Gene therapy for long-term decrease of intraocular pressure

Unmet Need

It is estimated that in the US alone, approximately 3 million individuals are living with glaucoma, making it the leading cause of blindness in the US. There currently is no cure for glaucoma, but reducing intraocular pressure (eye pressure) has proven an effective treatment. Reducing intraocular pressure currently requires either invasive laser surgery or medication. Medication is most common, but patient adherence is low due to the need for daily doses. There is an unmet need to develop new medications that are taken less regularly, specifically for glaucoma.

Technology

Dr. Gonzalez and co-workers have developed a gene therapy that reduces intraocular pressure. This technology is intended to treat glaucoma patients and improve patient adherence. Specifically, the inventors have found that increased expression of microRNAs from the miR-146 family can decrease intraocular pressure. In an animal model expressing miR-146a through both adenoviral and lentiviral delivery, sustained intraocular pressure decreases were observed in rats for up to eight months at levels competitve with current treatments. Additionally, no signs of inflammation, loss of visual acuity, or other side effects were observed with this treatment.

Advantages

  • Decreases intraocular pressure in the eye at levels competitive with current marketed medications without the need for daily medication
  • Intraocular pressure decreases ranging from 2.6 to 4.4 mmHg were sustained over 8 months in animal models
  • Demonstrated to be safe in animal models