Toward the next generation of gene therapy
Imagine a world where the progression of debilitating chronic conditions can be stopped—with a single dose of genetic medicine. Where gene therapy can truly alter the course of a disease, and change the course of people’s lives. It’s this vision that drives us forward. That spurs our efforts toward change.
At Spur Therapeutics, we’re leveraging the power of getting just the right gene expression, packaged and delivered to the body in just the right way, to develop transformational gene therapies for more diseases. Building on our success with a breakthrough gene therapy candidate for Gaucher disease, we’re moving from rare diseases toward more widespread conditions, including forms of Parkinson’s and dementia, and even certain cardiovascular diseases. Expanding our impact, and pushing forward to new frontiers of genetic medicine. Toward life-changing therapies, and brighter futures. Toward more.
Advancing the practice
of genetic medicine
We’re optimizing every component of our product candidates, improving genetic expression and targeted delivery to realize outsized clinical results. And by increasing our focus on more prevalent chronic diseases with significant unmet treatment needs, we are moving toward a greater impact on more people around the world.
With current treatments, many people living with Gaucher disease type 1 still experience debilitating symptoms, including enlarged organs, fatigue, bone pain, and lung dysfunction. Our gene therapy candidate, FLT201, is designed to deliver GCase85, a longer-acting version of the enzyme deficient in people with Gaucher disease, with the goal of stopping disease progression, reducing or eliminating symptoms, and allowing patients to come off current lifelong treatments.
Preclinical and clinical data for FLT201 have shown robust and durable expression and a substantial reduction in the toxic buildup of substrate that results from the enzyme deficiency. FLT201 is currently in a Phase 1/2 study to identify the optimal dosage for a Phase 3 trial.
No disease-modifying therapies currently exist for Parkinson’s disease. Our longer-acting GCase85 enzyme could change that.
For people with Parkinson’s disease with GBA1 mutations, our enzyme provides an opportunity for a novel therapy that would combat the GCase deficiency that leads to the formation of Lewy bodies and death of dopaminergic neurons. In preclinical studies, GCase85 demonstrated activity levels in brain and neuronal cells up to 20 times higher than the wildtype GCase enzyme. This could be a first step toward a gene therapy for the hundreds of thousands of people with GBA1 Parkinson’s worldwide.
There is currently no treatment to slow or alter the progression of AMN, a neurodegenerative disease characterized by mobility loss, incontinence, debilitating pain, and sexual dysfunction. Our gene therapy candidate, SBT101, is designed to deliver a functioning ABCD1 gene, replacing the protein deficient in people with AMN. Preclinical studies have been promising, showing elevated ABCD1 protein expression and decreases in substrate in the spinal cord. SBT101 is currently in a Phase 1/2 clinical trial.