Emily’s Entourage to Host Scientific Symposium on the Promise of Gene-Based Therapies for Cystic Fibrosis

Philadelphia, PA – November 21, 2023 – Emily’s Entourage (EE), an innovative 501(c)3 that accelerates research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), including those with nonsense mutations, today announced its 2023 scientific symposium, The Promise of Gene-Based Therapies for Cystic Fibrosis, to be held on December 4 – 5, 2023, at Quorum at the University City Science Center.

The theme of the symposium acknowledges the recent progress in the development of gene-based therapies in CF that have the potential to reach the final 10% of the CF community who do not benefit from currently available therapies, and may lead to a curative treatment for everyone with CF. Despite significant advances, critical challenges remain in developing and delivering novel gene-based therapies for CF. The symposium brings together leading minds from within — and outside — the CF scientific community, industry, and FDA to share openly, collaborate, ask tough questions, and ultimately create solutions and strategic, accelerated pathways forward.

Chaired by Drs. Patrick Harrison, PhD, Maria Limberis, PhD, and Batsheva Kerem, PhD, the invite-only symposium convenes over 34 speakers and 86 attendees from 18 countries. Among the esteemed invited speakers is Dr. Drew Weissman, BS, MS, PhD, MD, recipient of the 2023 Nobel Prize in Physiology or Medicine.

The symposium includes seven thought-provoking sessions unfolding over two days. Each session will offer presentations and ample opportunities for engaging in in-depth discussions. As the grand finale, the two concluding sessions will take the form of dynamic roundtable discussions, expertly moderated and enriched by the insights of multiple panelists.

I. CF Therapeutics – Past, Present & Future

Paul Negulescu, PhD | Vertex Pharmaceuticals
Jennifer Taylor-Cousar, MD, MSCS, ATSF | National Jewish Health
Kara Foshay, PhD | Cystic Fibrosis Foundation

II. RNA Based Approaches

Drew Weissman, BS, MS, PhD, MD | University of Pennsylvania
Fred Van Goor, PhD | Vertex Pharmaceuticals
John Lueck, PhD | University of Rochester, School of Medicine and Dentistry
Zoya Ignatova, PhD | University of Hamburg

III. Leveraging Novel Delivery Platforms for mRNA, tRNA and Oligonucleotide-Based Approaches

Rana Ghosh, PhD | University of Texas at Austin
Alexandra Piotrowski-Daspit, PhD | University of Michigan
Marc Abrams, PhD | Carbon Biosciences

IV. CFTR Gene Therapy and Gene Editing in CF

V. Current Landscape of Gene-Based Therapies

Matt Porteus, MD, PhD | Stanford University
Kiran Musunuru, MD, PhD, MPH, ML | University of Pennsylvania
Marcus Noyes, PhD | NYU Grossman School of Medicine

VI. Perspective on Rare Gene Therapies to Expedite the Development of Therapies for CF

VII. Challenges in Gene Therapy Clinical Trials through the CF Lens

“There has been tremendous progress in the development of gene-based therapies in recent years. We’re encouraged by the progress as it represents a beacon of hope for everyone in the CF community and especially those in the final 10% that do not benefit from existing mutation-targeted therapies and are still desperately waiting for treatment options,” said Chandra Ghose, Chief Scientific Officer of Emily’s Entourage. “We’re thrilled to convene world-class leaders at our symposium to collaboratively chart an expeditious path to a future where 100% of the CF community has lifesaving therapies that enable them to live a long, healthy life, regardless of their genetic mutation.”

EE thanks the 2023 symposium Presenting, Dream Team, and Violet-Level sponsors, Viatris, 4D Molecular Therapeutics, Vertex Pharmaceuticals, Inc., ReCode Therapeutics, Boomer Esiason Foundation, and more whose support makes this critical event and the progress that it yields possible.

About Emily’s Entourage

Emily’s Entourage is an innovative 501(c)3 that accelerates research for new treatments and a cure for individuals in the final 10% of the cystic fibrosis (CF) population that does not benefit from currently available mutation-targeted therapies. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development. The organization has been featured in national media, including the New York Times, STAT, CNN, People, and more. Learn more at emilysentourage.org.

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