The Working Group on Pediatric Gene Therapy & Medical Ethics (PGTME) at NYU Grossman School of Medicine will host its 6th Annual Lunchtime Lecture Series during the week of November 17-21 from 12-1:15pm ET daily.

Please join us for a week of virtual sessions on pressing topics in the gene therapy space featuring expert speakers and audience Q&A. All sessions are free, virtual, and open to the public!

This year's Lecture Series will feature sessions on bioethics basics, evidence and drug approval, communication and data sharing, ethical principles in research and development of genomic medicines, and the economics of rare disease.

What Cure Leaves Behind: Listening to the Lived Reality of Innovation

The COALESCE study is a multisite initiative dedicated to tracking health outcomes in persons with sickle cell disease who undergo curative therapies like bone marrow transplants or gene therapies. In order to make sure we are fully addressing the needs and concerns of the sickle cell community, we are dedicated to bringing together members of the sickle cell community and doctors studying and treating sickle cell disease. The goal of our work is to help empower doctors and persons living with sickle cell disease to develop more personalized approaches for treatment and using curative therapies.

Sickle cell disease is an inherited disorder caused by a single “typo” in the genetic code. CRISPR genome editing is a method for making targeted changes in DNA. CRISPR could be used as a treatment, or even cure, for sickle cell. In fact, the first patient to receive this experimental therapy is now thriving. This renewed hope comes after decades of slow research advancement and discriminatory treatment of sickle cell patients by doctors. It is no coincidence that in the U.S., sickle cell predominantly affects Americans with African ancestry. Our country’s long history of discrimination against Black people raises a host of ethical questions about where CRISPR belongs in the equation, and how a potentially curative—but inherently risky—therapy should be introduced to the sickle community.

This two-part event begins with a narrated virtual reality “tour” of a human body. A CRISPR scientist will explain what causes sickle cell disease, how it affects the body, and how CRISPR genome editing may be used to repair the sickle mutation. Following the tour, the audience will join a set of panelists to have a candid conversation on science & society, including topics like health disparities in the U.S., race and racism in medicine, access to emerging therapies, the ethics of CRISPR, and more.