Hope in Focus Makes $300,000 Pledge for LCA Treatments

 In News

The big news just keeps coming: fresh off the rebranding from Sofia Sees Hope to Hope in Focus, we are pleased to announce a pledge to raise $300,000 by 2025 to fund LCA treatments and research. 

“This is a lot of money for a small organization like ours,” Co-Founder and President Laura Manfre said. “It is the biggest commitment we have made yet.”

Individual contributions and donations to the organization go 100 percent toward funding research and LCA therapies, Manfre said. Meanwhile, grant funding and corporate support fuel the organization’s advocacy, outreach, and education programs.  

The news comes in conjunction with the Foundation Fighting Blindness recently announcing the formation of a new company called Opus Genetics that will help move LCA therapy research forward from clinical trials to federally approved treatments for people living with LCA and other retinal degenerative diseases. 

LCA Treatments on the Horizon

For perspective, zero treatments existed when Hope in Focus began in 2014; now five or six LCA treatments are moving forward, along with  LUXTURNA® gene therapy already having earned federal approval in 2017 to help improve vision in people living with LCA2 (RPE65). 

“Getting the research from the lab to the patient community is a huge lift,” Manfre said. “It’s complicated. There are many parties involved from regulatory bodies to industry to investors to the patient community.  It’s a significant big event, and while we have several LCA treatments that look promising for clinical trials in humans, this is a big gap to close.  I’m very excited for Opus Genetics, as it was created to help close that gap.” 

In late September, the Foundation’s venture arm known as the Retinal Degeneration Fund (RD Fund) launched Opus Genetics, a patient-first, science-driven gene therapy company tackling manufacturing obstacles standing in the way of treatments for ultra-rare blinding conditions.

Foundation CEO Ben Yerxa, PhD, said he was excited from the perspectives of the Foundation and the new company. 

“We see a lot of opportunity to develop new therapeutics in this space,” he said. “It starts with early translational research, it starts with genetic testing, and then once we figure that out, it’s time to develop therapeutics.” 

Yerxa, CEO of the RD Fund and Acting CEO of Opus, said he was happy to be working with Hope in Focus.

“We think you’re the best in the business when it comes to creating community, and without that community, those who are affected and the families, we can’t do our job.

“So just thank you for pulling it together and being with us. We’re here for you and we’re rooting for you, and we appreciate everything you do to support us, and stay tuned: More to come.”

The pledge by Hope in Focus marks a milestone in our fundraising efforts to drive LCA research forward.

“I’m very excited for our pledge to raise $300,000, and hope that our donors and supporters will be too,” says Manfre.  Not only will we be able to support early stage research for LCA treatments, but with Opus Genetics, we will actually be able to make an impact in getting those treatments to the people who need them.” 

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