Community, connection, collaboration

At Agios, building bonds and cultivating relationships are at the heart of our ability to create life-changing therapies for people with rare diseases.

Patients and caregivers, advocates, healthcare providers, payers, scientific collaborators and research partners. We’re all part of a team that helps bring life-changing therapies to market for people with rare diseases.

We’re committed to building trusted relationships with our extended teammates, each of whom lends mission-critical insight that enables us to discover, develop and deliver therapies faster and with greater success. We simply couldn’t do it without the unique contributions and perspectives of each person.

Our approach
in action

During a strategic advisory meeting with patients and healthcare providers, a patient told us, “We need to replace ‘I’ve created this for you,’ with ‘I’ve created this with you.’” That’s exactly the approach we took with our RISE UP Phase 2/3 clinical study researching an investigational medicine for sickle cell disease. The study protocol, name, and recruitment campaign were developed in partnership with a group of sickle cell warriors from around the world who spent months working with us, sharing incredible insights that informed the study design and set the direction for the communications campaign; they continued to collaborate and work closely with us during every step of the project.

Our hope was to create something together that would be meaningful to the community and represent the warriors’ resilience in the face of challenges, refusal to settle for the status quo, and influential efforts to educate the world about sickle cell and close the equality gap in healthcare. While this is a study sponsored by Agios, it is also a study created by the sickle cell community.

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For patients, advocates, and caregivers

At Agios, people living with rare diseases are at the center of everything we do and every decision we make. Their needs, concerns, input and collaboration are essential to fulfilling our mission: to make a meaningful difference in patients’ lives and fundamentally change the way rare diseases are treated.

If you or a loved one are living with a rare disease, explore more about the ways we can partner together.

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PK deficiency patient support program

We offer a customized, comprehensive support program to help patients and caregivers learn more and navigate the challenges of living with the disease and provide support for accessing medicine. Sign up at and get connected with a dedicated Patient Support Manager.

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Resources for education and connection

We know that reliable information can be hard to come by. We can help. Check out our list of vetted Agios and third-party resources for patients and caregivers.

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Ongoing clinical studies

Our clinical programs explore investigational, oral treatment approaches for rare diseases. Find out more about our programs and talk to your doctor if you’re interested in participating in studies that are currently recruiting.

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Resources for

We recognize and appreciate the significant role that physicians play in the lives of people with rare diseases. They are a cornerstone of the community that treats and supports patients.

With medical information, grant opportunities, educational resources and treatment options, we aim to further their efforts on behalf of patients.

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for partnership

Great science requires great teamwork. By truly listening to and trusting partners, collaborators, healthcare providers, advocates, and patients we are able to identify and pursue the ideas that can make the biggest impact for people living with rare diseases.

Explore our partnership opportunities and learn more about the ways we can work together to make a difference in patients’ lives.

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