New York and UK-based MeiraGTx has been awarded a research grant funded by Target ALS and ALS Finding a Cure to fund the testing of a gene therapy approach that targets the ALS-associated protein TDP-43.
The grant was received to confirm and extend the initial findings on the efficacy of a therapeutic approach conducted by Dr Greg Petsko and colleagues at Weill Cornell Medicine and Brandeis University.
Their research suggests that the nonsense-mediated decay (NMD) pathway appears to be able to alleviate TDP-43 mediated toxicity in cell and animal-based disease models of ALS. Increased expression of UPF1, the master regulator of NMD, can significantly protect mammalian motor neurons from TDP-43 mediated toxicity, explains a media release from MeiraGTx.
MeriaGTx will test the approach in well-characterized mouse models of ALS derived in the laboratory of Dr Neil Shneider at the Columbia College of Physicians and Surgeons, and through the use of biochemical methods defined in the Petsko and MeiraGTx lab will generate candidate biologics that are even more potent in generating the same response, the release continues.
“This award is part of our goal to seed and support ALS drug discovery programs in the pharma and biotech industries to realize new treatments for this devastating disease,” says Manish Raisinghani, president of the Target ALS Foundation, in the release
“Joining with Target ALS to increase the number of researchers developing novel treatments for ALS is a great opportunity to advance our ultimate mission to cure this deadly disease,” states Peter Foss, president of ALS Finding A Cure.
“We are honored to receive this prestigious grant to help us pursue our promising findings about NMD-based therapy,” shares Alexandria Forbes, PhD, president and CEO of MeiraGTx, per the release. “We believe this is a key development in the treatment of ALS and that our novel approach may help the thousands of people affected by this devastating neurodegenerative disease.”
[Source9s): MeiraGTx, PR Newswire]