In a compelling story of brotherly love, familial connection, and the power of entrepreneurial energies, a 28-year old Boston-based entrepreneur, whose brother has Duchene Muscular Dystrophy, launched a biotech nonprofit organization to cure his brother using a novel, personalized, gene-based therapy. He gained the attention of a prominent research scientist in the region, and they now are pursuing a target. A recent Harvard graduate, Rich Horgan, is an inspirational young man.
Trial Site News – Boston’s Cure Rare Disease on the Cusp of CRISPR-based Personalized Treatment for type of Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD)
DMD is a severe type of muscular dystrophy involving muscle weakness—it usually starts around the age of four in boys and rapidly deteriorates from there. Typically, muscle loss occurs first in the thighs and pelvis, followed by the arms. Thereafter, those afflicted have a hard time even standing. Most patients cannot walk by age 12. The disorder is X-linked recessive. About two-thirds of cases are inherited from a person’s mother, while one-third of cases are due to a new mutation. It is caused by a mutation in the gene for the protein dystrophin—important for the maintenance of the muscle fiber’s cell membrane. There is no known cure and affects about one in 5,000 males at birth.
The New Organization: Cure Rare Disease
The organization Rich Horgan founded to find a cure for his brother Terry, Cure Rare Disease, is not even two years old and is already extremely busy. Based in Boston, MA, the organization’s first target is a customized gene therapy treatment for Terry’s combat against Duchene muscular dystrophy (DMD). By January 2020, three other patients were in line as well. According to a report in January 2020, the organization was “on the cusp of a major breakthrough”, according to the recent Harvard Business School graduate.
The organization has secured over $1 million and benefited from the backing of global philanthropist and billionaire Len Blavatnik. Now collaborating with 15 researchers at seven institutions, they are also contributing to FDA policy on individualized medicine in addition to an insurance reimbursement—not to mention a database accumulating data about mice on different Duchenne mutations.