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LeadingBiotech: Cancer Advance
March 5, 2020
Koch Institute for Integrative Cancer Research at MITCambridge, MA

Kathleen McCarthy
Co-Founder & CSO at Skyhawk Therapeutics


Kathleen began her career by co-developing a treatment for Spinal Muscular Atrophy (SMA) as a pre-clinical scientist at the SMA Foundation. In this role she helped bring the first-ever small molecule splicing modifier towards clinical trials in SMA. This drug candidate was bought by Roche in 2011 and Kathleen moved to Roche to work there as a lead pre-clinical scientist. She was first author on the Nature Communications paper describing the SMA compound mechanism (2017). The SMA drug is now in pivotal clinical trials, and in early 2020, Roche announced the trial met its primary endpoint. Prior to Kathleen’s work in drug discovery and development, she completed a Fulbright Fellowship at the Swiss Federal Institute of Technology, ETH, and graduated with honors in Chemistry from Wellesley College. Kathleen co-founded Skyhawk Therapeutics in January 2018, and is Chief Scientific Officer of the now not-so-small start-up.

Agenda Sessions

  • Spotlight