4DMT is building a deep pipeline of gene therapy products delivered by routine intravitreal injection for patients with rare diseases of the retina. Intravitreal injections are used thousands of times daily in the US and the rest of the world to deliver treatments for wet AMD, including for blockbuster products such as Eyelea and Lucentis. In contrast, gene therapeutics derived from 1st generation AAV vectors (e.g. AAV2) have not been shown to efficiently treat the retina following intravitreal injection; these agents are instead being delivered by subretinal surgical injection (e.g. Luxturna, Spark Therapeutics, ONCE; NSR-REP1, Nighstar Therapeutics, NITE).
4DMT retina product advantages are designed to include the following:
Retina Product 4D-110 for Choroideremia Patients: Intravitreal Delivery
Choroideremia (CHM) is an X-linked monogenic recessive disease (caused by mutation of the REP-1 gene). Symptoms generally appear in the patients’ teen years, starting with reduced night vision and progressive loss of peripheral vision. Eventually, patients will lose visual acuity and progress to blindness. No effective treatments are approved for the patients with this devastating disease.
Our product for CHM patients 4D-110 will be administered by intravitreal injection, and is designed to result in widespread expression of the REP-1 protein within cells in the retina. In contrast to other AAV gene therapy agents for CHM patients, which are administered by subretinal surgical injection (e.g. those in development by Spark Therapeutics and Nightstar Therapeutics), 4D-110 is designed to treat the entire retina, and to treat patients with all stages of the disease.
Retina Product 4D-125 for a 2nd Rare Retinal Disease: Intravitreal Delivery
Our product for these patients will be administered by intravitreal injection, and is designed to result in widespread expression of the therapeutic protein within cells in the retina. The target patient population for this product will be disclosed at the time of clinical trial initiation.