Category: ARC News

Johnny Rosa
Marketing & Communications Manager
Amyloidosis Research Consortium

ASPIRE member company and ARC employees at this year’s annual ASPIRE meeting in April.

Improving outcomes for people living with amyloidosis takes more than good therapies. It takes a system that recognizes the disease early, connects patients to the right care, and reaches the communities most often left behind.

Organizations working on their own can make progress. But in a rare disease with a small patient population, limited resources, and stubborn systemic gaps, working in parallel means duplicating effort, missing connections, and leaving the hardest problems to no one in particular.

ASPIRE was built on a different premise.

When the life sciences companies working on amyloidosis come together, in a shared space and focused on shared goals, the whole field moves faster.

Those are the needs ASPIRE was built to address.

What is ASPIRE?

ASPIRE stands for Amyloidosis Stakeholder Partnerships for Impact, Reach and Equity. ARC founded it in 2021 as an industry collaborative. The idea was straightforward. Bring leading amyloidosis companies into one room, in a non-competitive space, and point their shared resources and expertise at the problems they all care about.

Today ASPIRE consists of nine member companies: Alnylam, AstraZeneca, BridgeBio, Intellia, Ionis, Janssen, and Pfizer. They fund the work through membership dues, and ARC serves as the neutral convener, independent of any member company, keeping the work focused on community benefit rather than any individual organization’s interests.

The mission is to power inclusive, accessible solutions alongside the broader amyloidosis community, so more patients live longer and live better. The work centers on three key categories of diagnosis, care, and health equity.

Why create ASPIRE?

Rare disease creates unique challenges. A low patient population, scattered across the country, limited funding from outside sources, the same barriers showing up again and again. When every company chases those barriers alone, effort gets duplicated and progress lacks efficiency.

Because ARC is a nonprofit accountable to the patient community, not to any member company, it is uniquely positioned to bring ASPIRE’s member companies together around shared goals and ensure the work stays centered on patient need, not commercial interest.

Shared problems get solved faster when people stop working in silos. Through ASPIRE, we can pool data, share expertise, and work together on the things too big for any one group.

What makes ASPIRE unique?

Plenty of organizations talk about collaboration. ASPIRE requires organizations that normally work independently to build something together, and to share ownership of the outcome with the patient community.

Projects run through three working groups, each led jointly by two member companies and tied to an Executive Steering Committee. Members propose projects, vote on priorities, and get to work. Together, these working groups are creating an impact.

“We’re not just collaborating. We’re accelerating real progress for the patients and families counting on us.”
— BridgeBio

ASPIRE’s three working groups

The proof is in the output. Each group turned its focus into real results last year.

Coordinated Care

This group focuses on improving access to care for patients who live far from a specialist center. A key achievement has been the launch of Amyloidosis Project ECHO, a virtual learning program that uses real patient cases to connect expert clinicians with community providers share expertise and understanding. After the first few sessions, the program has already reached healthcare professionals across 8 countries.

The group also recently published their best practices survey findings in the Clinical Medicine Insights: Cardiology journal. The survey examined best practices and barriers to amyloidosis care. Findings from the survey were shared with national audiences at the American Association of Heart Failure Nurses meeting and the ASCO annual meeting.

Diagnostic Pathways

Amyloidosis often takes one to three years to diagnose. Symptoms look like other conditions, awareness is low, and there is no single test. This group works to shorten that diagnosis timeframe.

In 2025 the group published a narrative review in the Journal of the American Heart Association on using AI with ECG and echocardiography to raise suspicion of cardiac amyloidosis earlier. The paper landed, being read thousands of times and cited multiple times by healthcare professionals.

It followed with a white paper on putting AI to work responsibly in cardiac amyloidosis, shaped by input from clinicians, technologists, and patient advocates. The group focused their priorities on the diagnostic journey of amyloidosis patients. They launched a claims analysis project to find where in the country diagnosis and treatment fall behind, plus a multiyear effort to trace the real patient path from first symptom to answer.

Health Equity

This group looks at who gets left behind, and why. In 2025 it ran an in person focus group in Cincinnati to hear directly from Black Americans living with amyloidosis, the kind of voice that doesn’t make it into rare disease research enough. A manuscript sharing those findings is on the way.

The next study widens the lens, expanding beyond Cincinnati and examining the black ATTR experience across different geographical regions.

What comes next?

Momentum has stayed high in 2026. One manuscript is accepted and three more are underway. Project ECHO keeps adding sessions, with more frequent programming planned. The health equity group will build on its existing work, broadening its focus to include additional underserved and underrepresented patient populations.

Why it matters

Patients do not experience amyloidosis as a market. They experience it as a long road to a diagnosis, a search for someone who understands the disease, and a hope that the system will help them. And because ARC holds the convener role, answerable to the community and not to any funder, that work will always be driven by what the field needs most.

Five years in, that work is only picking up speed.

Learn more about ASPIRE and read the 2025 Impact Report at arci.org/aspire. Companies interested in joining can contact Megan Glynn at mglynn@arci.org.