Rare disease consortium is good news for NC gene therapy companies

Rare disease consortium is good news for NC gene therapy companies


RESEARCH TRIANGLE PARK – The nearly 30 million Americans who suffer from rare diseases have received some good news.

The National Institutes of Health, the U.S. Food and Drug Administration, and a cadre of pharmaceutical companies and non-profit organizations have teamed up to speed the development of new gene therapy treatments. It’s good news for North Carolina as well, which is home to close to 50 gene therapy and rare disease-focused businesses that provide jobs for several thousand Tar Heel residents.

What’s called the Bespoke Gene Therapy Consortium – or BGTC – was launched a little less than two months ago. It’s part of the NIH’s Accelerating Medicines Partnership (AMP), a public/private collaboration to speed drug development across different diseases. Ten global pharmaceutical companies and half as many non-profit patient organizations, as well as 11 NIH institutes, centers and initiatives have signed on.

The project is managed by the Foundation for the National Institutes of Health, whose mission is to promote biomedical discoveries that improve people’s lives.

Currently there are about 7,000 rare diseases in this country, 5,000 of which are due to genetic factors, according to BGTC. A single damaged gene causes nearly 80% of rare genetic illnesses, leaving millions of patients in the U.S. to suffer without much hope of improvement. Currently, only two of these diseases have FDA-approved gene therapy treatments.

BGTC said most rare inherited diseases stem from a specific gene mutation that is already known, which makes gene therapy a promising solution.

A customized – or “bespoke” – therapy could correct or replace defective genes with functional ones, according to NIH Director Francis S. Collins, M.D., Ph.D. “There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies,” he said.

But development is costly, complex and time consuming. And rare disorders – by definition – affect only a small number of patients. So most pharma companies aren’t willing to invest years of research and millions of dollars to bring a single-disease gene therapy to market, said Joni L. Rutter, Ph.D., acting director of NIH’s National Center for Advancing Translational Sciences.

BGTC hopes to change that paradigm. The consortium wants to start with a common gene delivery vector known as the adeno-associated virus (AAV). It’s considered one of the most effective gene delivery platforms for many human diseases.

The partnership said it will support a series of research projects and clinical trials to create new tools for AAV clinical development and regulatory evaluation. “The BGTC aims to make it easier, faster and less expensive to pursue bespoke therapies in order to incentivize more companies to invest in this space and bring treatments to patients,” Rutter pointed out.

The goal, over time, is to find ways to cut up-front gene therapy development costs, standardize the technology, and make it available for a broader range of diseases. The BCTC program will create a universal set of analytical tests to speed up gene and vector manufacturing. And the consortium will look for ways to streamline the regulatory framework.

“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them the most,” said Peter Marks, M.D., PhD., director of the FDA’s Center for Biologics Evaluation and Research.

The consortium members will contribute about $76 million over the next five years to back its projects. BGTC said it will fund research for between four and six clinical trials – each focused on a different rare, single-gene disease for which no gene therapies or commercial programs currently exist. Three disease areas are targeted: Lupus, Alzheimer’s and Type 2 diabetes.

“People with terrible genetic disorders are in dire need of solutions,” Collins said. “The BGTC promises to transform the field of gene therapy so we can treat, or even cure rare diseases for which no current therapy exists.”

North Carolina, with its substantial biotechnology and academic resources, has become a prominent incubator for leading-edge biotechnology startups. “Gene therapy is a fast-developing area within our state’s biotech infrastructure,” said Sara Imhof, Ph.D., senior director of precision health for the North Carolina Biotechnology Center. “Our related ecosystem is well positioned to contribute to – and benefit from – the Bespoke Gene Therapy Consortium. It’s an exciting time, both for the patients who desperately need the benefits gene therapy can provide and for our innovators and industry leaders who are dedicated to this important area of science.”





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Kassie Hoffman
Kassie pens down all the news from the world of politics on ANH.