Company


Company
Ninevah Therapeutics (NTX) is a discovery company developing gene therapies for kidney rare diseases.

NTX is building a discovery pipeline with gene delivery platform technologies.

Our lead program is an AAV-based therapy for genetic nephrotic syndrome (NTX014). NTX is translating NTX014 from discovery to a first-in-human trial.

Initially, we are focused in genetic nephrotic syndrome with the goal to expand our platform technologies to other kidney diseases.

NTX was founded in Barcelona Q2-2019

Pipeline


AAV-based Kidney

A discovery program to develop an AAV-based therapy for nephrotic syndrome (NTX014)

Un-disclosed IP

A discovery program to develop a novel platform focused on genetic kidney diseases (NTX077)

Disease


Disease
Nephrotic syndrome (NS) is a collection of symptoms due to kidney damage.
NS is defined as heavy proteinuria severe enough to cause hypoalbuminemia, edema, and hypercholesterolemia.

  • The second most frequent cause of end-stage kidney disease (ESKD) in children.
  • 20% of patients become resistant to steroids (SRNS) and only ~20-30% of pediatric patients are steroid-sensitive.
  • SRNS leads to end stage renal disease (ESRD), the last stage of chronic kidney disease (CKD) in more than 50% of patients.
  • 30% of cases (or more) of SRNS are caused by mutations of genes.
  • Genetic forms of NS affects 1 to 3 per 100,000 children worldwide.

The features of nephrotic syndrome are caused by failure of the kidneys to filter waste products from the blood and remove them in urine. Signs and symptoms of this condition are excessive protein in the urine (proteinuria), increased cholesterol in the blood (hypercholesterolemia), an abnormal buildup of fluid in the abdominal cavity (ascites), and swelling (edema). Affected individuals may also have blood in the urine (hematuria), which can lead to a reduced number of red blood cells (anemia) in the body, abnormal blood clotting, or reduced amounts of certain white blood cells. Low white blood cell counts can lead to a weakened immune system and frequent infections in people with congenital nephrotic syndrome.

Children with genetic forms of nephrotic syndrome typically develop end-stage renal disease between ages 2 and 8, although with treatment, some may not have kidney failure until adolescence or early adulthood.

This condition is inherited in an autosomal recessive pattern, which means both copies of the gene in each cell have mutations. The parents of an individual with an autosomal recessive condition each carry one copy of the mutated gene, but they typically do not show signs and symptoms of the condition

Unfortunately, there is no specific treatment available.

Team


Marc Ramis Castelltort, PhD

Co-founder & Interim CEO

Marc is passionate about creating new biomedical ventures. He is a co-founder and CEO at Senolytic Therapeutics Inc., a pharmaceutical company that develops a novel class of medicines by targeting senescence cells. He also supported investments and strategic partnerships at Life Biosciences Inc. in Europe. Currently, Marc is a strategic advisor at Retinset Therapeutics, Cyclomed, Cebiotex and Origen Ventures. He is a co-founder and Partner at Chasing Science, an early-stage biomedical venture builder. Marc brings experience as advisor and board member for multiple organizations and as co-founder of several companies. Marc gained a PhD in Biochemistry from the University of Oxford in 2006, and a Harvard Business School PLD Program in 2011.

Gonzalo Fernández-Miranda, PhD

CSO & COO

Gonzalo combines a solid scientific background from his 15 years working in top research centers (CNIO, CRG and IRB Barcelona) with a passionate attitude and hands-on experience in entrepreneurship in the biotechnology sector. Since 2016, he linked his research in the laboratory with the creation of a spin-off whose main objective was to attract private investors to develop an antitumor molecule. He was selected as Project leader of the CaixaImpulse grant (2017 edition) and performed executive training from ESADE “From Science to Business Course”. He also serves as a consultant and venture builder at Chasing Science to support the development of therapeutic programs . He holds a PhD with European distinction in Molecular Biology from Universidad Autónoma de Madrid.

To be announced soon

Gene Therapy expert

To be announced soon

Renal biology expert

To be announced soon

Pediatric nephrologist 1

To be announced soon

Pediatric nephrologist 2

Contact


  • Carrer de Baldiri Reixac, 4, Barcelona, Spain