Fighting inter-generational kidney disease is my 'why'

Today Judo Bio announced its $100M Series A to create siRNA therapies targeted specifically at kidneys. And I relish reading this as ending inter-generational kidney disease is truly my ‘why’ for working passionately for biotech.

Family and personal history

My family has been impacted over generations by Autosomal Dominant Polycystic Kidney Disease (ADPKD). My grandmother died at the age of 42 when cysts — which are technically benign — overtook her kidney function. This was in 1973 when dialysis was not even a mainstream option for extending life with failing kidneys. Her age when she died is particularly relevant as this is the birthday milestone I most recently passed. Fast forward several decades to 2005, my mother’s kidneys had also reached the threshold of failure at >10% function. With the cystic invasion, each of her kidneys reached the the size of an American football. Fortunately, science had advanced with the advent of mainstream organ transplantation. Her sister, my Aunt Anita, kindly provided this kidney and scientists had brilliantly created therapies to reduce rates of rejection of donated organs. That my mother continues to travel around the world nearly 20 years later is a miracle of scientific advancement! Fast-forward to my own journey where I have been tracking the progress of my cysts since my teenage years.

While they still function, kidney failure is likely in my future though with the help of science (yet again!) my journey is already different than my mother’s. For the last couple of years, I’ve been on a $17,000/month medication approved by the FDA just the year before to slow the growth of these cysts. The FDA tracks my health as part of a Risk Evaluation and Mitigation Strategies (REMS) program, so I’m already part of a longitudinal study. My hope is that this medication extends the time horizon long enough that by the time I do need a replacement, we’ll have the ability to 3D print kidneys from stem cells. (I suppose I could also use a genetically modified pig kidney, as has been recently successful in a patient, though I’d prefer we didn’t resort to modifying colonies of pigs for this purpose.)

Really, it’s our shared ‘why’

Which all gets to the purpose of this industry: to advance ways to prevent and treat life-changing health conditions as soon as possible. When I think of my kidney function, I think I’ve got 10ish years. So the clock is ticking on companies like Judo Bio, Alnylam, Borealis Biosciences, and Vertex to help me. And most importantly, when I look at my children, I truly believe that within their lifetime we will have treatments to reverse the condition and be able to edit this autosomal dominant condition out of family line for my potential grandchildren onward.

Truly I believe this is all of our ‘why.’ Those of us in the everyday work of biotech and pharma can get stuck in the daily grind that is inherent in the many-(many-)years-long journey full of failures, heartache, and lost millions of dollars that go into creating successful new treatments. But ask any of us, no matter what part of the ‘big group project’ we are working on, we will all say that we’re out here to make this difference. It’s empowering to share a massive mission to improve the human condition. That we’re all part of the better-each-generation process of scientific advancement. We will all say this is the underlying reason we wake up each morning, because we know we can open new ways of approaching old conditions. So thank you for your part in this progress generally, and potentially for my family specifically.

With gratitude,

Kate

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The Funnel is actually The Tube - Part 1