Sangamo Announces ASGCT 2024

Areas of Insight

Long-Term Medical Monitoring

Long Term Monitoring

Patients who receive a traditional prescription medicine are monitored by a physician for as long as they take the treatment. If or when a treatment course ends and a patient stops taking the medicine, it is no longer necessary for their physician to monitor the impact on a patient.

The treatment period for genomic medicine is very short. A patient may receive just one gene therapy treatment administered through an intravenous infusion, but the effects are expected to last in the body for years. For this reason, genomic medicine needs to be monitored for a longer period, even though it is intended to be a one-time treatment

Current guidance from the U.S. Food & Drug Administration on the design of long-term follow-up after the administration of human gene therapy products7 suggests follow up timeframes of 5 to 15 years, depending on the type of treatment being studied.

As one component of a bioethical framework to guide the future of genomic medicine, a medical monitoring system must be established to ensure patients receive long-term monitoring to evaluate the safety and durability of genomic therapies.

Long-Term Medical Monitoring Requires Unique Approaches

We cannot overstate our gratitude to the patients who volunteer to participate in Sangamo- sponsored clinical trials and consent to long-term follow up and monitoring. As this technology progresses, it will take ongoing collaboration with all stakeholders to remain nimble and address potential challenges to effectively conduct ongoing medical monitoring. To this end, a new bioethical framework should acknowledge the lasting nature of genomic medicines vs. traditional therapies and identify pathways for medical monitoring for patients who receive a genomic therapy.

Our first step comes with informed consent and the diligence to explain the complex science to patients, including the potential benefits and risks and nature of the lifelong commitment prior to agreeing to participate in a clinical trial. The success of a trial and the science is dependent on the ongoing and active partnership of the patient, their loved ones, and health care providers.

Circumstances such as a patient moving out of state, or the retirement of a treating physician will continue to pose challenges with ongoing monitoring. We must identify paths for the data to follow the patient, regardless of changes in proximity to treatment providers or milestones in life.

In partnership with rare disease patient advocacy organizations, we are looking at the possibilities of using real-world evidence as a mechanism for long-term medical monitoring. This could include observational studies, patient registries, or databases to track follow-up, in addition to ongoing conversations with both patients and providers to support understanding related to long-term monitoring.

At Sangamo, we prioritize our relationships with patient advocacy organizations through a dialogue to ask questions, listen to the needs of patient communities and incorporate the patient voice throughout our development process.

From a bioethics perspective, we have legal and regulatory obligations we must meet. But as an individual pharmaceutical company addressing unmet needs and replacing today’s treatments with tomorrow’s cures, we want to do more than what is required – we want to do what is right for the patients we serve.

An example of a patient registry that could support long-term medical monitoring for rare diseases is the National Organization for Rare Disorders (NORD) IAMRARE Natural History Study Patient Registry. This registry program brings rare disease communities together and collects data which could be used to study interventional outcomes, support the design of clinical trials for new treatments and improve the quality of life for patients.
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A Patient’s Perspective:
Parkinson’s Disease

Parkinson’s disease is a neurodegenerative disease marked by progressive symptoms including slow movement, rigidity, unstable posture, tremors, loss of smell, sleep disorders, and neuropathic pain. In the US, Parkinson’s disease impacts more than one million patients, with 50,000 newly diagnosed patients each year. There are treatments that target some of the movement symptoms, but these therapies don’t slow the underlying disease progression.

In his late 30s, one patient started to notice small, almost imperceptible changes in his movement. His physician ran tests and mentioned Parkinson’s, but both patient and doctor dismissed the possibility. He was too young and healthy; he didn’t look like a typical Parkinson’s patient. Additional tests and an MRI followed, and a neurologist delivered the devastating Parkinson’s diagnosis.

Staying engaged and finding support has helped him come to terms with the disease and how it could progress in the future. He’s a member of an early onset Parkinson’s group, finding comfort in others who share his concerns and questions about how the disease has impacted their lives.

A Patient’s Perspective: Parkinson’s Disease
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Area of Insight:

Patient Informed Consent

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Area of Insight:

Benefit vs. Risk

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Area of Insight:

Long-Term Medical Monitoring

Area of Insight:

Health Information

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