Gene Therapy
New therapeutic strategy Diabetic Retinopathy Treatment
ABOUT GENERx
We’re a clinical-stage drug platform company focused on treating Sickle Cell inflammation and Pain management. Our mission is making a significant impact for patients and society through a safe and oral medication for Sickle’s cell disease and for people with rare diseases
GeneRx, Inc. has developed the first and only Sickle Cell product that safely reaches therapeutic levels without GI-side effects creating treatment possibilities in many indications.
Our Mission
GeneRx is Flagship Biotech for Sickle Cell SC 411 Phase 3 using Artificial Intelligence (AI) in drug discovery for rare diseases biotechnology company based in Boca Raton, Florida.
GeneRx is focused on developing and commercializing pharmaceutical products for patients living rare life-threatening diseases.
Brain and Heart
new therapeutic strategy in the treatment
BRAIN TUMORS
Meningiomas are the most common tumors of the central nervous system, where the incidence is around 25% of all primary brain tumors. The optimal treatment is represented by total resection accompanied by the removal of the dura mater and bone when infiltrated by the tumor.
X-Linked and CPVT Claudin-5
reductions can be independent of connexin-43, a gap junction protein previously reported to be reduced in failing heart samples. Samples are focus on CPVT, and XLMTM. Other cell junction proteins including alpha-catenin, beta-catenin, and N-cadherin are reduced in only a small number of failing samples and only in combination with reduced claudin-5 or connexin-43 levels. We also show that reduced claudin-5 levels can be present independently from dystrophin alterations, which are known to be capable of causing and resulting from cardiomyopathy.
Duchenne Muscular Dystrophy (DMD)
is fatal, progressive, X-linked disorder characterized by lack of dystrophin production leading to skeletal muscle degeneration, respiratory distress, and cardiomyopathy. Nearly all patients who suffer from DMD develop cardiomyopathy by their early 20s. Studies in the Rafael-Fortney lab showed that reduced expression of the tight junction protein claudin-5 at the cardiomyocyte lateral membrane near the extracellular matrix (ECM) is linked to cardiomyopathy
FDA APPROVAL FOR GENE THERAPY
The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease.
Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc this approval "marks another first in the field of gene therapy - both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss."