Emily’s Entourage Celebrates Landmark Achievement: First Patient Dosed with Novel Gene Therapy Developed Through EE’s Visionary Model

Philadelphia, Pennsylvania—November 18, 2024—Emily’s Entourage (EE), an innovative 501(c)3 accelerating research for new treatments and a cure for the final 10% of people with cystic fibrosis (CF), proudly announces a pivotal milestone in its mission to speed breakthroughs for those with CF who do not benefit from currently available mutation-targeted therapies: Spirovant Sciences, a clinical-stage gene therapy company that EE helped launch, has dosed the first patient in its SAAVe Phase 1/2 clinical trial of SP-101 + Augmenter for CF treatment.

This achievement represents a full circle moment for EE. In 2015, EE catalyzed the creation of Spirovant—then named Talee Bio—conceiving of, engaging the scientific community and a management company, and providing the seed funding to launch Spirovant Sciences. Nearly a decade later, that initial investment has led to the development of SP-101, a mutation-agnostic gene therapy delivered via inhalation, now advancing in a clinical trial.

“This milestone is nothing short of extraordinary,” said Emily Kramer-Golinkoff, MBE, co-founder of Emily’s Entourage. “What began as a dream to accelerate lifesaving science for those in the final 10% of the CF community has evolved into this transformative moment: a novel gene therapy being administered to a person with CF. Together, we’ve taken an idea from preclinical research to human trials. This progress exemplifies what our work is all about.”

EE’s visionary ‘Venture Philanthropy’ approach to drive research and therapeutic development for CF accelerated the creation of Spirovant and propelled its work on SP-101, which leverages an adeno-associated virus (AAV) vector to deliver the CFTR gene, targeting the root cause of CF. SP-101 is mutation agnostic, meaning that if proven effective, it would work for all mutations of CF, including those not currently benefiting from existing mutation-targeted therapies.

“The first patient dosed with SP-101 is an enheartening moment in our steadfast mission toward addressing unmet needs in the CF community, most especially in the final 10%,” said Chandra Ghose, PhD, Chief Scientific Officer at Emily’s Entourage. “This milestone reflects years of dedication, innovation, and collaboration—proof that with strategic investments and collective effort, we can turn scientific potential into meaningful progress for those waiting for new options.”

The SAAVe Phase 1/2 trial is designed to evaluate safety, tolerability, and efficacy in individuals for whom existing treatments are insufficient or unavailable. Key secondary endpoints include lung function, quality of life, and biomarkers indicative of therapeutic impact.

“This achievement would not have been possible without the support of our incredible community,” added Kramer-Golinkoff. “To everyone who has donated, participated, or shared our mission in any way over the years: this milestone is yours, too, and we thank you with all of our hearts.”

For more information about the SP-101 clinical trial, visit the full announcement from Spirovant on PRNewswire.

Sobre a comitiva de Emily

A Emily's Entourage é uma organização inovadora 501(c)3 que acelera a investigação de novos tratamentos e de uma cura para os indivíduos na última 10% da população com fibrose cística (FC) que não beneficia das terapias direcionadas para as mutações atualmente disponíveis. Desde 2011, a Emily's Entourage concedeu milhões de dólares em bolsas de investigação, lançou uma empresa de terapia genética para a FC, agora adquirida, desenvolveu uma base de dados de doentes e um programa de seleção de ensaios clínicos para acelerar o recrutamento para ensaios clínicos e liderou esforços a nível mundial para impulsionar a investigação de alto impacto e o desenvolvimento de medicamentos. A organização tem sido referida nos meios de comunicação social nacionais, incluindo o New York Times, STAT, CNN, People, entre outros. Saiba mais em emilysentourage.org.

Contacto para a imprensa

Hannah Kleckner

Diretor de Marketing e Comunicação

hannah@emilysentourage.org