Our Science Explained

The Science

For countless patients, having rare and ultra-rare genetic mutations means you’re left behind by the pipelines of established biotech companies who remain focused on treatments that impact the greatest numbers of patients.

But science is evolving—quickly. The technologies are out there to create customized treatments, known as n=1 therapies, that target each patient’s unique genetic error. We’re simply putting them together, today. Because every day counts for patients who are waiting.

How We Are Doing It

A New Model

Our custom drug development process relies on one essential ideal: interdisciplinary collaboration.

We’ve brought together the best-available technologies and brightest minds in the field—researchers, drug manufacturers, and clinicians—all aligned toward one essential goal: saving patients with unparalleled speed through a novel treatment approach.

Steps to Build a Cure

Our researchers then develop cell lines to test and optimize in-vitro (in a dish) the therapeutic strategy, then in-vivo (in animal models). We work closely with the FDA with an emphasis on patient safety and drug efficacy.

Our Collaborators

The Strategy To End Duchenne

As many as 7,000 rare diseases impact up to 30 million Americans. One of them is Duchenne muscular dystrophy, a degenerative condition caused by a mutation on the DMD gene, which is responsible for the production of dystrophin, a protein that helps muscles recover.

Traditional one-size-fits-all therapeutics leave millions behind. The revolution in genomics has given us the technology to target and treat genetic typos with precision so no patient is left behind.

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For patients with Duchenne muscular dystrophy, CRISPR technology offers one of the greatest opportunities to restore the dystrophin protein to muscles by editing the DMD gene. Short for clusters of regularly interspaced short palindromic repeats, CRISPR relies on a complex immune response bacteria use to fend off viruses by cutting their DNA.

Researchers discovered how to harness this mechanism by programming CRISPR to act as “molecular scissors,” deleting or replacing strands of mutated DNA in order to modify gene function. In the case of Duchenne it can be used to restore the ability of the DMD gene to produce dystrophin. This has already been proven by our animal models.

The vision of Cure Rare Disease is to show that this model can work and then drive towards insurance coverage so that all families can benefit.

Customized Therapeutics Webinar

Have more questions about our drug development process?

Watch our founder, Rich Horgan, explain the Cure Rare Disease customized therapeutics approach in a one-hour long webinar.

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Learn More

Take a deeper dive into the science behind curing rare disease—and our approach to make it happen.

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