Nephrogen
About Nephrogen
Nephrogen is developing curative gene therapies for autosomal dominant polycystic kidney disease (ADPKD) using CRISPR-Cas genome editing and engineered adeno-associated viruses for targeted delivery to the kidneys. This approach aims to halt disease progression and provide a definitive cure for the 600,000 Americans affected by ADPKD, a condition with no current effective treatment.
```xml <problem> Autosomal dominant polycystic kidney disease (ADPKD) affects a large population and currently lacks a definitive cure or truly effective treatment, leading to significant morbidity and mortality. Existing treatments do not halt disease progression, leaving a critical unmet need for therapies that target the underlying genetic cause of ADPKD. </problem> <solution> Nephrogen is developing curative gene therapies for ADPKD using a platform that combines CRISPR-Cas genome editing with advanced nucleic acid delivery methods, such as adeno-associated viruses (AAVs). Their approach focuses on delivering gene therapy directly to the kidneys to correct the genetic mutations responsible for ADPKD. By targeting the source of the disease, Nephrogen aims to halt the progression of ADPKD and provide a lasting cure. The company's platform, NeFIND TM, utilizes AI and high-throughput in vivo screening to engineer novel gene delivery vectors that are more efficient and less immunogenic than existing vectors. </solution> <features> - CRISPR-Cas genome editing for targeted correction of ADPKD-causing mutations - Engineered AAV vectors for efficient and specific delivery of gene therapy to the kidneys - NeFIND TM platform for AI-driven discovery of novel gene delivery vectors - Non-invasive method for local delivery of gene therapy to the kidney - High-throughput in vivo screening to identify optimized gene therapy candidates </features> <target_audience> The primary target audience includes patients with autosomal dominant polycystic kidney disease (ADPKD) and the healthcare providers who treat them, such as nephrologists and genetic counselors. </target_audience> ```
What does Nephrogen do?
Nephrogen is developing curative gene therapies for autosomal dominant polycystic kidney disease (ADPKD) using CRISPR-Cas genome editing and engineered adeno-associated viruses for targeted delivery to the kidneys. This approach aims to halt disease progression and provide a definitive cure for the 600,000 Americans affected by ADPKD, a condition with no current effective treatment.
Where is Nephrogen located?
Nephrogen is based in East New York, United States.
When was Nephrogen founded?
Nephrogen was founded in 2015.
How much funding has Nephrogen raised?
Nephrogen has raised 330000.
- Location
- East New York, United States
- Founded
- 2015
- Funding
- 330000
- Employees
- 3 employees