JAN 11, 2017
The FDA has granted ‘safe to proceed’ status to Hemera Biosciences’ investigational new drug application for their dry AMD gene therapy candidate, HMR59.
HMR59 uses an AAV2 vector to induce cells in the retina to express sCD59, a soluble version of the naturally-occurring membrane bound CD59 protein that blocks formation of the membrane attack complex (MAC). As the final step of the innate immune system’s complement cascade, the MAC forms transmembrane channels that disrupt cell function, leading to cell lysis and death. Recent studies have linked mutations in genes encoding complement cascade proteins to the development and progression of dry AMD.
Company officials say a phase 1 trial evaluating a single intravitreal injection of HMR59 in patients with advanced dry AMD is slated to begin in the 1st quarter of 2017.
“HMR59 has the potential to slow down or stop the progression of dry AMD with a single intravitreal injection,” said Hemera cofounder Jay S. Duker, MD. “If shown to be safe and effective, this therapy would prove to be a huge advance for a disease that has no FDA-approved therapy.”