NATIONAL HARBOR, Maryland — One of the biggest drawbacks of gene therapy for rare diseases, besides the price factor, is that it’s generally considered a “one-and-done” that offers no opportunity for redosing as the patient grows.

But Generation Bio, a preclinical biotech company based in Cambridge, Massachusetts, thinks it may have found a way around that.

Doug Kerr, MD, PhD, the company’s chief medical officer, offered his conclusions in a video presented August 25, 2021, at the World Orphan Drug Congress USA 2021.

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He said Generation Bio is investing heavily in a proprietary nonviral genetic medicine platform that focuses on 3 core elements: closed-ended DNA (ceDNA), which enables durable expression of transgenes and expression of large genetic payloads; cell-targeted LNP (ctLNP), which allows redosable delivery to multiple tissues; and enzymatic manufacturing.

Cell and Gene Therapies: The Future of Treatment for Patients With Rare Diseases

The technology offers particular promise for people with hemophilia A, as well as phenylketonuria (PKU), Gaucher disease, Wilson disease, and Stargardt macular degeneration.

“Not everything we hope to do will come to pass. There’s risk and uncertainty. But we believe these core platform elements may enable a new generation of gene therapy,” Dr. Kerr said. “Transgene expression is durable [for] months to years, very much like AAV [adeno-associated virus]. But unlike AAV, it is redosable, so that if expression wanes over time, we could retreat that particular patient and give even longer benefit.”

Importantly, Dr. Kerr said, redosability may one day enable physicians to treat children at the onset of disease, and then additionally treat them as needed throughout childhood, adolescence, and adulthood.


Toward a redosable gene therapy for hemophilia A. Presented at: World Orphan Drug Congress USA 2021: August 25, 2021; National Harbor, MD.