MeiraGTx, a gene therapy company, has announced that it has been awarded a grant from Target ALS, a privately funded foundation bringing together a consortia of academic and biotech/pharmaceutical researchers focused on finding treatments for ALS patients. Target ALS is jointly funding this grant with the research organization ALS Finding A Cure. The grant will fund testing of a novel gene therapy approach to treat this devastating neurodegenerative disease that targets TDP-43, a protein that is misregulated in over 95 per cent of all ALS patients. The approach would control misregulated proteins in the cell. Out of 43 applicants for the industry-led consortium funding, MeiraGTx was one of just five awardees. The grant was reviewed by an independent review committee comprised of senior investigators in academia and in the biotech/pharmaceutical industry.

The ALS-associated protein TDP-43 has been emerging as a possible Achilles’ Heel in ALS treatment.  Research conducted by Dr. Greg Petsko and colleagues at Weill Cornell Medicine and Brandeis University has revealed that one of the cell’s own quality control mechanisms, 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. The research found that increased expression of UPF1, the master regulator of NMD, can significantly protect mammalian motor neurons from TDP-43 mediated toxicity.

The grant was received to confirm and extend initial findings on the efficacy of this therapeutic approach. 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.

“Target ALS is pleased to support the MeiraGTx led collaborative consortium,” said Manish Raisinghani, President of the Target ALS Foundation. “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.”

“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,” said Peter Foss, President of ALS Finding A Cure. “We are proud to be a part of this innovative effort.”

“We are honored to receive this prestigious grant to help us pursue our promising findings about NMD-based therapy,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “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.”