Renthera Programs
TG297 for PKAN Disease
TG297 for PKAN Disease
PKAN is an autosomal recessive genetic disorder caused by mutations in the Pantothenate Kinase (PANK2) gene. PKAN leads to progressive degeneration of the nervous system and build up of iron in the basal ganglia region of the brain.
Classic PKAN usually presents in a child’s first ten to fifteen years, though there is also an atypical form that can occur up to age 40. PKAN is a progressively degenerative disease, that leads to loss of musculoskeletal function with a devastating effect on quality of life.
One to three million individuals are diagnosed world-wide every year with a patient population potentially three times larger that is not formally diagnosed.
Target and Approach
Renthera has patented six distinct vectors of PKAN treatment including rAAV vector and Baculovirus vector (AcNPV).
The goal of the gene replacement/implementation is to correct pantothenate kinase related neurodegeneration, specifically brain iron accumulation, NBIA, and parkinsonism.
Further Reading
About Renthera
Sepi-24 for Neurological Disorders
Small molecule program aiming to provide homeostasis for glutamatergic dysregulation signaling found in
diseases such as Alzheimer's, schizophrenia, and brain tumors
Further Reading
About Renthera
KBP990 Cardiovascular and Material Science Breakthrough
The proliferation of anti-opiates, such as Narcan have been a weapon against a generation of people lost to this addiction, however, rarely is Narcan self-administered and often, health services arrive too late to provide life-saving care.
Target and Approach
KBP 990 is a transdermal smart patch, powered by the wearers own electrolytes that monitors hemoglobin saturation of oxygen in real-time. When the oximeter detects the wearer’s percentage of hemoglobin saturation to be less than or equal to 90%, which is an indication of impending respiratory failure in otherwise healthy individuals; the microcontroller initiates the subcutaneous administration of the opioid antidote, naloxone at 2mg SC.
Results by Design
Currently, 187 people die everyday in the US from an opiate overdose, KBP990 will help bring that number closer to zero
Bar-IS2- Rare Fungal
Fungal infections kill 1.5M people per year, as many as tuberculosis or malaria. These numbers are projected to increase with an increased population having compromised immune function. Invasive fungal infections have a mortality rate often exceeding 50%. 90% of deaths are caused by the following forms: Candida, Cryptococcus and Aspergillus. Currently, only antifungal drugs are approved for the treatment of systemic infections and resistance. One of the main limitations of the azole class of antifungals is the clinically significant drug-drug interactions experienced due to inhibition of off-target CYP450.
Target and Approach
An investigational arylamidine, structurally similar to a class of aromatic diamidines that includes pentamidine. BarIS2 causes the collapse of fungal mitochondrial membrane inhibiting the respiratory chain complexes in whole yeast cells and isolated yeast mitochondria, which is key for selective disruption of yeast mitochondrial function and antifungal activity.
Results by Design
In vitro activity, of BARIS2 was far superior to the activities of fluconazole, voriconazole, micafungin, and AmB. T-2307 was active against Aspergillus spp.
Further Reading
Gödel Software
Gödel is Renthera's molecular simulation software.
Approach
Protein preparation, ligand docking, collaborative design, and other fundamentals of small molecule drug discovery. Molecular and periodic quantum mechanics, all atom molecular dynamics, Computational target analysis fully integrated into a seamless informatic software interface.
Results by Design
KBP990’s drug patch cellular membrane of zirconia and cerium oxide was designed on the Gödel platform. The patented wearable battery technology harnessing the sodium channel ions of the wearers own electrolytes is revolutionary in the implant/wearable space.
The targets for TG297 and Sepi-24 were all modeled on the Gödel gene editing platform.
Further Reading: