More than 6,800 diseases are classified as rare, meaning each affects fewer than 200,000 people. Collectively, rare diseases are hardly uncommon and affect nearly 30 million Americans. Approved treatments exist for less than 10% of these diseases, despite their impact on longevity and quality of life.
The Duke Clinical Research Institute (DCRI) is now assuming a greater role in national efforts to deliver effective, personalized therapies to patients more broadly and rapidly.
The institute now serves as the Clinical Data Coordinating Center for the National Institutes of Health (NIH)-sponsored Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) clinical trials. The role was announced at the AMP BGTC session during the American Society of Gene & Cell Therapy Annual Meeting in May 2026.
Launched in October 2021, the BGTC is focused on generating a standard operational regulatory playbook using up to six clinical trial test cases to create a pathway toward the commercial viability and sustainability of gene therapies for very rare diseases.
Part of the Accelerating Medicines Partnership, the BGTC brings together the NIH, the U.S. Food and Drug Administration (FDA), life sciences companies, academic institutions, and nonprofit organizations. Managed by the Foundation for the National Institutes of Health, the consortium aims to develop shared platforms, standards, and processes to streamline gene therapy development, with a particular focus on conditions too rare to support traditional commercial investment and conventional clinical trials.
“We are looking forward to working with the DCRI team as the BGTC is initiating AAV gene therapy clinical trials,” said P.J. Brooks, PhD, BGTC co-chair and acting director of the Division of Rare Disease Research Innovation at the NIH’s National Center for Advancing Translational Sciences (NCATS).
About 80% of rare diseases are caused by alterations in a single gene, meaning there is an immense opportunity for gene therapy, which entails replacing or correcting a defective gene. But developing gene therapies for rare and ultra-rare diseases is complex, time-consuming, and expensive.
The consortium focuses on advancing “bespoke” gene therapies through highly tailored treatments for very small patient populations, often with urgent, unmet medical needs. By shifting to a more scalable platform approach, it aims to make gene therapy development faster and more accessible.
The BGTC aims to address these challenges by creating shared infrastructure and standardized approaches, such as vector development, manufacturing, and regulatory pathways, to reduce duplication and accelerate trials across multiple diseases.
The DCRI’s role in the consortium began with the Multiple Sulfatase Deficiency (MSD) Gene Therapy Trial, which recently received FDA clearance for the Investigational New Drug (IND) Application for AAV9/SUMF1. The institute is currently leading trial startup activities for the MSD Gene Therapy Trial is providing clinical operations, data management, protocol execution, and data analysis support to the IND sponsor, the National Center for Advancing Translational Sciences.
These complex, high-touch studies require a markedly different approach from large-scale, multicenter trials, according to DCRI faculty member Chris Lindsell, PhD, who will serve as the data coordinating center lead.
“This is a different kind of research environment than traditional large trials,” Lindsell said. “These are small sample size, high-intensity trials with very high risk, but deeply engaged populations and physicians. There are also significant social and behavioral challenges that differ from mega trials.”
BGTC trials often involve small numbers of patients, many of them children with rare or ultra-rare genetic conditions. With so few eligible participants, every aspect of study design and execution must be carefully tailored to the unique realities of rare disease research.
“Every study or platform needs to be designed and executed for an intended purpose, which means we have to solve different problems,” Lindsell said. “For example, recruitment is completely different in studies of rare disease.”
The DCRI will build processes to coordinate trial activities across sites and institutions while streamlining how data are collected, analyzed, and used to inform decisions.
“This is an opportunity for the DCRI to develop the research framework that could ultimately provide options for millions of people who have gone without effective treatments for far too long,” Lindsell said. “A consortium like this doesn’t just change how trials are run; it changes what’s possible for patients and families.”