Until recently, “bespoke” meant one thing to me: a custom suit from London’s Savile Row. They’re beautiful—and expensive—so I assumed I’d never own anything bespoke.

A tailored approach

I was wrong. CIRM is now part of a bespoke effort far more important than clothing. This group aims to create tailor‑made gene therapies for rare diseases.

It’s called the Bespoke Gene Therapy Consortium (BGTC). Fair warning: many acronyms ahead. The BGTC is part of the Accelerating Medicines Partnership (AMP). AMP is a public‑private collaboration between the NIH, the FDA, and multiple organizations, including CIRM.

The Foundation for the NIH manages the program. Its goal is to develop platforms and standards that speed the development of customized gene therapies for millions of people with rare diseases.

The importance of rare disease research

Why is this needed? There are an estimated 7,000 rare diseases affecting 25 to 30 million Americans. Some conditions affect only a few hundred or even a few dozen people. These small patient groups rarely have the resources to fund research. Yet many rare diseases stem from a single‑gene mutation, making them strong candidates for bespoke gene therapy.

Today, each disease program often builds its own approach. That work is slow and costly. The BGTC believes a standardized method could create a template that speeds development and lowers costs across many rare diseases.

“At CIRM we have funded several gene therapy projects that treat, and even cure, rare diseases like severe combined immunodeficiency,” says Dr. Maria T. Millan, CIRM’s President and CEO. “But even with our resources, we can only go so far. Partnering with the BGTC allows us to join a larger effort that can advance the field, remove barriers, and drive breakthroughs for many rare diseases.”

Gene therapy

Gene therapy works by finding the genetic defect causing a disease and delivering a normal copy of that gene to the right tissues. The big question is: what is the best way to deliver it?

The BGTC is focusing on adeno‑associated virus (AAV) as its delivery vehicle. AAV gene therapy already works for Leber congenital amaurosis, retinitis pigmentosa, and spinal muscular atrophy. The consortium will test multiple AAV‑based approaches, moving projects from basic research through clinical trials. This collaboration will generate shared knowledge on AAV manufacturing and regulatory pathways. That template can guide future gene therapies for other rare diseases.

CIRM will identify rare‑disease gene therapy programs in California that qualify for the AMP BGTC. CIRM funding will support their IND‑enabling studies, manufacturing, and clinical trial activities.

“This knowledge‑network model aligns perfectly with our mission to accelerate transformative regenerative medicine for a diverse California and world,” says Dr. Millan. “Small, isolated patient groups cannot fund this research alone. By pooling resources and expertise, the consortium ensures that work we support here can ultimately help patients everywhere.”

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