Revolutionary Progress in Gene Therapy with Immusoft's Patient Re-dosing of ISP-001 for MPS I
In a remarkable advancement for the field of gene therapy, Immusoft, a clinical-stage company based in California, has achieved a significant milestone with the re-dosing of a patient treated with ISP-001, targeting mucopolysaccharidosis type I (MPS I). This news marks a pivotal moment not only for Immusoft but for the gene therapy community as a whole, as it opens the door to potentially re-administering gene therapies—a feature that has been elusive until now.
On October 7, 2025, Immusoft announced that the re-dosing has been well-tolerated by the patient, who has now received two doses of their innovative therapy, each separated by an 18-month interval. The initial dose was administered during the company’s first-in-human clinical trial, and the patient has exhibited encouraging results, continuing to show benefits from the treatment for over a year. This success raises hopes for extending therapeutic effects well beyond standard treatment durations, making it possible to continuously deliver therapeutic proteins throughout the patient's lifetime.
Sean Ainsworth, the CEO of Immusoft, emphasized the importance of this breakthrough, stating, "The vast majority of gene therapies are not re-dosable with current technologies for various reasons. Our engineered B cell approach allows us to avoid dangerous immune responses typically linked to viral gene therapies, thereby facilitating safe re-dosing."
The innovative technology behind ISP-001 relies on the modification of a patient’s B cells, transforming them into biofactories capable of producing therapeutic proteins. Unlike conventional gene therapies that may require a toxic chemotherapy regimen for administration, Immusoft's method allows for a gentler approach that minimizes the risk associated with re-treatment. This method ensures not only better safety profiles but also improved patient tolerability.
As B cells migrate to and engraft in the bone marrow naturally, they eliminate the need for harsh preparatory treatments seen in traditional gene-modified stem cell approaches. This is crucial as such preparations usually involve significant hospital stays and increased morbidity risk, making re-treatment often impractical.
The first patient re-dosed with ISP-001 received a doubled amount compared to their initial treatment, aiming to optimize therapeutic effects through titration. Alongside this, the second patient in the trial has received a mid-range dose, showing a strong safety profile and continued optimism regarding the treatment’s efficacy. Initial pharmacodynamic results from this second patient are promising, making a strong case for the ongoing eligibility of these therapies.
Dr. Paul Orchard, a leading investigator at the University of Minnesota, noted, "The ability to re-dose is a game-changer that the gene therapy sector has been aiming for. So far, the results from Immusoft's clinical trial are very promising, especially as we’re able to do this without necessitating myeloablation or immunosuppression."
Continuing its mission, Immusoft's initiative is also bolstered by an $8 million grant from the California Institute for Regenerative Medicine (CIRM), which funds various research endeavors in cell and gene therapy in the state. Such support highlights the importance of innovative therapies like ISP-001 that can alter how genetic diseases are tackled long-term.
As Immusoft strides forward, their engineered B cell approach represents a hopeful path not just for individuals with MPS I, but for the broader landscape of genetic therapies aimed at numerous genetic disorders. With the successful implementation of ISP-001, Immusoft is at the forefront of a significant shift in how we approach treatment for rare diseases, potentially redefining the future of patient care in gene therapy.
On October 7, 2025, Immusoft announced that the re-dosing has been well-tolerated by the patient, who has now received two doses of their innovative therapy, each separated by an 18-month interval. The initial dose was administered during the company’s first-in-human clinical trial, and the patient has exhibited encouraging results, continuing to show benefits from the treatment for over a year. This success raises hopes for extending therapeutic effects well beyond standard treatment durations, making it possible to continuously deliver therapeutic proteins throughout the patient's lifetime.
Sean Ainsworth, the CEO of Immusoft, emphasized the importance of this breakthrough, stating, "The vast majority of gene therapies are not re-dosable with current technologies for various reasons. Our engineered B cell approach allows us to avoid dangerous immune responses typically linked to viral gene therapies, thereby facilitating safe re-dosing."
The innovative technology behind ISP-001 relies on the modification of a patient’s B cells, transforming them into biofactories capable of producing therapeutic proteins. Unlike conventional gene therapies that may require a toxic chemotherapy regimen for administration, Immusoft's method allows for a gentler approach that minimizes the risk associated with re-treatment. This method ensures not only better safety profiles but also improved patient tolerability.
As B cells migrate to and engraft in the bone marrow naturally, they eliminate the need for harsh preparatory treatments seen in traditional gene-modified stem cell approaches. This is crucial as such preparations usually involve significant hospital stays and increased morbidity risk, making re-treatment often impractical.
The first patient re-dosed with ISP-001 received a doubled amount compared to their initial treatment, aiming to optimize therapeutic effects through titration. Alongside this, the second patient in the trial has received a mid-range dose, showing a strong safety profile and continued optimism regarding the treatment’s efficacy. Initial pharmacodynamic results from this second patient are promising, making a strong case for the ongoing eligibility of these therapies.
Dr. Paul Orchard, a leading investigator at the University of Minnesota, noted, "The ability to re-dose is a game-changer that the gene therapy sector has been aiming for. So far, the results from Immusoft's clinical trial are very promising, especially as we’re able to do this without necessitating myeloablation or immunosuppression."
Continuing its mission, Immusoft's initiative is also bolstered by an $8 million grant from the California Institute for Regenerative Medicine (CIRM), which funds various research endeavors in cell and gene therapy in the state. Such support highlights the importance of innovative therapies like ISP-001 that can alter how genetic diseases are tackled long-term.
As Immusoft strides forward, their engineered B cell approach represents a hopeful path not just for individuals with MPS I, but for the broader landscape of genetic therapies aimed at numerous genetic disorders. With the successful implementation of ISP-001, Immusoft is at the forefront of a significant shift in how we approach treatment for rare diseases, potentially redefining the future of patient care in gene therapy.
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