New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’

Though GNN has reported that several sickle cell disease patients have already been successfully treated with CRISPR gene editing technology, a new method of addressing this debilitating condition has been successfully demonstrated.

Braden Baptiste has had sickle cell crises since he was a toddler. It left him missing large chunks of school, left him repeatedly hospitalized, forced him to get replacement hips, and even threatened his life when his blood cells, through forming sickle shapes, had trouble reaching his heart.

“Now I’m going to the gym every day, doing cardio and weight lifting,” said Baptiste, the 20 year old recipient of a more precise gene editing protocol called base editing.

Put simply, wherein CRISPR involves the artificial facilitation of a break across one or both strands of DNA, base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.

Boston Children’s Hosptial, where Baptiste was treated, describe base editing as a spell check. Using the targeting ability of a CRISPR product, physicians were able to reach a single base. There are four: adenine (A), cytosine (C), guanine (G), and thymine (T).

The experimental pharma company Beam Therapeutics has a base editing treatment that chemically transform one base into another—changing C to T, or A to G, for example. These small changes can correct a point mutation, silence a disease-causing gene, or help activate a specific gene.

In this case, it was correcting for a mutation in the hemoglobin that’s part of an ancient human adaptation to protecting the body from the malaria parasite and which has the occasional side effect of causing red blood cells to change into sickle shapes and get caught in the blood stream.

“Sickle cell disease has a broad spectrum of severity, and the severity and frequency of complications can wax and wane,” Matthew Heeney, MD, Baptiste’s long-time hematologist at Boston Children’s Hospital said in a news release.

“Unfortunately, Branden was quickly acquiring many of the chronic complications of sickle cell disease, including organ dysfunction affecting his kidneys, lungs, joints, and eyes.”

Baptiste became eligible for an experimental trial of base editing called BEACON. By October 2023, after a year of tests to ensure he was physical capable in his diminished state to handle the procedure, it began with a sample of his blood stem cells.

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These were then transferred to a separate facility where the base editing would take place. Using chemotherapy, his team then killed off all the diseased blood stem cells in his bone marrow, after which he was ready to receive his own stem cells back in November.

Boston Children’s Hospital write that while waiting for the effects of the infusion to kick in, Baptiste watched all eight seasons of Netflix’ legal drama, Suits. 

Apart from being seemingly cured, Baptiste was back at home in time for Christmas Eve to everyone’s surprise.

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“In my opinion, I’m perfect. I never felt fine before—before, ‘fine’ was moderate pain I could take deep breaths through. Now I’m more than fine. I’m operating in every way possible.”

“I used to always try to exercise, but every little movement would cause joint pain, and exhaustion would also cause pain,” he says. “Now I’m going to the gym every day, doing cardio and weight lifting.”

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