The Michael J. Fox Parkinson’s Disease Research Foundation (MJFF) is conducting a second study to accelerate the preclinical development of new therapeutic compounds targeting the genetic causes of Parkinson’s disease by California-based Seronthera. A $2.5 million grant was awarded to Seronterra.
Serontella is specifically researching whether compounds that increase the activity of Parkin, a protein associated with cases of familial Parkinson’s disease, may help improve cognitive function and alleviate some of the disease’s motor symptoms. It is planned to be tested in a clinical model.
Preclinical studies help determine whether the company’s cutting-edge compounds can be safely used in Phase 1 clinical trials in patients.
“MJFF’s funding for therapeutic development is an important way we are working to enable a diverse treatment pipeline for people with Parkinson’s disease,” said Brian Fisk, Ph.D., the foundation’s chief scientist, in a press release from Seronterra. stated in the release. “We highly value our partnerships with pharmaceutical manufacturers to provide them with the resources to develop innovative results.”
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Initial $2.5 million MJFF grant funded early research on Serontella
Dr. Ann Wolfer Buchwalder, Seronterra’s co-founder and president, said Parkinson’s disease has a “strong genetic component” that the company’s research is targeting.
“The existence of dysfunctional pathways in common with other neurodegenerative diseases has led us to fundamental common underlying mechanisms operating in cognitive impairment,” Wolfer-Buchwalder said. “With this new funding, we will continue to develop small molecules to address these challenges for the benefit of Parkinson’s patients.”
Parkinson’s disease is characterized by the progressive degeneration and death of nerve cells in the brain responsible for producing the chemical messenger dopamine. Loss of dopamine causes symptoms of the disease, including both motor and non-motor symptoms.
Although the cause of Parkinson’s disease is not completely understood, several genetic mutations and variants are thought to be strongly associated with the risk of developing the disease.
Seronterra is developing treatments that specifically target mutations in the SCNA gene called A53T and the G2019S mutation in the LRRK2 gene. However, rather than focusing on the effects of mutations on the proteins these genes produce, the company is focusing on target genes near SCNA and LRRK2, whose activity can be dysregulated by disease-causing mutations. We are developing small molecules that can normalize activity.
The existence of dysfunctional pathways in common with other neurodegenerative diseases led us (towards) fundamental common underlying mechanisms operating in cognitive disorders. … With this new funding, we will develop small molecules to address these challenges for the benefit of Parkinson’s disease patients.
The company had previously identified compounds with the potential to improve the function of genes affected by SCNA and LRRK2 mutations. In a separate project, also funded by $2.5 million from MJFF, Seronterra investigated whether restoring the activity of dysregulated target genes could improve cognitive and motor function in Parkinson’s disease.
For this project, the researchers enhanced the structure of the test compound for oral administration by changing its chemical structure and analyzing the effects on the compound’s stability and activity. These new compounds were then tested in animal models of the disease.
The newly funded research will further support preclinical development of compounds aimed at increasing Parkin activity and will also drive optimization of other compounds developed by Seronterra.
Dr. Roman Erfer, Co-Founder and CEO of Seronterra, said: “The continued support of the Michael J. Fox Foundation to advance our discoveries to meaningfully impact people with Parkinson’s disease. We are very grateful for the grant support.”
The company says the newly developed compound could also be used to treat other conditions, particularly Alzheimer’s disease, which is characterized by cognitive problems and pathways in common with Parkinson’s disease.
