With the help of a 2023 XSeed Award, Rohini Kavlakuntla, MSc, and her two graduate school buddies are poised to make genetic medicine accessible and affordable for those with debilitating kidney diseases. Kavlakuntla is Co-founder and Chief Growth Officer, Helex, a startup focused on developing gene editing-based therapeutics to meet the significant underserved medical needs of patients with genetic kidney diseases.
Kavlakuntla recently spoke with Cure about the impact of winning the XSeed Award and where Helex is headed. This conversation has been edited for length and clarity.
Cure: You have a business background. Did you always want to go into healthcare?
Rohini Kalvakuntla: I worked in public health for about nine years on the strategy and pricing of large-scale public health programs. I realized there was a huge gap in the accessibility and affordability of medicines for the people who need them. My driving force has been to work at the intersection of science, technology and health systems to benefit patients and their families.
Cure: How did you come to form your company, Helex?
Kalvakuntla: My co-founders and I lived together in London while we were all doing our masters degrees at King's College in 2009. After graduating, we all moved back to India and worked in our respective jobs. Fast forward to 2021: We had all seen how the cell and gene therapy field was blowing up in the West, but the accessibility of these therapeutics in India was very, very limited. India is a country of nearly 1.5 billion people, so even a rare genetic disease is not really rare there.
So, we formed Helex with the mission to not only develop high-quality genetic medicines, but to make them accessible, scalable and affordable to all patients and healthcare systems. One of my co-founders, Poulami Chaudhuri, has a PhD in molecular biology and has worked with lipid chemistry experts in India on the underlying technology for non-viral delivery into specific cell and tissue types. She has also worked with variable 3D genome architectures within cell types that facilitate customizing genetic material for greater precision to target cells. That is the technology we currently leverage at Helex.
My other co-founder, Anirudh Nishtala, has a master's in molecular biology and worked for 10 years in pharma in clinical operations and manufacturing, including regulatory compliance. With our complementary skill sets and an aligned mission, the three of us have been able to build Helex with the aim of providing a better quality of life to patients.
Cure: Tell us a little more about how the technology works.
Kalvakuntla: It’s a non-viral lipid nanoparticle-based drug delivery platform focused on extra-hepatic tissues like the kidney. We also modify the genetic material (such as mRNA, gRNA, etc.) to suit the specific cell type and disease background for higher precision to accelerate the development of de-risked therapeutics.
The field of gene therapy and other advanced therapies is rapidly growing, but one of its most significant limitations is delivery, especially for certain organs like the kidney. The kidney is a natural filtration unit where everything that goes into it gets filtered out, so we have to employ some novel strategies; we go through many iterations and optimizations to make our lipid nanoparticles (LNPs) go into specific cell types within the kidney.
Cure: That’s fascinating. You’re currently focusing on applying this technology to treat a genetic kidney disease called Autosomal Dominant Polycystic Kidney Disease (ADPKD). Why is this disease your target indication?
Kalvakuntla: We started focusing on how our technology could treat an eye disease called Stargardt’s disease, but quickly realized the technology could be more valuable in disease areas with an even higher unmet need, such as renal indications and the kidney.
There are 200 million people worldwide who suffer from genetic forms of kidney disease, most of them eventually ending up on dialysis. There are far fewer researchers trying to address the issue. Our lead indication, Autosomal Dominant Polycystic Kidney Disease (ADPKD), is a rare disease, but it's one of the most prevalent rare diseases, affecting 12 million people around the world.
Due to genetic inheritance, the kidneys develop cysts, becoming so cystic over time that they cannot function. Patients end up on dialysis a few times a week, and by the age of 55 or 60, they reach end-stage renal failure, where they need a kidney transplant. Since this is a genetic disease, family members most likely also have the disease and cannot donate their kidneys, so it becomes incredibly difficult to find a donor. There is an approved drug that slows down cystic progression, but it causes liver damage.
As you can appreciate, this patient community needs a paradigm shift in their care pathway. We believe our approach to treating ADPKD can be a game changer for patients and their families.
Cure: What are some of the challenges you’ve faced as a start-up, and how did you overcome them?
Kalvakuntla: There are many challenges. As first-time founders, we had to do a deep level of introspection to see what business model to pursue to justify our ultimate vision. We knew the underlying platform technology could do so much, and the company could take many different paths. But thinking about it from the perspectives of patients, market competition, and health economics, and with our own close personal experiences with people who suffer from chronic kidney conditions, we decided to focus on building meaningful therapeutics for renal indications.
Another challenge is staying focused on the big picture, given the day-to-day scientific grind for our team. We invited people who have ADPKD to talk to the team about their life challenges, and this helped us all recommit to what we're trying to solve. So, our challenges have been multifold, but we view them as opportunities to be more creative and grow from them.
Cure: You won a 2023 XSeed Award. What were you able to do with the funding?
Kalvakuntla: The funding was critical in developing our program from what was conceptual to generating proof of concept data by performing key experiments to de-risk our technology. We showed proof of concept in patient cells, and we've also been able to show in vivo data for kidney delivery of our lipid nanoparticles into specific cell types. Building out this data set gave us conviction in the strategy we're pursuing.
Cure: Other than the funding, how did the XSeed Award help you?
Kalvakuntla: As soon as we were recognized as XSeed winners, people were willing to chat with us because they knew we must be doing something right. And we've been able to interact with the entire Cure network — Deerfield Management, advisors from Eli Lilly and a number of other pharmaceutical companies — people we can ask for feedback about the direction we're taking. And Seema Kumar, the CEO of Cure, has been a driving force in pushing us forward on multiple fronts to give us visibility and to help us make connections.
So, we’ve been incredibly lucky to have these mentors, advisors and peers who also won the XSeed Award, with whom we meet all the time. This culture — the human social capital of the Cure biotech network and beyond — provides so much. Being plugged into it is critical for a young company like ours.
Cure: The XSeed Award is given to minority- and women-led businesses. Have you dealt with any issues as a minority founder that some other founders may not have encountered?
Kalvakuntla: I’m a minority, a woman and an immigrant — I moved to the U.S. a few years ago. It may seem like the odds are stacked against us, and we have to work that much harder. But even though there are challenges, I don't know that they have held us back. If anything, we're taking advantage of the resources available to us as minorities to continue to move forward.
Cure: What has your experience in the U.S. been like — and living in New York in particular?
Kalvakuntla: The U.S. is a place where innovation thrives. It's true to its word. With all the people we meet, there's so much openness to our ideas and what we can accomplish. The thinking is unlimited here, and that has given me the space and opportunity to push boundaries, both professionally and personally. That's something I really enjoy. New York has made me feel at home — this place can belong to everybody. That's what makes it so special.
Now in its fourth year, the XSeed Award program provides up to $250,000 grants to New York City minority- and women-led life science and healthcare startups working on novel preclinical drug development projects. Winning teams also join the ecosystem of Cure.®, a healthcare innovation campus in New York City. The teams receive peer-learning and office hours with investors, entrepreneurs, and business experts. Learn more.