Mahzi Therapeutics

great couple of days at at the #globalgenes meeting. Inspiring stories, great conversations. Thank you Kansas City, you’ve been good to us. “There is no place like hope”! Charlene Son Rigby Nicole Boice Nasha Fitter Sunitha Malepati Pangkong Fox Effie Parks Emma James E. Gay Grossman Terry Jo Bichell Sierra Phillips Wendy Erler Bruce Bloom Matthew Fuller Sandra Bedrosian Sermone Stacey Seeloff Eric Scheeff Anna Pfalzer Wendy Chung and many others…

Proud of this team!

I was lucky to be in town for the inaugural Rocknrare event, organized by Julia Vitarello the Broad Institute and The Termeer Foundation A wonderful combination of music by Casey McPherson and Alpha Rev and Alan Walts and an outstanding lineup of participants - @Sammy basso Tim Yu David R. Liu Weston and Azlan and inspiring young patient advocates who were Mila’s best friends. I hope those of you who care about rare and weren’t able to attend will watch the recording! Erica Mawby-Roche Catharine Smith Dan Ouellette

Drinks on me!

Exciting panel - Empowering Patient Advocacy in Rare Disease Therapies! Collaboration and transparency are key! Great discussion on incorporating the patient voice early in development PMWC - Precision Medicine World Conference Charlene Son Rigby J. Michael Graglia Justin West, MD Wendy Erler Christina Theodore-Oklota Eric Crombez ?? Adam Shaywitz

On to our second panel PMWC - Precision Medicine World Conference - led by ?? Adam Shaywitz with Salvador Rico, MD, PhD Eric Crombez Elizabeth Berry- Kravis discussing novel clinical design approaches in rare diseases Natural history studies, placebo/sham controlled studies, gene therapy

Kicking off the day with our first panel at #PMWC: Accelerated Approval for Small Populations: Biomarkers and Endpoints Led by Peter Marks, with Tim Yu Jennifer Panagoulias Katherine High, MD Jennifer Puck Maria Grazia Roncarolo

Join an amazing team!! Sunitha Malepati is #hiring. Know anyone who might be interested?