Recent research has focused on the idea that misfolded proteins play a critical role in damaging neuronal function and progression to neuronal cell death. MeiraGTx’s new program, in partnership with Greg Petsko, PhD, of Weill Cornell Medicine, targets neurodegenerative disease by altering neuronal protein processing.
“We are excited to initiate this important collaboration,” Petsko said in a press release. “I believe that using gene therapy approaches to correct trafficking defects in the brains of Alzheimer’s patients by increasing retromer pathway function, addresses what may be the seminal cellular problem in this — and probably other — neurodegenerative diseases.”
Grounded on Petsko’s influential work, the new collaborative program targets the protein complex known as “retorter,” one of the most important regulators of protein trafficking. The research team is developing genetic strategies to reprogram retromer, impacting protein trafficking, processing and aggregation.
Petsko and his colleagues aim to modulate retromer function using gene therapy, in order to treat several inherited and acquired neurodegenerative diseases, such as AD and PD.
“My colleagues and I think this is an innovative and important approach that holds great promise for the treatment of Alzheimer’s disease. While we realize that there is still a long way to go before our ideas can be tested in patients, partnering with MeiraGTx, one of the world’s leading companies in the exciting field of gene therapy, puts the translation of this science to the clinic in the best possible hands,” Petsko added.
Gene therapy is an experimental technique that uses genes to treat or prevent certain diseases. Physicians hope that this technique one day will allow them to treat a disorder by replacing a “sick” gene with a healthy gene, to “knock-out” a mutated gene that is causing trouble, or to introduce new genes that might help fight disease.
Although gene therapy is a promising option, the technique is still in its early stages and treatments are only being sought for diseases that have no other cures.
MeiraGTx is a gene therapy company working on acquired and inherited disorders, including ocular diseases like rare inherited blindness or age-related macular degeneration.
“We are very excited to begin this collaboration with Dr. Petsko and his colleagues. We believe that retromer is one of the most important new targets in the field of Alzheimer’s and neurodegenerative disease. Our approaches may lead to effective new therapies for patients with these diseases,” said Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx.
In related news, Voyager Therapeutics has recently reported positive interim six- and 12-month data from its Phase 1b clinical trial, showing that its gene therapy VY-AADC01 was well-tolerated and resulted in clinically relevant outcomes for patients with advanced PD.