient of the EY Entrepreneur of the Year award and POLITICO Women Who Rule award.
Riley Parsons
Riley studied Bioengineering at Santa Clara University and began his career working as a data scientist at Roche Diagnostics on a system engineering team. While at Roche, he worked on building custom web applications for his team to better visualize and understand data during development of a next generation sequencing instrument. He then moved on to Ionpath, a digital pathology start-up based in Menlo Park, California. There, he worked as a full-stack software engineer developing a web-based instrument control application.
As Rarebase's first full-time software engineer, he is building software solutions to streamline rare disease research. He looks forward to leveraging his background in bioengineering and software to make a significant impact in rare disease drug discovery.
Clayton Mellina
Clayton is an engineer passionate about developing impactful applications of machine learning and computer vision. He received his BS in Symbolic Systems and MS in Computer Science from Stanford. After graduating, he spent several years in a startup and then at Yahoo where he worked on billions-scale image search and deep learning applications for Flickr. Most recently before joining Rarebase, Clayton developed industrial computer vision applications such as automated visual inspection for manufacturing at Google Cloud.
Shortly before graduating, Clayton was diagnosed with a rare genetic disorder called NF2 which causes tumors to grow from his nerves. He believes that breakthrough technologies in machine learning and high throughput biology applied in a patient-first approach will drive a revolution in human health.
Arun Mahadevan, PhD
Arun is an engineer with a passion for fundamental biological research. He earned a PhD in Bioengineering at Rice University, where he developed high-content imaging experiments to analyze the transformation of human neural stem cells into neurons. Here, he experienced first-hand the power of cell-based experiments in capturing features of normal human neural development as well as features of aberrant development in neurological disorders. He went on to complete postdoctoral training at the University of Pennsylvania with a focus on systems neuroscience approaches applied to clinical neuroimaging data.
Arun is excited to apply his broad scientific expertise in neuroscience and cell-based experimental design to aid the development of therapies for rare genetic disorders at Rarebase.
Joe Bellucci, PhD
Joe is an experienced scientist with a strong background in business and strategic development. He has played key roles in developing multiple early-stage biotechnology companies, including a functional genomics platform that was acquired by a leading pharmaceutical company developing treatments for neurodegenerative and inflammatory diseases. Joe specializes in working with cross-functional teams to apply high-throughput genetic screening to drug discovery, and has contributed to the development of small molecules, protein therapeutics, and cell and gene therapies.
Joe earned his PhD in Biomedical Engineering from Duke University, and holds a BS in Chemical and Biomolecular Engineering from Cornell University. He is excited to apply his background in business development and functional genomics to solving problems at Rarebase that will improve the lives of rare disease patients.
Kayla Capper, PhD
Kayla is a scientist with a passion for genetics, science communication, and developing customer-focused products. She enjoys the challenge of conveying complex scientific topics by simplifying them in a creative and engaging manner and strives to develop actionable products. Prior to joining Rarebase, Kayla was on the Health Product team at 23andMe where she worked on Genetic Health Risk reports, Carrier Status reports for many rare genetic diseases, as well as other health reports and product features.
Kayla received her bachelor’s degree in Genetics from the University of Kansas and her PhD in Cancer Biology from the University of Michigan. During her graduate career, she studied DNA repair mechanisms and several DNA repair genes that are linked to rare genetic diseases. She’s excited to use her background in genetics, science communication, and product development to make a difference in the lives of rare disease patients.
Brittany Anderton, PhD
Brittany has spent her career at the nexus of biology research, education and communication. She received her PhD in biomedicine from UCSF. After that, she did a nontraditional postdoc at UC Davis in science communication. She has taught introductory biology at Sacramento State and UC Davis. Prior to joining Rarebase, Brittany was Associate Director of iBiology at the Science Communication Lab, where she served as a video producer, communication trainer, and program evaluator.
As a child, Brittany lost her grandfather prematurely to muscular dystrophy. As an adult, she saw her friend’s life be transformed by the development of a new drug for their rare disease. Brittany aims to unite her expertise in preclinical research, STEM education, and science communication to support rare disease patients, families, and communities.
Devin Cavero
Devin started his career as a scientific researcher, using functional genomics and sequencing strategies to better understand disease and find potential therapies. After working at UCSF and the Chan Zuckerberg Biohub, Devin shifted his focus to education and advocacy, teaching STEM to youth experiencing homelessness in San Francisco. As the Community Engagement Coordinator at Rarebase, Devin advocates for and partners with our community of families and foundations affected by rare disease.
Devin is driven in this work by the experiences of family members and clients living with rare diseases. He hopes to unite his backgrounds in scientific research, education, and advocacy to serve as a fierce advocate and resource for all of our families in the rare disease community.
Alex Guin, PhD
Alex got her PhD from the University of Illinois at Urbana-Champaign in 2019. Her thesis focused on studying the effect of temperature on protein folding in living systems. During her PhD she worked at the intersection of data analysis and cell biology and so discovered her passion for data.
Post PhD she worked at Synthego corporation as a software engineer and data scientist developing image analysis pipelines to characterize gene edited cell lines using CRISPR. After Synthego, she worked as a computational biologist for Upside Foods, a cell-based meat company, supporting the research and development of cell lines optimized to form tissues in labs using data.
Alex is passionate about using data to make a difference in the life of people. At Rarebase, she hopes to bring together her experience in cell biology, software development and bioinformatics to help discover drug candidates for rare diseases.
Ursula Leone Haditsch, PhD
Ursula is an experienced neuroscientist with a Ph.D. in Neurobiology from the Swiss Federal Institute of Technology Zurich. Her professional focus is on understanding how genetic mutations alter the formation and communication of neurons in the brain. She began her career by introducing genetic mutations into mouse models to improve neurogenesis and cognition. She later moved on to modeling human diseases in a dish by differentiating patient-derived induced Pluripotent Stem Cells (iPSCs) into brain-specific cells, including neurons, astrocytes, and microglia. iPSCs have recently gained prominence in disease modeling by recapitulating identical genetic and phenotypic aberrations, enabling the demonstration of personalized medicine.
Ursula looks forward to establishing iPSC-based cellular platforms at Rarebase to model rare diseases in vitro. She will apply her broad scientific expertise in cell-based drug screening to rapidly advance therapeutic discovery for rare diseases.
Mo Rahman
Mo's research career began in the Misono lab modeling membrane-bound receptors using X-ray crystallography, giving him a taste for in silico methods. He spent over two years at Novartis focused on characterizing liposome based nanostructures.
After that he started Genedrop, a company developing software for scientists to make computational biology more broadly accessible. The products include a native app to visualize and interact with genomic data, an AI trainable variant caller, a collaborative molecule editor and pipelines for pathway analysis.
Michael Genin, PhD
After surviving cancer as a young teen Michael has dedicated his life to drug discovery. He has been fortunate to learn from exceptional mentors and be part of efforts leading to marketed products and a compound that has recently been designated fast track by the FDA.
Michael has extensive experience building and leading teams/departments locally and internationally in autoimmune, cardiovascular, diabetes, metabolic and infectious disease areas.
Matt Deardorff, MD PhD
Matt is a clinical geneticist and molecular biologist who spent 15 years at the Children’s Hospital of Philadelphia investigating the genetic and cell biological basis of a number of rare disorders of syndromic intellectual disability. He is currently the Director of Personalized Care at the Children’s Hospital of Los Angeles, where he is passionate about applying genomic medicine to pediatric care and increasing therapeutic options for children with rare disorders.
Matt Might, PhD
Matt Might has been the Director of the Hugh Kaul Precision Medicine Institute at the University of Alabama at Birmingham (UAB) since 2017. At UAB, Matt is the Hugh Kaul Endowed Chair of Personalized Medicine, a Professor of Internal Medicine and a Professor of Computer Science.
From 2016 to 2018, Matt was a Strategist in the Executive Office of the President in The White House. In 2015, Matt joined the faculty of the Department of Biomedical Informatics at the Harvard Medical School, first as Visiting Professor and since 2017 as Senior Lecturer.
Matt is co-founder and Chief Scientific Officer of NGLY1.org, and he was a co-founder and Scientific Advisor to Pairnomix. Q State Biosciences acquired Pairnomix in October 2018 and Matt remains a Scientific Advisor and Board Member.
Prior to medicine, Matt spent nearly a decade as a professor of computer science at the University of Utah. In 2014, he was named one of University of Utah's first six Presidential Scholars.
Carolina Garcia Rizo, PhD
Carolina Garcia Rizo has been a leader of cross-functional teams worldwide with an extensive network across VC’s, Biotech, Big Pharma, Providers, Payers, and Regulatory Agencies.
She has a passion for leveraging Machine Learning to improve the efficiency and speed of drug discovery and development and has made significant contributions to the field of precision medicine by developing data-driven business models and bioinformatic strategy.