#United States #New York #Gene Editing #NKF Innovation Fund #Nephrogen

NKF Innovation Fund Invests in Nephrogen

The National Kidney Foundation (NKF) has unveiled a strategic investment from its Innovation Fund into Nephrogen, a cutting-edge biotechnology firm focused on developing one-time gene editing therapies for various kidney diseases. This partnership is expected to propel advancements in delivering gene therapies specifically targeting the kidneys, an area that has historically faced significant challenges.

The Challenge of Kidney Disease Therapies

Kidney disease affects over 37 million adults in the United States alone, with most individuals unaware of their condition. While gene therapy presents revolutionary possibilities for treating diseases at their genetic roots, its application in kidney disease has proven ineffective until now. According to the American Society of Gene and Cell Therapy (ASGCT), no gene therapy for kidney ailments has yet been approved, and only one known therapeutic program for kidney cells has begun clinical trials. The complexities of the kidney's structure and its filtering capabilities create barriers for effective gene therapy delivery, with disease stemming from more than 15 different cell types.

Nephrogen's Innovative Approach

Nephrogen is developing the NeFIND™ platform, which innovatively combines artificial intelligence with high-throughput screening to invent new gene therapy delivery solutions. This technological edge aims to breach the barriers that have previously hindered progress in treating kidney diseases. Early preclinical studies on mice indicate a promising delivery rate of up to 60% to kidney tubular epithelial cells. Notably, Nephrogen has also tested gene delivery methods on intact human kidneys maintained on normothermic machine perfusion, marking a significant milestone in directed delivery techniques.

Kevin Longino, NKF’s Chief Investment Officer and a recipient of a kidney transplant, emphasized the importance of this investment, stating, "Nephrogen addresses a fundamental problem that has impeded the introduction of transformative therapies in kidney treatment. The Innovation Fund aims to accelerate innovations with the potential to drastically alter the landscape of kidney disease management."

Focus on Major Kidney Conditions

Nephrogen’s flagship project targets autosomal dominant polycystic kidney disease (ADPKD), the most prevalent genetic kidney disorder. Moreover, the company has plans to utilize its NeFIND™ platform to tackle APOL1-mediated kidney diseases and eventually extend its therapeutic reach to a wider range of kidney conditions, including chronic kidney disease.

Demetri Maxim, the CEO and co-founder of Nephrogen, shared a personal connection to the mission, saying, "Families dealing with inherited kidney diseases have long been told to merely manage decline instead of addressing its root causes. The inefficiency in delivering genetic material to the responsible kidney cells has been a major challenge that we intend to overcome with our platform."

Advancing Towards Future Treatment

The NKF's investment will not only facilitate the expansion of the NeFIND™ platform but will also support Nephrogen's ADPKD project, moving it closer towards studies required for Investigational New Drug (IND) applications. Launched in 2021, the NKF Innovation Fund is dedicated to accelerating the funding and development of kidney-focused therapies and aims to overhaul kidney care experiences for patients, eliminate transplant waitlists, and enhance treatment regimens for those on dialysis.

For more details about the NKF Innovation Fund, visit their website at kidney.org/innovationfund.

Conclusion

The investment from the NKF Innovation Fund into Nephrogen signifies a pivotal step forward in addressing kidney diseases through innovative genetic therapies. As renal health continues to decline for millions, this collaboration ushers in hope for new, effective approaches that could very well change the course of treatment for a community long in need of advancement. The focus on research and development emphasizes a commitment to finding long-lasting solutions that go beyond mere management of kidney diseases.