Organization Looks To Help Families Impacted By Rare Diseases

STAMFORD, CT — Cure Rare Disease, a nonprofit biotechnology research organization, will hold a hybrid in-person/virtual fundraising event on June 5 at The Village in Stamford to celebrate progress in developing customized therapeutics and to raise funds to support patient testing programs. This will be the inaugural event at The Village.

The organization, which is based in Boston, Mass., was formed three years ago with researchers and clinicians working across the country to tailor medicines and customize therapeutics based on an individual's genetic mutation.

Cure Rare Disease has recently worked with four Connecticut families who each have a boy who has been diagnosed with Duchenne Muscular Dystrophy (DMD), a rare and fatal disease that weakens and limits muscle function over time. The families are located in Greenwich, Darien, Ridgefield and Branford.

For Cure Rare Disease founder, Rich Horgan, the mission to help families hits home.

"We exist to ideate, develop and treat patients who are impacted by rare diseases that are too rare to attract traditional drug development interest. The journey is inspired by my younger brother, Terry, who has a rare mutation of DMD," Horgan said. "Terry had no path to treatment, and like many families who are impacted by rare diseases, we were more or less told to go home and love him for the time that we had left, and that's an unacceptable answer."

Cure Rare Disease is approaching the final stages of developing a CRISPR-based gene therapy treatment for DMD. The process involves editing a genome to eliminate a mutation or correcting it in a way that allows a gene to produce a healthy protein, in this case dystrophin. Terry will be the first recipient of the treatment later this year. Horgan hopes the treatment leads to helping more people after Terry.

"It's huge for us. Before I started this organization, my family personally had no shot on goal for a treatment, but with the success we've had in the last couple years, we're right now on the precipice of dosing my brother with what will be a first in human CRISPR therapy for DMD. I think beyond that, it really helps to validate the framework that we've built to then pass other patients through it," Horgan said.

Horgan described it as "an honor" to work with the four Connecticut families.

"I really I think the traditional perspective of the family being subservient to the doctor or researcher is not the framework that we operate on. We treat the family as an equal in the research and development process since they bring an incredible perspective," Horgan said. "Our families work side by side with our researchers and clinicians in this journey to develop custom drugs for each of the families involved in our program."

The fundraising goal for the June 5 event is $250,000. All proceeds will go towards supporting pre-clinical development of other patients down the road. It will be sponsored by Charles River Laboratories and several other companies, and will also feature a live auction and an awards ceremony recognizing clinical and scientific contributions.

For more information on the event, organization, or for details on how to donate, click here.