$250,000 Research Grant to Dr. Christian Lorson, University of Missouri
We are thrilled to announce a $250,000 research grant to Dr. Christian Lorson and his research team at University of Missouri. This grant will be jointly funded with our friends in the fight, FightSMA.
The award will directly support Dr. Lorson’s work to increase production of Survival Motor Neuron (SMN), a protein that is critical for muscle strength in persons diagnosed with SMA. Specifically, the funds will be used to move Dr. Lorson's promising anti-sense oligonucleotide (ASO) drug candidate closer to human clinical trial focusing on toxicity, biodistribution, dose response, and timing studies.
Dr. Lorson's team has been working for years on potential SMA therapeutics and has optimized an ASO compound that is showing promising results. Their drug candidate binds to a powerful regulatory element called Element 1 or "E1." According to Dr. Lorson, inhibition of this region dramatically increases the production of SMN.
“Based upon our preliminary results,” said Dr. Lorson, “our ASO exhibits no detectable toxicity and from a single injection, we observe a 400-600% increase in average survival with some animals living 10-fold longer than untreated littermates. The response we observe is truly exciting and opens the door for a completely new genetic target in SMA.”
“It’s truly a new day in the world of SMA research with several potential therapeutics making their way through human clinical trials,” said Bill Strong, Gwendolyn Strong Foundation co-founder. “And we’re honored to expand that pipeline by supporting the impressive work of Dr. Lorson’s lab to move their ASO drug closer to the clinic.”
“Additional therapeutic targets such as E1 need to be developed as it is an unfortunate reality that the first drug candidates are rarely the last or only therapeutic options,” said Dr. Lorson. “Development of additional high-impact targets will be essential.”
We're proud to be supporting this exciting program in a major way and look forward to tracking its progress over the coming years.