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kidney diseases.

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OUR MISSION

Nephrogen is a discovery-stage therapeutics company developing gene therapy for kidney disease.

Our beachhead indication is autosomal dominant polycystic kidney disease (ADPKD), which affects 600,000 Americans and has no cure or effective treatment. Tolvaptan (JYNARQUE®) is the only FDA approved drug for ADPKD, costs $13K for a 28-day supply, and must be taken twice daily for decades for maximum benefit. Our solution aims to halt ADPKD from the onset and provide an outright cure rather than a temporary Band-Aid®.

We're boldly committed to reimagining therapeutics for life threatening kidney disease. Our broad patent portfolio will allow us to rapidly translate our technologies into foundational partnerships and transformative treatments.

Our technology

Gene therapy for kidney disease.

Nephrogen is harnessing recent advances in genome editing (CRISPR-Cas9) and nucleic acid delivery (AAV, nanoparticles) to develop outright cures for genetic kidney disease. We are advancing several different therapeutic approaches including ex vivo perfusion and re-transplantation, in vivo local delivery, and in vivo systemic delivery.

Ex Vivo Editing

We take the kidney out of a patient, perfuse it with our editing solution, and then put it back in.

In Vivo Localized Delivery

We inject our editing solution directly into the kidney.

In Vivo Systemic Delivery

We inject our editing solution into the bloodstream (or even orally) and it finds its way to the kidney.

Rapid drug discovery

Nephrogen's drug discovery platform leverages innovations in computer vision and high-throughput screening (HTS) to find new small molecule drugs for other cystic kidney diseases, including and nephronophthisis.

Our team

Leadership

Demetri Maxim
Founder and CEO
Antonio Varone
Chief Scientist

Advisors

George Church, PhD
Robert Winthrop ​Professor of Genetics, Harvard Medical School
Scientific Advisor
Terry Watnick, MD
Director, Baltimore Polycystic Kidney Disease Research and Clinical Core Center
Scientific Advisor
Ron Perrone, MD
Professor of Nephrology, Tufts University School of Medicine
Scientific Advisor
Anil Chandraker, MD
Medical Director of Kidney Transplants, Brigham and Women's Hospital
Scientific Advisor
news & publication

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