A Emily's Entourage atribui uma subvenção de investigação no valor de $220 000 ao Dr. Andrew Berical da Universidade de Boston para fazer avançar a investigação sobre métodos de administração de genes

A investigação incide sobre developing partially humanized mouse airways to be utilized for testing the efficiency of gene delivery methods, including AAV vectors and lipid nanoparticles.

Andrew Berical, MD
Boston University

Lower Merion, PA - novembro 20, 2023 - A Emily's Entourage (EE), uma organização inovadora 501(c)3 que acelera a investigação de novos tratamentos e de uma cura para o último 10% das pessoas com fibrose quística (FC), anunciou hoje que recebeu $220,000 in funding to Dr. Andrew Berical, MD, Physician Scientist and Assistant Professor of Medicine at Boston University Chobanian & Avedisian School of Medicine, Pulmonary and Critical Care Medicine. The grant supports research to develop an in-vivo model of human airway epithelial cells to test gene therapy.

Dr. Berical is currently a faculty member and scientist in the Center for Regenerative Medicine, where his research focuses on airway disease modeling as well as the role of cellular regeneration as a potential therapy for CF and primary ciliary dyskinesia. In this research project, titled “In vivo human airway epithelial cells for CFTR gene therapy,” he plans to study the barriers to various gene delivery methods using W1282X human airway epithelial cells and a novel humanized mouse model.

The research proposal includes assessing the ability of viral vectors and lipid nanoparticles (LNP) to deliver mRNA to human airway epithelial cells with the goal of understanding delivery agent preferences for certain cell types of the human airway and the longevity of these gene delivery technologies.

“We are proud to support Dr. Berical in this groundbreaking research,” said Dr. Chandra Ghose, PhD, Chief Scientific Officer of EE. “Dr. Berical’s pioneering research aims to create an in vivo model of human airway epithelial cells to facilitate testing of gene therapy delivery mechanisms, which currently represents a major barrier to progress and is critical to accelerating the development of new therapies for individuals with CF, including those in the final 10%.”

Leveraging expertise in pluripotent stem cell directed differentiations and recent progress in transplanting human induced pluripotent stem cell (iPSC)-derived basal cells into a mouse airway, the project aims to create a partially humanized mouse airway for testing the efficiency of gene delivery methods. The team will then use this system to assess the ability of two specific agents to deliver genes to human airway epithelial cells: an Adeno-Associated Virus (AAV) vector and LNPs. 

“This generous support from Emily’s Entourage will enable our lab to address a significant gap in CF research. By creating a model system that includes human CF airway epithelial cells, we can advance our understanding of gene delivery mechanisms and pave the way for mutation-agnostic therapies that can benefit all individuals with CF, regardless of their specific CFTR mutation,” said Dr. Berical.

Through this research, Dr. Berical and his research team hope to bridge a significant knowledge gap in the CF field, ultimately contributing to the development of efficacious therapies for all individuals with CF.

Sobre o programa de subsídios da Emily's Entourage (EE) 

O Programa de Subsídios da Emily's Entourage (EE) concede subsídios para acelerar a investigação e o desenvolvimento de terapêuticas para pessoas com fibrose quística (FC) que não beneficiam das terapias existentes direccionadas para as mutações. O EE fornece financiamento através de vários mecanismos de financiamento de subsídios, incluindo subsídios de translação, subsídios de colaboração e subsídios exploratórios pré-clínicos, bem como através de investimentos de filantropia de risco.

Até à data, a EE concedeu milhões de dólares a equipas multidisciplinares em todo o mundo e ajudou a garantir milhões de dólares em financiamento subsequente. Para ver as subvenções concedidas, visite https://www.emilysentourage.org/awarded-grants/. Para saber mais sobre as oportunidades de financiamento da EE, visite https://www.emilysentourage.org/funding-opportunities/. 

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 direccionadas 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 um registo de doentes e um programa de combinação 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

Entourage de Emily

admin@emilysentourage.org