Without fail, David Liu of the Broad Institute gets at least 20 messages every week from desperate parents. Their child, they\u2019ll explain, has an ultrarare and devastating disorder caused by a mutant gene. Can he help them develop a gene therapy?<\/p>\n\n\n\n
Dr. Liu, a gene-editing researcher, answers honestly: Although the science is there \u2014 researchers can edit and silence the genes that cause disease relatively easily \u2014 the system for developing treatments for rare diseases is just not economically feasible. It typically takes years and hundreds of millions of dollars.<\/p>\n\n\n\n
The catchphrase, said Dr. Wendy Chung of Boston Children\u2019s Hospital, is \u201ctoo rare to care.\u201d<\/p>\n\n\n\n
Now, Dr. Liu, Dr. Chung and their colleagues at the Broad Institute, Boston Children\u2019s and the Jackson Laboratory are hoping to change that mentality. On Tuesday, they announced the start of a new nonprofit, the Center for Therapeutic Genetics<\/a>, to develop gene therapy treatments that can be used and reused in disease after disease.<\/p>\n\n\n\n Treatments developed by the center would be viewed more like routine procedures than drugs. In each case, all that would be changed are the instructions to a gene editor. There would be no need to start anew for every patient.<\/p>\n\n\n\n Read it all<\/a>.<\/p> \u2018Too Rare to Care\u2019? A New Center for Rare Diseases Hopes to Change That https:\/\/t.co\/ahZC0LpW1d<\/a><\/p>— Global Health Observ (@GlobalPHObserv) July 21, 2026<\/a><\/blockquote>