Delaware Doctors Work to Reverse Genetic Disease

ORIGINAL SOURCE: Delaware Today

Delaware researchers and physicians are involved in precision medicine in general and genetic therapy specifically—especially a gene-editing technique known as CRISPR, which allows scientists to perform microsurgery on the body’s DNA.

This past June, KJ Muldoon, less than a year old, made medical history in Philadelphia as the world’s first patient to be treated with personalized gene-editing therapy, at the Children’s Hospital of Philadelphia and Penn Medicine. Muldoon inherited two mutated genes that prevented the breakdown of ammonia, which could have destroyed his liver.

Eric Kmiec, chief science officer at ChristianaCare’s Gene Editing Institute and CEO of the institute’s spinoff company, CorriXR Therapeutics, is one of a cadre of Delaware researchers and physicians involved in precision medicine in general and genetic therapy specifically—especially a gene-editing technique known as CRISPR, which allows scientists to perform microsurgery on the body’s DNA.

“We were all very excited for the baby’s family and for the great demonstration of how the technology works in humans—that it’s safe for patients and that it can reverse genetic disease. …It’s especially relevant for those of us studying rare diseases and cancer care,” he says. “The exciting thing is that CRISPR can be used to guide therapeutics to certain parts of the body and put them directly where they are needed. We’re developing them for every organ.”

The process is what Nicholas J. Petrelli, M.D., medical director of the Helen F. Graham Cancer Center and Research Institute at ChristianaCare, refers to as “personalized medicine,” where even patients with the same type of cancer, the most-prominent CRISPR therapeutic target, may still be treated individually.

In the case of CorriXR, Kmiec and his team are developing products to be used as adjunct therapy in cancer patients by knocking out genes that hinder existing cancer treatments from working effectively. The process, if successful, should also lessen the serious side effects of broad-spectrum chemotherapy.

Kmiec says the company is currently working on two fronts that will hopefully allow clinical trials to begin in 2026. “The FDA first wants you to be able to show you can manufacture the product before entering clinical trials,” he says, “and we are now working on signing a contract with a CRISPR drug manufacturer.”

Additionally, CorriXR held a technology conference this past June to get input from key leaders in the field. “The feedback we got was [that] they thought we should expand use in clinical trials to a basket of cancers, including tumors of the head and neck, skin, and pancreas,” he adds. “The universal opinion was that ‘you guys are tumor agnostic.’”

Another Delaware company, Cellergy Pharma, is also in the early stages of developing a gene therapy, called CAR T to fight uncontrollable severe allergic asthma and severe food allergies, says CEO Chris Cano. “We’re also developing a universal CAR T in oncology and working on a new antibody therapy.”

Although baby Muldoon was the first patient to receive gene therapy, the hope is that gene-related therapy will become commonplace in the near future.