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Expansion Therapeutics Announces Key Additions and Updates to Scientific Advisory Board

-- World-renowned scientists and clinicians will guide Expansion’s efforts to continue advancement of its small molecule RNA platform (SMIRNA™) --

Boston, March 2, 2023

Expansion Therapeutics, Inc., a biotechnology company focused on developing transformative oral medicines for severe RNA-mediated diseases, today announced the addition of Kenneth S. Kosik, M.D., Leonard Petrucelli, Ph.D., and Melissa A. Starovasnik, Ph.D., to its Scientific Advisory Board (SAB). The addition of these distinguished leaders in the field significantly bolsters Expansion’s efforts to continue advancement of its small molecule ribonucleic acid (RNA) platform, called SMIRNA™, to identify and develop innovative therapies to treat severe RNA-mediated diseases, including neurodegenerative and neurological disorders.

“We are honored and excited to have this group of esteemed scientists and clinicians join us as we work to leverage our differentiated platform to positively impact the lives of patients suffering from debilitating diseases who currently have limited treatment options,” said Renato Skerlj, Ph.D., Chief Executive Officer and President of Expansion Therapeutics. “These scientific thought leaders have decades of experience in their respective fields, and their input and feedback will be invaluable as we accelerate the preclinical and clinical development of our novel medicines designed to modulate RNA utilizing our proprietary approach.”

The new members of Expansion’s SAB include:

  • Kenneth S. Kosik, M.D. – Dr. Kosik is the Harriman Professor of Neuroscience Research and Co-Director of the Neuroscience Research Institute at the University of California, Santa Barbara. Previously, he was full professor in neurology at Harvard Medical School and held appointments at McLean Hospital, Brigham and Women's Hospital, the Massachusetts General Hospital and the Dana-Farber Cancer Institute. Dr. Kosik has wide-ranging interests in brain physiology, cell fate, synaptic plasticity and neurodegenerative disease. He has pioneered the investigation of tau biology as it relates to neurodevelopment and disease, with particular emphasis on the regulation of tau splicing, translation, aggregation and interactions with RNA. He is also an expert on RNA biology in general, including that of microRNAs, long non-coding RNAs and RNA-protein complexes. He earned his B.A. and M.A. in English literature from Case Western Reserve University and holds an M.D. from the Medical College of Pennsylvania.

  • Leonard Petrucelli, Ph.D. – Dr. Petrucelli is the Ralph B. and Ruth K. Abrams Professor of Neuroscience at the Mayo Clinic College of Medicine and Science in Jacksonville, Florida. His research focuses on investigating the pathological mechanisms in patients with neurodegenerative diseases including Alzheimer’s disease (AD), frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Dr. Petrucelli and his team were one of the first groups to describe both Repeat Associated Non-AUG (RAN) translation and antisense pathology in the subtype of ALS caused by repeat expansions in the C9orf72 gene and to develop a mouse model recapitulating behavioral and neuropathological features. He is also interested in TDP43 disorders and mechanisms underlying tau spread in dementia. His research focuses heavily on translation to the clinic, with efforts to identify novel therapeutic approaches to ALS, FTD and AD, as well as studies of clinically relevant biomarkers. He holds a B.S. in Biology from Barry University and a Ph.D. in Molecular and Cellular Biochemistry from Loyola University & the Stritch School of Medicine.

  • Melissa A. Starovasnik, Ph.D. – Dr. Starovasnik is an accomplished scientific leader in the fields of protein sciences, including protein/antibody therapeutics and small molecule drug discovery. She spent nearly three decades at Genentech in roles of increasing responsibility and was an integral member of the research leadership team. As Vice President, Protein Sciences, and head of large-molecule drug discovery from 2011-2017, she was responsible for overseeing the discovery and optimization of more than 20 protein therapeutic candidates that progressed to clinical trials. Prior to that, as Vice President, Research Operations and Structural Biology, she coordinated all operational activities for the more than 1300-member Genentech research organization, while also leading the Structural Biology department and serving on the small molecule leadership team. Currently, Dr. Starovasnik serves on the Board of Directors for Twist Bioscience and the Scientific Advisory Boards for multiple therapeutics companies. She earned a B.S. in Chemistry and a Ph.D. in Biochemistry from the University of Washington and has co-authored more than 70 scientific publications

Lynne E. Maquat, Ph.D., Awarded 2023 Gruber Genetics Prize

Expansion Therapeutics also congratulates current SAB member, Lynne E. Maquat, Ph.D., the recipient of the 2023 Gruber Genetics Prize awarded by Yale University for her innovative work and steadfast commitment to research in the field of RNA biology. The Gruber International Prize Program honors scientists from around the world whose groundbreaking work leads to fundamental shifts in knowledge and benefits mankind.

About Expansion Therapeutics

Expansion Therapeutics is a biotechnology company focused on developing transformative oral medicines for severe RNA-mediated diseases including neurodegenerative disorders. Based on exclusive worldwide rights to groundbreaking research from the laboratory of Matthew D. Disney, Ph.D., at Scripps Research, Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule RNA modulators. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of severe RNA-mediated disease indications. Expansion has sites in Boston and Cambridge, Massachusetts, and Jupiter, Florida. For more information, visit



Media Contact: Mike Beyer Sam Brown Inc. 312-961-2502


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