Sep 9, 2024

Stem Cell Therapy Gets FDA Approval for Clinical Trials

FBC-funding Contributed to Therapy Development

Major research breakthroughs rarely happen overnight. They are the result of years, and sometimes, decades, of work. One discovery leads to another which leads to another — sometimes culminating in a clinical trial. This was the case for Dr. David Gamm of the University of Wisconsin who received a Restore Vision 20/20 grant from Fighting Blindness Canada (FBC) in 2018.

BlueRock Therapeutics LP recently announced the US Food and Drug Administration (FDA) cleared their investigational new drug application (IND) for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. In other words, the cell replacement therapy is going into clinical trial for treating primary photoreceptor diseases like retinitis pigmentosa — and it’s the first such clinical trial.

With FBC funding, Dr. Gamm, optimized the stem cell development and delivery process and demonstrated that stem cells grown in lab can reach out, integrate and function within retinal tissue.

Photoreceptor cells are found in the eye’s retina and are responsible for converting light into signals that are sent to the brain. The death of photoreceptor cells leads to vision loss in many retinal diseases, such as inherited retinal diseases (e.g. retinitis pigmentosa or Usher syndrome) or age-related macular degeneration.

While gene therapies may prevent the death of photoreceptor cells, thereby slowing down vision loss, they cannot restore retinal degeneration. This is where stem cell therapy comes in. New cells such as photoreceptors cells grown in a lab are transplanted back into the eye hopefully replacing cells that have died or been damaged.

In 2022, Dr. Gamm’s team showed that lab-grown photoreceptors do respond to light and extend out “cords” or axons towards other retinal cells in the organoid. Then, in 2023, his team showed that cells, including photoreceptors, did make new connections.

On Sept. 3, 2024, BlueRock Therapeutics, a clinical stage cell therapy company and a wholly owned, independently operated subsidiary of Bayer AG, announced the FDA approved clinical trials for its IND based on Dr. Gamm’s work.

“This is a major accomplishment for our collaborative team of academic and industry scientists and clinicians. It is also the culmination of nearly a decade of concerted effort to develop an iPSC-derived photoreceptor replacement therapeutic. Importantly, multiple aspects of this work (and next-generation products) have been inspired or advanced via support from Fighting Blindness Canada,” he says.

You can learn more about FBC-funded research by clicking here or make a donation to fund more projects like this.