Rare Disease BioScience Nonprofit Will Depart Boston for Greater New Haven

Connecticut tends to lament the departure of businesses when they leave the Nutmeg State and head to Boston.  The most recent, most prominent example is GE (although that ultimately didn’t work out so well).  Now, a potentially groundbreaking bioscience firm is headed the opposite way – to Greater New Haven, specifically Woodbridge.

A clinical-stage nonprofit biotechnology company, Cure Rare Disease, has acquired its first laboratory space in Woodbridge, with plans to relocate its Boston headquarters to the 30,000-square-foot facility at 4 Research Drive early next year, expand its research and development operations, and lease approximately 15,000 square feet to other life science and healthcare firms.

Cure Rare Disease has formed a collaboration with the leading researchers and clinicians across the United States “to develop a customized therapeutics platform, in time to save our loved ones.” Cure Rare Disease develops therapeutics for rare and ultra-rare neuromuscular diseases, such as two rare types of muscular dystrophy, known as Duchenne and Becker.

The nonprofit company points out that “there are over 7,000 different rare diseases impacting over 300 million people worldwide. Drug development for rare disease is challenging. The “one size fits all” approach to drug development is not sufficient. We are determined to change this.”

The nonprofit’s mission is to offer “effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors.”  Among the organization’s researchers and clinicians are three - Kaiyue Ma, Shushu Huang, and Monkol Lek - with connections to Yale Medical School, among a half dozen universities and research partners of Cure Rare Disease in the U.S., Canada and Europe.   

Founder Rich Horgan was inspired to launch the initiative by the life’s experience of his brother.  As described on the Cure Rare Disease website, “From the moment of diagnosis, time was ticking away for my brother… Terry’s time, independence, and mobility continued to slip away with no ability to fight back. The feeling of hopelessness became a never-ending theme in my life and the lives of my family. A family impacted by rare disease only has two options: fight or stand idly by. Standing by wasn’t an option.”

That was two decades ago.  Now, “Cure Rare Disease boasts a therapeutic pipeline of over 19 programs, each being engineered to improve the lives of patients across the globe who are impacted by devastating rare diseases for which there are no treatments.”

Horgan recently reported progress in correspondence with investors: “Over the last few months, we have made significant progress with many of our programs, ranging from our DMD programs to our limb-girdle muscular dystrophy (LGMD) and SCA3 therapeutics,” and predicted “throughout the remainder of 2022, we will be conducting more pharmacology studies and preIND (investigational new drug) meetings than ever before.”

Cure Rare Disease is planning an “Inspire Gala” on November 12 on-site at the facility in Woodbridge, “to celebrate our achievements thus far and look ahead to what's next.”  The fundraising event aims to “demonstrate our collective strength to drive genetic medicines forward for rare disease patients,” according to company officials. 

“Support of the Inspire Gala will directly impact our ability to dose our first patient while advancing next-generation therapeutics for many others. Specifically, proceeds from the event will fund preclinical testing of individualized genetic medicine—next-generation treatments targeted to specific gene mutations—for our next cohort of patients, and will raise awareness about our work across the country.”

Cure Rare Disease is making their presence felt in the New Haven region, even before they arrive. To date, the event has already raised $131,000 towards a goal of $250,000.  Information about the event and tickets may be seen here.