A Cure in Progress
Since our foundation’s inception in 2013, we’ve been racing to find a cure for LBSL. The research we’ve funded has led us to our main effort: a DARS2 AAV9 gene therapy, led by our partners at Kennedy Krieger Institute in Baltimore. Our $3 million Accelerate Hope Initiative aims to bring this groundbreaking therapy to clinical trials.
Paving the Way
AAV9 Gene Therapy & LBSL
AAV9 is a modified virus that has been engineered to safely deliver genetic material into cells. It’s like a microscopic delivery vehicle that carries a healthy copy of a gene to where it's needed in the body. AAV9 is a trusted vector that is used in other FDA-approved gene therapies.
For patients with LBSL, AAV9 would transport a working copy of the DARS2 gene to improve gene function. DARS2 produces an essential enzyme responsible for mitochondrial protein production, and its dysfunction primarily affects the nervous system. Because AAV9 can cross the blood-brain barrier, it’s well-suited to addressing neurologic diseases — and LBSL in particular.
Preclinical AAV9-DARS2 data show a number of successes, including improved mitochondrial function, an increase in nerve growth, decrease in lactate and benefits that lasted at least six months after a single treatment.
Accelerate Hope
To make this gene therapy a reality, we’ve launched our Accelerate Hope initiative with the goal of raising $3 million to fund the critical work needed to get to clinical trials. As we say in the LBSL community, time is myelin — every second that ticks by is disease progression in our patients — so we have no time to waste to change lives with a lasting LBSL therapy.