Silverstein Foundation, Q-State Launch Company Aiming to Treat Parkinson’s Linked to GBA Mutations

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The Silverstein Foundation for Parkinson’s with GBA together with Q-State Biosciences have launched Chamishi Therapeutics, a company aiming to develop innovative treatments for Parkinson’s disease and other neurodegenerative diseases.

Chamishi will initially focus on developing antisense oligonucleotide (ASO) therapies for Parkinson’s patients carrying mutations in the GBA gene, one of this disease’s most common and significant genetic risk factors.

“We are at a turning point in the treatment paradigm for Parkinson’s disease where insights from underlying genetic pathways can enable development of disease-modifying therapies instead of the solely symptomatic treatments available today,” Jonathan Silverstein, founder of The Silverstein Foundation for Parkinson’s with GBA, said in a press release.

The GBA gene holds the instructions to produce the enzyme beta-glucocerebrosidase (GCase). This enzyme is active in lysosomes, special compartments within cells that digest and recycle different types of molecules.

GBA mutations stop the normal activity of the GCase enzyme, allowing toxic substances to accumulate inside cells — particularly as people age — that lead to inflammation. Mutations in the GBA gene are considered to increase a person’s risk of developing Parkinson’s disease. Between 7%–10% of all Parkinson’s cases are related to GBA mutations.

The DNA contained in our genes is transformed into RNA, which is then processed (translated) to give rise to proteins. Antisense oligonucleotides (ASOs) are small RNA-targeting molecules designed to correct the genetic defects (mutations) in a gene, rescuing the production of a particular protein. These molecules influence RNA processing and modulate protein expression.

Chamishi Therapeutics will combine the Silverstein Foundation’s scientific knowledge on GBA-linked Parkinson’s disease with Q-State’s lab-made assays, ASO design and precision medicine platforms.

“We are excited to partner our precision medicine technologies with Chamishi to develop highly innovative therapies for patients with Parkinson’s disease and other related disorders,” said Matthew Fox, CEO of Q-State Biosciences. “This collaboration is an excellent opportunity for Q-State to expand the reach of our ASO capabilities and proprietary assay platform to help a new population of patients and their families.”

Jim Sullivan, PhD, is the CEO of Chamishi. Sullivan has over 25 years of experience in biopharmaceuticals. A former vice president of discovery at AbbVie, he was involved in the development of approved medicines for hepatitis C, cancer, and rheumatoid arthritis.

“Chamishi will harness the latest innovations in ASO technology and our emerging understanding of the immune system’s role in neurodegenerative disorders to develop disease-modifying therapeutics,” Sullivan said.

“By developing ASOs that prevent expression of toxic proteins or restore expression of beneficial proteins, Chamishi has the opportunity to introduce new therapies for patients with GBA-Parkinson’s disease and many other indications,” he added.