Celenex: Determined Parents Champion Treatment to Save Children’s Lives

The story of Celenex is special.

It starts with two passionate, devoted parents whose daughters were born with Batten disease (also known as Neuronal ceroid lipofuscinosis – NCL), one of a group of rate, fatal, inherited, degenerative disorders of the nervous system with virtually no treatment options, and no cure. Most children with Batten disease lose their vision, their motor function, and then their lives the age of 12.

Driven by devotion and love, these parents, Gordon and Kristen Gray< refused to give up and let their young daughters, Charlotte and Gwenyth, suffer and die. The family needed a miracle. The Grays formed a foundation (raising more than $5MM in less than a year) and searched far and wide to find a treatment to save their daughters. Eventually connected with the ground-breaking work of Drs. Brian Kaspar and Kathrin Meyer at Nationwide Children’s Hospital in Columbus, along with collaborator, Arthur Burghes, Ph.D., at The Ohio State University. The therapy involves injecting a virus that places missing genetic material in brains cells that creates missing proteins to attack the waste that accumulates in the brain of patients with Batten disease.

From Heartbreak to Hope

Celenex was formed as a Nationwide Children’s spinout under the leadership of CEO Samit Varma, a 15-year entrepreneur and venture capital investor, with licensed commercial rights to 10 gene therapy programs, three of which address CLN6, CLN3, and CLN8 Batten disease.

Together the Grays, researchers, and scientists pressed every lever to accelerate pre-clinical research and safety studies for the gene therapy. (On average it takes about 12 years to bring a drug to market in the U.S.)

Imagine their feelings when eight months later (less than a year after their children’s Batten diagnosis) Charlotte was treated. Since then, her sister and more children have been treated. There are more trials ahead and much work yet to be done, but two-years of preclinical data is positive.

Moving to Transform Thousands of Children’s Lives

In September, Amicus Therapeutics (Nasdaq: FOLD) announced the acquisition of Celenex for $100 million plus up to $277 million in development milestones plus sales-based payments.

Amicus Therapeutics CEO John Crowley is also the parent of two children with Pompe Disease, a severe and often fatal neuromuscular disorder. In his quest to save his children’s lives, Crowley became an entrepreneur and co-founded Novazyme Pharmaceuticals. Novazyme Pharmaceuticals was subsequently acquired by Genzyme Corporation, the firm that ultimately produced a therapy to treat Pompe Disease. Crowley credits Genzyme with saving his children’s lives.

Sometimes in the world of biotech, it seems that the stars align. Sometimes that’s because loving parents like the Grays and the Crowleys have moved heaven and earth. Dedicated scientists and researchers at internationally acclaimed institutions like Nationwide Children’s and OSU are not afraid to aim for the moon.