I am Harsha Rajasimha, Founder and Chairman of IndoUSrare, an independent non-profit 501(c)(3) tax-exempt organization focused on helping patients with rare diseases of Indian origin in the USA, Indian subcontinent and globally with a vision to build collaborative bridges between the east and the west to accelerate research and development of diagnostics and therapies through education, advocacy, grants, and investments.
My first encounter with a rare disease was in 2004 when I was a graduate student at the Virginia Bioinformatics Institute analyzing next-generation sequencing (NGS) data of bacteria and viruses causing rare infectious diseases including the Coronavirus. A few years later, in 2010, I was a post-doctoral data scientist at the neurobiology, neurodegeneration and repair laboratory (NNRL Chief: Anand Swaroop) at the National Eye Institute, NIH, analyzing NGS data sets from inherited retinal diseases.
As we were discovering novel rare and common mutations in FBN2 (an extracellular matrix gene) associated with macular degeneration in 2012 at NEI, I and my family were introduced to the sufferings and pain that over 300 million patients and families with rare disease(s) deal with world-wide. I had become the father of a child with Edwards Syndrome, a rare congenital disease. The baby was diagnosed at full-term birth and did not make it back home from the NICU (neonatal intensive care unit). I and my family were devastated. As we went through several months of soul searching and education, my eyes were opened up to the other side of rare disease research, patient advocacy. I decided to dedicate a certain percentage of my time volunteering for the rare diseases community.
In the majority of my time, I decided to apply years of post-doctoral training in genomics and precision medicine towards accelerating the discovery and development of novel diagnostics and therapeutics for rare and orphan diseases.
After my 15+ years of involvement in the rare diseases community spanning genomics research, policy, data sharing, technology consulting, AI and digital health, entrepreneurship, volunteering, and advocacy, for various non-profits in capacities ranging from a volunteer, scientist, director, board member, co-founder, chairman, etc., having collaborated extensively with numerous pioneering organizations and individuals, I have gained unique insights into the world of rare diseases and orphan drugs from various stakeholder perspectives.
Realizing that enormous work is being done by numerous organizations globally, I teamed up with key opinion leaders and colleagues to identify the unmet needs and gaps by listening to the community members.
There are about 4 Million Indian Americans representing the fastest-growing ethnic minority group in the United States, over 18+ million Asian Americans, and over 22% of the World's population living in the Indian subcontinent. The worldwide population of Indian or Asian origins is roughly half!!! Thus, IndoUSrare is born in the USA (the single largest funding nation for rare diseases research) to address the unique needs of these groups that no other organization is currently focused on.
Our primary goal is to accelerate the discovery and development of diagnostics and therapeutics for rare diseases. We wish to create viable commercialization pathways or alternative access (and affordability!) for global patients to access approved therapies. Our approach will be to invest in results-oriented projects with a commitment to our core mission and a laser focus on the measurable impact. This is a complex problem requiring global cooperation with organizations, initiatives, resources (without duplicating efforts!), and key stakeholders including patient groups, national and international policy think tanks, sponsors, funding agencies, and researchers. I am grateful to the commitment and guidance by our founding board of directors towards this cause and helping shape the programs towards impact, sustainability, and scale.
I welcome each one of you to engage with IndoUSrare and other organizations to be a part of this rare revolution that is unfolding before us! With four FDA approved gene therapies, there is much promise of having the availability of gene therapy for most single-gene disorders within our lifetimes. Let us work together to ensure these therapies are affordable and accessible to patients worldwide!
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