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September 8, 2021

Stamford nonprofit teams up with Moderna on rare disease

CLOE POISSON | CT MIRROR A first dose of the Moderna COVID-19 vaccine is given to a senior citizen at a clinic at St. Bernard Church in Vernon.
By Natalie Missakian

Coronavirus vaccine maker Moderna is giving away one of its mRNA-based treatments to a newly launched Stamford nonprofit — with the goal of developing a free treatment for people with an ultra-rare disease.

Moderna and the new Stamford-based Institute for Life Changing Medicines (ICLM) announced on Tuesday they would collaborate on a new mRNA-based therapeutic for Crigler-Najjar Syndrome Type 1 (CN-1). 

The disease is caused by a gene mutation that prevents the body from breaking down bilirubin, a substance made in the liver, and there are just 100 or fewer known cases worldwide. 

Dr. James M. Wilson, who leads the gene therapy program at the University of Pennsylvania’s Perelman School of Medicine, and Alex Karnal, partner and managing director of health-care investment firm Deerfield Management, announced their launch of the nonprofit institute in Stamford last week. 

Its mission is to develop and promote access to drugs that have “transformative potential” but aren’t commercially viable because they treat diseases that affect only a small number of people. 

Crigler-Najjar Syndrome is one of the first diseases the institute plans to tackle. Usually diagnosed in newborns, the condition can lead to the buildup of bilirubin in the body, causing jaundice and damage to the brain, muscles and nerves. Current treatments include as much as 12 hours a day of blue-light therapy for life, or a liver transplant.

Under the agreement announced Tuesday, the Boston-area vaccine maker will license its experimental therapeutic, mRNA-3351, to the institute at no upfront cost, and without any downstream payments. 

The institute will be responsible for developing the drug, and expects to begin human testing in 2022. The therapy works by restoring the missing or dysfunctional proteins that cause the disease, according to Moderna. 

“Ultra-rare diseases are always a challenge for our industry given the very small number of patients who could benefit from the medicine,” Moderna CEO Stéphane Bancel said in a statement. “We decided that rather than charge a high price for the medicine candidate, which is not aligned with our values, we would rather give it away for free.”

Wilson, who is ICLM’s chief scientific officer, said the partnership with Moderna could serve as a model for developing other medicines for people with rare diseases “when traditional business models for drug development fall short.”

The institute is also working on treatments for AADC deficiency, which causes severe congenital Parkinson's disease, and Lesch-Nyhan Syndrome, which causes cerebral palsy and other neurological symptoms, according to the ICLM website. Just 10 babies are born with each condition in the U.S. each year, the institute said.

Contact Natalie Missakian at news@newhavenbiz.com
 

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