In a world first, Immusoft gives engineered B cell in a clinical trial, starting new phase of 15-year odyssey
15 Dec 2023
Cell TherapyPhase 1ImmunotherapyGene Therapy
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Source: FierceBiotech
The cell therapy is designed to produce the enzyme alpha-L-iduronidase.
Immusoft has administered an engineered B cell to a human for the first time, marking a milestone in its long-running quest to create a new type of medicine.
The West Coast biotech set up shop in 2009 and made headlines five years later when it received money from Tim Draper, a venture capitalist involved with companies from Hotmail to SpaceX, and Peter Thiel’s FF Science. But progress has been slower than hoped. In 2013, then-CEO Matthew Scholz spoke of skipping primate testing and going “directly into humans ... in the next couple of years, hopefully.”
While that comment proved optimistic, Immusoft has ridden out multiple tricky periods for the biotech sector and lulls in the cell therapy hype cycle, landing a Takeda deal along the way, to finally reach the promised land. Friday, the biotech said it had dosed the first patient with its engineered B cell candidate, ISP-001.
The cell therapy is designed to produce alpha-L-iduronidase, an enzyme that is lacking in people with the rare genetic disease mucopolysaccharidosis type I. Immusoft was able to dose a patient with the therapy without providing preconditioning or immunosuppression, giving its B-cell platform a potential advantage over gene modified stem cells and viral vector-based delivery.
“A nonviral methodology for engineering cells, such as ISP-001 that does not require myeloablative conditioning, could be of tremendous advantage for patients with rare disorders,” Paul Orchard, M.D., principal investigator in the phase 1 trial, said in a statement. “There is a clear unmet need in providing safer and more effective therapeutic modalities for these patients.”
The study marks the next phase of efforts to show if engineered B cells are a safer and more effective modality. As with any new approach, there are unresolved questions about the potential for unintended harms. It is possible for genome editing to activate genes that promote cancer, for example. Regulators are still learning about the safety of T-cell therapies, while B-cell therapies are years behind.
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