The Programs

Building a pipeline backed by validated science and long-standing expertise in small molecule drug discovery

picture of mitochondria

Driving Cellular Function

Natural biological networks or pathways signal to cells to perform essential operations to maintain order and efficiency in the body. When those networks become impaired by age, disease or genetic mutations, cells no longer function as they should.

Cells are sophisticated structures equipped with specific functions that help to maintain order and efficiency in the body. Cellular function is maintained in part by natural biological networks that orchestrate pathways involved in the regulation of lysosomal biogenesis and autophagy, critical processes for removing cellular waste. Nutrient sensing also contributes to how cells respond. In fact, nutrient sensing can become dysregulated with age and disease – contributing to disease by affecting the processes that convert nutrients to energy which are essential for cell survival.

Generian is amplifying (or boosting) the intrinsic activity of the body’s natural signaling networks to improve cellular function treating and ultimately, reversing the course of disease.

Targets that Matter

Leveraging decades of research from some of the foremost experts in age-related diseases and E3 ligase biology, Generian has identified novel therapeutic intervention strategies to modulate the activity of network regulators (TFEB and AMPK) which are validated therapeutic targets essential to cellular function that diminish with age, disease or genetic mutations.

TARGET: TFEB

TFEB (or Transcription Factor EB) plays an essential role in cellular quality control by regulating autophagy and lysosomal biogenesis. TFEB augments clearance of intracellular aggregates, damaged organelles and granules, and extracellular material. This makes it a potential therapeutic target for diseases of protein or lipid aggregation, impaired quality control, and defective pathogen clearance including: 

  • Lysosomal storage diseases 
  • Pancreatitis 
  • IBD and Crohn’s disease  
  • CNS Proteopathies (Parkinson’s disease, Lewy body dementia)  
  • Dry AMD 

TARGET: AMPK 

AMPK (or AMP-activated protein kinase) helps to maintain cellular fitness, playing an essential role in nutrient sensing and appropriate cell metabolism. AMPK’s role as a key regulator of several metabolic pathways make it a potential therapeutic target for treating diseases of impaired energy metabolism, including: 

  • Primary renal disease 
  • Myotonic Dystrophy Type 1 
  • Diabetic nephropathy/NASH 
  • Primary mitochondrial diseases 

Our Pipeline 

Generian’s pipeline is backed by unique expertise in E3 ubiquitin ligase biology and small molecule drug discovery.

Pipeline of Network Activator Programs 

Pipeline of network activator programs

Our Pipeline 

Generian’s pipeline is backed by unique expertise in E3 ubiquitin ligase biology and small molecule drug discovery.

Pipeline of Network Activator Programs 

Pipeline progress bar of network activator programs

TFEB 

Autophagy & Lysosomal Biogenesis

Pipeline progress bar of network activator programs

AMPK 

Metabolism & Mitochondrial Function 

Pipeline progress bar of network activator programs

Novel Target Discovery 

E3-regulated Signaling and Transcription Factors

Track Record of Small Molecule Drug Discovery 

Leadership