On May 29, Emily’s Entourage (EE) attended Translate Bio’s Cystic Fibrosis (CF) Awareness Month event in Boston. EE Co-Founder, Emily Kramer-Golinkoff, provided opening remarks to launch the day’s events as well as took part in a Q&A panel discussion.
On the panel, Emily was joined by key CF community members, Bob Coughlin, CEO of MassBio, Dr. Jonathan Zuckerman, a physician involved in Translate Bio’s ongoing clinical trial, and Dr. Ann Barbier, Translate Bio’s CMO. The panel was moderated by Dr. Meagan Vaughn, Senior Clinical Research Scientist at Rho, and delved into the urgent need for new treatment options in CF and how “someday isn’t good enough for the final 10%.”
The session was livestreamed and can be viewed below. Members of the CF community were encouraged to tune in and sent in their questions.
During her remarks, Emily spoke about her own journey with nonsense mutations of CF as well as shed light on EE’s laser focused mission to develop life-saving treatments and drug development for the remaining 10%.
Following her remarks, Emily participated in a panel discussion covering topics such as the current state of CF treatment innovation, the potential role that emerging therapies may play in the future, and the burden that 10% of the CF community is still carrying.
Translate Bio is a mRNA therapeutics company currently developing medicines to treat diseases caused by protein or gene dysfunction, such as CF. With game-changing breakthroughs on the immediate horizon for roughly 90% of individuals with CF, those with nonsense mutations represent the majority of the outlying 10% who will not benefit from these breakthroughs.
Emily’s Entourage is incredibly honored to have participated alongside such a special lineup of key players in the CF community to raise awareness of the critical work that still needs to be done for the final 10% of the CF community. Huge thank you to Translate Bio for organizing such a wonderful event and for including EE and Emily in it!