Graybug is a clinical-stage biopharmaceutical company focused on developing transformative medicines for ocular diseases. Our diversified portfolio is designed to treat vision-threatening diseases of the retina, optic nerve, and cornea, by either maintaining effective drug levels in ocular tissues for long periods of time, using innovative technologies such as injectable sustained-release formulations, or by curing diseases with gene therapies.
Our Gene Therapy APPROACH
Graybug’s gene therapy platform is based on recombinant adeno-associated virus (rAAV) technology designed to deliver a functional copy of the gene that encodes alpha-L-iduronidase (IDUA), an enzyme that breaks down intracellular and extracellular glycosaminoglycans (GAGs), directly to the cornea of Mucopolysaccharidosis Type 1 patients (MPS1) with a single injection.
Our proprietary technologies are designed to allow sustained delivery of pharmacologic agents to the eye in a well-controlled manner to achieve extended duration of effectiveness with the goal of
- Improving patient compliance
- Reducing healthcare burden
- Improving clinical outcomes
Applying Our Proprietary Technologies to Our Pipeline
Click on each compound to learn more.
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