According to a story from Eye on the Cure, the biopharmaceutical company Ophthotech is hard at work researching and developing gene therapy solutions for a variety of rare retinal conditions. The company is utilizing a multi-pronged approach that includes a “knockdown and replace” method for treating autosomal dominant retinal diseases, retinal gene therapy, and the delivery of over-sized genes for treatment.
Current Treatment Targets
Some of the diseases that Ophthotech is currently working on include Leber congenital amaurosis, Stargardt disease, and retinitis pigmentosa. All of these genetic eye diseases cause progressive visual impairment, and, in the case of Leber’s congenital amaurosis, complete blindness. The decision to focus on rare, genetic, retinal diseases is a bold one, as extensive study will be necessary, but it is also a huge opportunity to fulfill many unmet medical needs; many of these diseases have limited or no disease-modifying therapies.
An Assemblage of Experts
The chief medical officer for the company, Dr. Kourous Rezaei, says that the company has assembled a capable team of experts that specialize in retinal disease; Dr. Rezaei himself is a retinal specialist as well as an ophthamology professor. This concentration of expertise will allow for more valuable input, particularly for challenges like clinical trial design.
Ophthotech is currently focusing on a tried and true method for gene therapy: adeno-associated viruses. However, the company is hoping to develop completely novel therapies that nevertheless use the viruses for delivery. Part of the reason for this choice is that adeno-associated viruses have been firmly established as safe for use in humans, and while there are other methods out there that offer some advantages over viruses, their safety profile is not as strong so far.
The company hopes to develop therapies that are single delivery, meaning that a patient only has to have them administered one time in order to be cured. This helps minimize the quality of life burden for patients with these retinal conditions.
Ophthotech has a strong research basis through its partnerships with a variety of universities. Currently, its candidate Zimura is in phase II clinical trials for the treatment of Stargardt disease. The drug is a C5 inhibitor, a complement factor that, while essential for fighting infections, also can exacerbate the progression of retinal diseases.