A team of veteran scientists from elite institutions has come together to form a new center dedicated to the crafting of personalized genetic medicines, adding to the growing list of similar efforts in the wake of Baby KJ’s groundbreaking custom CRISPR treatment.
The nonprofit Center for Therapeutic Genetics (CTG) will have a small physical space at the Broad Institute of MIT and Harvard, Director Winston Yan, M.D., Ph.D. told Fierce, but its work will ultimately be spread across the Broad, Boston Children’s Hospital and The Jackson Laboratory in Maine.
Yan, who previously co-founded and spent a decade at Arbor Biotechnologies, kicked off the new endeavor with a cold email to Harvard biologist David Liu, Ph.D., who pioneered the next-gen gene editing techniques called base editing and prime editing.
“This was before the announcement of Baby KJ—this was before all the regulatory announcements,” Yan recalled. With all the progress being made in gene editing at the time, he said, “it felt not right to just let it continue” without taking action to bring new therapies to patients.
Yan and Liu were soon joined by other trailblazing scientists who had also realized that the time had come to scale up personalized medicine for infants born with rare genetic diseases. This includes Timothy Yu, M.D., Ph.D., who spearheaded the development of the first-ever custom genetic therapy, an antisense oligonucleotide, back in 2018, and Cat Lutz, Ph.D., who leads Jackson Lab’s efforts to develop animal models for rare diseases.
CTG’s ultimate goal is to provide a haven where patients with all manner of rare disease can be treated with a standardized procedure, not unlike surgery. This new field, while still in its infancy, has been dubbed interventional genetics.
“If the disease is a genetic mutation, and we have the means to, in a clinically responsible way, safely deliver an edit to correct that mutation to the target organ, then it allows one to begin thinking about that procedural approach,” Yu told Fierce. “Instead of segregating and siloing” genetic conditions into 8,000 different rare diseases, he added, this approach allows us to “re-bucket our definition of disease.”
The CTG founders all recognize that dream is still far off, and for now are leveraging new funding from ARPA-H to focus on a more manageable goal: developing custom gene therapies for severe forms of childhood epilepsies.
“Most children with rare genetic diseases don't make it to kindergarten,” Lutz told Fierce. “There's no meeting your first-grade teacher. And a lot of these are involving the brain and central nervous system and epilepsy.”
The new center will have its own laboratory space down the line, Yan said, but will largely function much like the virtual biotechs that are becoming more common in today’s day and age. The lightweight CTG team will nimbly develop custom therapies for epilepsy patients and collect data while in discussion with regulators. Securing insurance reimbursement for CTG’s work is key, Yan explained, as that is what will ultimately support the center financially in the long term.
While the FDA has put forth a new pathway for bespoke genetic medicines to secure approval, Yan wondered whether approval is needed at all.
“Approval is one end goal,” he said. “Another one is just reimbursement for the actual activities that you do.”
CTG joins a suite of other efforts that have cropped up to capitalize on the excitement of Baby KJ’s custom treatment last year. That includes startup Aurora Therapeutics and the Center for Pediatric CRISPR Cures, both backed by CRISPR co-inventor Jennifer Doudna, Ph.D.