Breakthrough Discovery Offers Hope for Precision Therapy for inherited form of Parkinson’s disease

Parkinson’s disease is the second most prevalent neurodegenerative disease and presents a global health challenge. About 90,000 people are diagnosed in the US every year. About 10 million people world-wide live with Parkinson’s disease, according to Michael J Fox Foundation and Parkinson’s Foundation USA. Specific genetic mutations lead to early onset of PD that account for about 10-20% of all cases of Parkinsons’s disease patients. Inactivating mutations in human Parkin, a ubiquitin E3 ligase and PINK1, a kinase, genes present a significant population of inherited Parkinson’s disease.

In a groundbreaking advance, Progenra Inc., has discovered a novel class of small-molecule PINK1 activators that restore function in mutant forms of the PINK1 kinase, a key mitochondrial regulator whose dysfunction leads to familial or early onset Parkinson’s disease (PD). This discovery represents the first evidence that a drug for defective PINK1 variants is possible, and therapeutically viable. Restoring the function of pathogenic PINK1 mutations found in Parkinson’s patients with small molecule drugs, is a paradigm shift in Parkinson’s disease. The data hints that the drug could reverse disease progression—and benefit late-stage Parkinson’s disease as well.

PINK1/Parkin Role in Neurodegenerative Diseases: Aggregation of proteins in neurons is the main cause of several neurodegenerative disorders. Tau protein aggregates for Alzheimer’s disease and alpha synuclein aggregates for Parkinson’s disease. Protein aggregates damage mitochondria leading to loss of ATP and neuronal cell death. Aggregated proteins or proteinapathies such as Lewy Body disease are also responsible for dementia.

Since the PINK1 drug removes protein aggregates and restores mitochondrial health, its application for Lewy body disease and other proteinathies are also apparent. “This discovery opens an entirely new avenue for neurodegenerative diseases and especially for personalized medicine for PD patients,” added Dr. Tauseef Butt, PhD, President& CEO of Progenra Inc. “Instead of compensating for PINK1 loss, we can now target the defective protein directly, potentially halting disease progression at its origin.”

About Progenra Inc:

Progenra (www.progenra.com), a biotech company, aims to develop novel medicines by exploring mitochondria and ubiquitin proteasome pathways. Progenra is developing novel modulators of PINK1/Parkin signaling pathway as therapeutics for antiaging, neurodegenerative and mitochondrial diseases such as Parkinson’s, Alzheimer’s as well as cardiovascular diseases.

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About 90,000 people are diagnosed in the US a year with Parkinson’s disease. Genetic mutations lead to early onset of PD that account for about 10-20% of all cases of Parkinsons’s disease patients. Progenra drug restores function for most of the patients.