BlueRock Therapeutics announced that the US Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for OpCT-001, an induced pluripotent stem cell (iPSC)–derived therapy aimed at treating primary photoreceptor diseases. This clearance marks the first time an iPSC-derived therapy will be clinically evaluated for this purpose.

OpCT-001 is designed to restore vision in patients with inherited retinal disorders, such as retinitis pigmentosa and cone-rod dystrophy, which affect the structure and function of photoreceptor cells in the retina and can cause irreversible vision loss. OpCT-001 aims to replace degenerated cells in the retina with functional cells

In a press release, BlueRock said it plans to initiate a phase 1/2a clinical trial in the United States to assess the safety, tolerability, and impact on visual function in patients with these conditions. “This is a major milestone for BlueRock,” said Amit Rakhit, the company’s chief development and medical officer. “We believe OpCT-001 has the potential to significantly impact the lives of patients with limited treatment options.” The trial will evaluate several dose levels of OpCT-001 and will enroll participants at multiple sites across the US.

BlueRock Therapeutics is a wholly owned, independently operated subsidiary of Bayer AG.