Rare Leader: Christina Waters, CEO and Founder, RARE Science
August 30, 2018
Name: Christina Waters
Title: CEO and Founder
Organization: RARE Science
Disease focus: RARE Science is a non-profit research organization that focuses on accelerating identification of more immediate therapeutic solutions for kids with rare disease potentially through repurposing currently approved drugs and other therapeutic approaches.
Headquarters: Encinitas, California
How did you become involved in rare disease: I was always interested in genetics. I grew up on a farm and we raised Suffolk sheep. At the time, there was a problem with breeding, so at a young age I was exposed to animal genetics. I was also a very curious biologist as a kid. When I went to school I was focused on getting my degree in biology. As an undergraduate I worked in genetics, and then I went on to get a Ph.D. in genetics. One of my first jobs after my post-doc was at the Genomics Institute (GNF) of the Novartis Research Foundation. At that point, we were working in all diseases, but we had a lot of effort in neglected and orphan diseases. That’s when I really understood that my purpose was to work for vulnerable patient groups and it’s when I understood that I needed to work on kids’ disease. RARE Science became an official non-profit organization February 2015.
Previous career: Currently serves as senior vice president and general manager of the Global Rare Disease Program at WuXi NextCODE
Education: B.S. in biology with a certificate in recombinant DNA from San Diego State University; Ph.D. in genetics from the University of California, Davis; Post-Doc at University of California, Berkeley; Howard Hughes Fellow at Caltech; and MBA from the University of California, Los Angeles
Organization’s mandate: Our mission is to accelerate finding more immediate therapies for kids with rare disease.
Organization’s strategy: The strategy is to work directly with patient families and foundations to navigate the fastest way to get to therapies that will help. As we know, drug development can take up to 14 years and costs more than $1 billion dollars, and families with kids with rare disease can’t wait. We want to address the urgency. Different families and foundations need different support, but we can help all of them unite families together to create a disease communities, which is the initial and most important part of research. If we can understand what’s similar and different across kids in one rare disease, that can help us understand the biology, and potentially repurpose drugs. From uniting families around the world, we can build ways of understanding the disease.
Funding strategy: We have a unique funding strategy. First, we are a completely volunteer organization and we put a lot of effort into the first part of the process of uniting families together to understand the biology of a specific rare diseases. We do this through our RARE Bear program. We have a global community and have about 4,000 volunteers, in 12 countries and every state in the U.S., and companies that work with us to make RARE Bears that we send to rare kids around the world. We gift the RARE Bears to kids and use them to raise awareness about specific rare diseases. We share what different rare diseases look like, and help the foundations and families find other families through this program.
The idea started with just one quilting group in San Diego that wanted to make bears for some local rare disease kids. Quilters have remnant fabric that is left over from the quilts they make. The RARE bears are made up of all kinds of different remnant fabric which results in one of a kind bears for one of a kind kids. The bear-makers use a pattern from Simplicity (of which we receive a portion of the proceeds), so they all are the same type, and we provide tags that have serial numbers. The unstuffed RARE bears are made by the bear makers and sent flat (coined “bear skins”) to our organization in Encinitas. The community stuffs them and sews them up at independent events and helps raise the money to ship the bears home to kids and thereby support our program. We have relationships with Simplicity, Spoonflower (which makes our special feet fabric), Mountain Mist, and Bernina (which is the Ferrari of sewing machines). We’ve tapped into a whole community of companies that have nothing to do with rare diseases or even science, but with home and family.
Right now, RARE Science and the RARE Bear Army has touched the lives of about 5,000 kids across 50 countries representing about 1000 different rare diseases. It is an interesting network and community of support as everybody knows somebody who is rare. The Rare Bear program has allowed us to work with industries outside of science and bring community together in a different way to support kids with rare disease. Everyone wants to help kids with rare disease, but often don’t know how, but we made a way for everyone to help. By tapping into this whole other world, we’ve built a program that brings in donations to support our programs, and we’ve enlisted companies that support craft, and home, and community. Like every nonprofit, we receive donations from our community and pharma, but they are one-off. What we want to do is grow and create a sustainable revenue source. Our RARE Bear program offers a revenue stream every quarter, so we can rely on some funding coming in instead of being concerned about the peaks and valleys of fundraising that happens in normal nonprofit fundraising strategies.
What’s changing at your organization in the next year: We work with a large number of families and we want to continue to expand and help more of these families around the world. One of the things I’ve learned over this year is that we can scale certain parts of our organization to achieve this. The RARE Bear program essentially runs itself. However, shipping costs are challenging, plus we have a large database that keeps track of RARE Bear families. We’re looking at ways to increase our sustainable revenue stream to send the bears out to kids and make connections, but also to ensure we are building ways to support families outside of the bear program in the area of research. An example of this is the inducible pluripotent stem cell (iPSC) program that we conducted which was supported by the California Institute for Regenerative Medicine. Our first iPSC collection for—ACDY5—will be made available to scientists around the world this fall. But to scale this, and to scale science, research, and discovery, we need to increase our revenue stream to support growth of our technological offerings and our research support to families. That’s what’s going to happen in the new year.
Management philosophy: As a completely volunteer organization, we know that for the people who come to our organization, it’s really about passion and making impactful changes in the lives of kids. They want to make a difference. Everyone on our team is connected to rare disease or has a passion to help kids. You’d think managing 4,000 volunteers across the globe would be a management challenge, but we created ways that these groups and countries can work independently and contribute to the mission of our organization. It’s similar to a model like the American Red Cross, where you have chapters. Our chapters are quilt guilds around the world. We also have a core team in San Diego.
For the local operational team, we want to support people in learning new things of interest. We have volunteers for a long time and some for a short time. We align responsibilities with their passions and make sure it helps them with their career by giving them experience in areas that they want to pursue. We have graduate students whose master projects are aligned with where they want to go in their career, but also aligned to the goals and mission of RARE Science. We also support young women in STEM. Young ladies can volunteer and get experience in genetics, data science, and more within a group that is going to support them in a fun and impactful learning environment. We have found that everyone has a way in which they can contribute even outside of our core group, we have found ways that other people in our community can participate.
For example, when the bears come back, they are flat and need to be stuffed and sewn up. It’s a huge operation and we’ve tapped into a whole group of people who want to participate in something that is meaningful and valuable but have never had an opportunity to do that. We work with active senior adults through the YMCA. We’re also offer our program in senior homes and memory care units. In addition, we also work with companies wanting community service events to full programs for Girl Scouts (we even have our own patch). Everybody can stuff, and everybody can sew. Now everyone can help a child with a rare disease. It’s been interesting to see how tapping into quilters, sewists, and community have really driven forward an understanding of rare diseases by a whole group of individuals we really didn’t know would be so instrumental when we started RARE Science.
Guiding principles for running an effective organization: I lead the organization like a company, even though we are a volunteer-based nonprofit. We have a strategic plan and a budget. What’s important is to have a mission and goal, and communicate that clearly to our volunteers, and have that match the strategic plan and budget for the year. The plan can and should change because we are always listening to the needs of our patient groups. But if something changes, it is not an add-on but a reprioritization. Making sure we have a sustainable organization that can move into the future is our responsibility to the families that we serve and the people who volunteer for RARE Science. It also goes without saying one of the most important principles of an effective organizations is integrity – which is not only doing what we say but showing results.
Best way to keep your organization relevant: In the rare disease space, things are moving very fast, which is a great thing. Policies are being put together right now on data-sharing and guidance on the technology that we use to analyze the data. The only way we’re going to identify what genes are relevant to rare disease is through data-sharing—globally. An important part of keeping relevant is listening to the parents’ needs and keeping up with technology, policies and global collaboration. You can make a plan that you follow, but to keep relevant, you need to evolve and change, and make sure you are flexible and able to prioritize based on the urgent needs of the families and being able to reach the goal.
Why people like working for you: I think people like working for RARE Science, which has been created out of my heart, because they align with the passion of what we’re doing. It comes back to the fact that we’re all volunteers. You don’t have to wonder about anyone’s agenda within the organization. We try to align so everyone gets what they want out of the experience. I think the reason we keep growing is that we align with everyone’s passion and we’re able to deliver on our mission as an organization to urgently address the needs of rare families, and work towards creating a foundational understanding of rare diseases by uniting families. We are also able to fulfill the desires of the community to participate in a way that works for them. Not everyone is going to understand genetics, science, rare disease, and sometimes it’s scary. However, everyone can identify with a bear. We made a way through the RARE Bear program to drive deep research through a community where everyone—scientists to sewists—can contribute to helping these special kids.
Mentor: There are different people who have really made an impact on my life. When I was at GNF, one of my supervisors was Paul Herrling, who at the time was head of research for Novartis. He told me one time that there was a lot I could contribute working at the laboratory bench, but one of the most important things I would learn would be from talking to patients. I had the opportunity and fortune of working with professor Herrling while visiting small hospitals in Singapore working on rare and neglected diseases. He was one of the most impactful people who made me understand the connection between knowing and understanding research, but that when you want to apply it you have to listen to the patient.
On the Job
What inspires you: I get calls or emails from families every day. Many of them are on the diagnostic journey. Right now, 30 percent of the time when we sequence kids that come into the hospital, we can identify causal biology or genes. Seventy percent of the time we still have no idea what is causing the disease. It is the families on this diagnostic journey, driving research forward to understand how to help their child’s disease, that are absolutely my inspiration—their courage, their determination, their tenacity. It gives me no excuse to be tired.
What makes you hopeful: The new technology we have that’s coming out makes me hopeful. The ability to start understanding how we bring large genomic, other omics, and phenotypic data together to really start identifying new genes and new gene variants that are causal, and the data science that goes along with it—the deep learning that we can build to understand and validate these new causal genes. This is the way of the future. This is where we are going to identify the molecular pathways and the targets that are going to help us repurpose drugs for the now and seed new drug discovery projects for the future.
Best organization decision: I think some of the best decisions we’ve made is to focus as every day we don’t lack for ideas in our organization. We have so many ideas and a lot of people who want to help. The hardest decisions and the best organization decisions I have made has been to focus our activities on what we do well. It is easy to add more activities but then we get spread too thin and we are not able to do the deep efforts on those things that are really impactful. We have a strong focus on the events and activities that we participate in, and how we are going to develop our technology and research offerings for the future.
Hardest lesson learned: The hardest lesson learned, and what I continue to work on, is how to communicate what we can do in science, policy and regulation clearly, and how to educate people on what we’re doing along these lines with listening to what patient families need. How do we make sure we are using words we both understand? We work to keep the message the same to patient families, clinicians, and researchers the same but use different words, so we create understanding and educate as we go along
Toughest organization decision: The toughest decisions relate to things we have had to cut out of our offerings. There are things people have really identified with and loved to do. It is tough to say “no” to some of the activities volunteers propose and I try to ensure they understand why we need to prioritize. There are only so many resources, budget, and time—it is better to have quality vs. quantity.
Biggest missed opportunity: We are putting together a way to generate revenue to be able to do what we have missed doing in the past. We want to be able to build a way for united families to share information—on what works for them and what doesn’t work for them. When I talk to families individually, they have nuggets of gold to share with each other. I currently connect moms with older children with the same disease with moms with babies that have just been diagnosed to share learnings, support, and resources. If we can somehow automate the way we can connect the families that we have already brought together through building communities, it would be so valuable. We can really help with quality of life, resources, and with maybe some nuggets of information that can help them along their journey.
Like best about the job: Being able to bring hope and helping people. The whole reason why RARE Science was created from my heart was because I saw the urgency of these families that no one was addressing. To see the progress these small rare disease groups are making is inspiring. Just to have the ability to help them along the way is everything to me.
Like least about the job: Not having enough time to do everything I want to do.
Pet peeve: People saying they are going to do something and not doing it.
First choice for a new career: I’ve worked in genetics my whole life. This is my purpose. I can’t imagine doing anything else. RARE Science is my passion. I am so grateful that my paying job as leading the Global Rare Disease program at WuXi NextCODE aligns with my passion. I want the success of the program for WuXi NextCODE to help bring technology to the families and I want to see RARE Science grow to really help these families move forward. It is my hope that one day the work I do at WuXi NextCODE and RARE Science is not needed, but until then, I am dedicated to helping rare disease patients
Most influential book: Readers Digest Great World Atlas from 1963, which is on my coffee table. I would pull this out as a kid and always look at it. I was so curious about the world, where people were, and what was happening in all these different places.
Favorite movie: I love old sci-fi movies. I’m a sci-fi buff.
Favorite music: I love rhythm and blues, acoustic music, and my new favorite group is The California Honey Drops.
Favorite food: Every morning I chop up kale, ginger, turmeric lemon, apple and puree it all. That’s my go-to get going food in the morning. It’s my own concoction
Guilty pleasure: I live close to the beach. I often run down to go swimming in the ocean. That’s my thing. That’s what I do to energize, clear my mind and stay focused
Favorite way to spend free time: Long walks, yoga, and meditation. You see so many things when you are walking and is a great way to keep a clear mind.
August 30, 2018
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