Emily's Entourage assegna un contributo di ricerca di $220.000 alla Queen's University Belfast per sostenere lo sviluppo della nuova terapia genica rigenerativa di OmniSpirant Limited per il trattamento della fibrosi cistica

The innovative technology platform aims to deliver inhaled gene therapy directly to the lung to treat the underlying cause of cystic fibrosis (CF)

Lower Merion, PA – February 14, 2023 — Emily’s Entourage (EE), an innovative 501(c)3 foundation 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 it has awarded $220,000 in funding to Lorraine Martin, PhD, of Queen’s University Belfast to support the development of a novel regenerative gene therapy platform technology that may provide a new treatment approach for people with CF, including individuals with nonsense mutations.

In addition to funding from EE, Cystic Fibrosis Trust, the UK’s leading charity supporting people with cystic fibrosis, has provided a grant from their Venture and Innovation Award fund to support Professor Martin’s pioneering research. This co-funding model reflects EE’s focus on forming collaborative funding partnerships with other organizations to maximize its impact in areas that align with EE’s research priorities.

With this grant, Professor Martin, in collaboration with the biotechnology company OmniSpirant Therapeutics, aims to advance the development of an inhaled gene therapy platform for CF. This technology is based on biological, non-viral nanoparticles called extracellular vesicles (EVs) that are produced from stem cells. Nonsense mutations of CF result in truncated and nonfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein. The gene therapy is engineered to transport and deliver a genetic, protein-making template for the full length CFTR protein, thus addressing CF at the genetic level.

“By addressing the underlying cause of CF, the gene therapy technology developed by OmniSpirant in collaboration with Professor Martin’s lab has the potential to treat the majority of people with CF regardless of genetic mutation, including those with nonsense and rare mutations,” said Chandra Ghose, PhD, EE’s chief scientific officer. “This is a significant grant for EE. It is our first time co-funding a grant with Cystic Fibrosis Trust. In addition, it represents the diversification of our grant funding mechanisms, including for public/private partnerships, which are designed to accelerate therapeutic development for the final 10%.” 

Funding from EE and the Trust will allow Professor Martin’s team at Queen’s to conduct additional preclinical studies including functional gene transfer studies and safety studies – an important part of advancing the technology toward future clinical trials.

“We are thrilled to partner with Emily’s Entourage for the first time through this collaborative grant,” said Dr Lucy Allen, Director of Research and Healthcare Data, at Cystic Fibrosis Trust. “Developing treatment options for people with CF who cannot take CFTR modulators has recently been identified as the top priority for research by people with cystic fibrosis. It is exciting to jointly support this important work as this technology represents a way to reach people who do not benefit from currently available CF therapies.”

These two funding sources will significantly contribute to the advancement of biological non-viral therapies and will provide critical support of the development of gene therapy technologies for nonsense mutations of CF.

“I am really delighted to receive this funding from Emily’s Entourage, and the additional support provided by Cystic Fibrosis Trust, as it will allow us to take the next steps to advance this novel technology,” said Professor Martin, who is based in the School of Pharmacy at Queen’s. “Using this funding, we will conduct further research with the goal of unlocking the extraordinary potential of nucleic acid-based therapeutics for nonsense mutations that cannot be treated with the therapeutics currently approved.”

Professor Martin and her team at Queen’s will continue to work collaboratively with OmniSpirant Therapeutics to advance the development of the gene therapy platform. 

“OmniSpirant Limited is developing OS001, a first-in-class regenerative gene therapy for CF, and we have demonstrated promising results in preclinical disease models,” said Gerry McCauley, Chief Executive Officer, OmniSpirant, “We are excited to progress the development of OS001 with EE funding in partnership with Queen’s University Belfast. We believe OS001 has the potential to restore leading levels of CFTR activity regardless of CF genotype. We are incredibly grateful for the support and scientific expertise of Emily’s Entourage and the Trust, which will allow OmniSpirant to advance this potentially game-changing therapeutic program in collaboration with Professor Martin.”

Per saperne di più sulle sovvenzioni che Emily's Entourage ha finanziato, visitate il sito www.emilysentourage.org/awarded-grants. 

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Informazioni su Emily's Entourage

Emily's Entourage è una 501(c)3 innovativa che accelera la ricerca di nuovi trattamenti e di una cura per le persone che rientrano nell'ultimo 10% della popolazione affetta da fibrosi cistica (FC) e che non traggono beneficio dalle terapie mirate alle mutazioni attualmente disponibili, comprese quelle con mutazioni nonsense della FC. Dal 2011, Emily's Entourage ha assegnato milioni di dollari in sovvenzioni per la ricerca, ha lanciato un'azienda di terapia genica per la FC, ora acquisita, ha sviluppato un registro dei pazienti e un programma di abbinamento per gli studi clinici per accelerare il reclutamento degli studi clinici e ha guidato gli sforzi a livello mondiale per promuovere la ricerca e lo sviluppo di farmaci ad alto impatto. L'organizzazione è stata citata dai media nazionali, tra cui New York Times, STAT, CNN, People e altri. Per saperne di più emilysentourage.org. 

About Cystic Fibrosis Trust

Cystic Fibrosis Trust is the only UK-wide charity dedicated to uniting for a life unlimited for everyone affected by cystic fibrosis. We fund vital research, improve care, speak out and race towards effective treatments for all. We won’t stop until everyone with cystic fibrosis can live without limits. The Trust provides confidential advice, support, and information on any aspect of cystic fibrosis, including help with financial support. The work the Trust does is only made possible by the generous donations from supporters. Visit www.cysticfibrosis.org.uk to find out more about cystic fibrosis, the work of the Trust and how you can help.

About Queen’s University Belfast

Queen’s University Belfast is a member of the Russell Group UK research-intensive universities, providing world-class education underpinned by world-class research. Founded as Queen’s College in 1845, it became a university in its own right in 1908. Today, it is an international centre of research and education, with a student-centred ethos. Queen’s is globally connected and networked with strategic partnerships across the world. It is a dynamic and diverse institution, a magnet for inward investment, a patron of the arts and a global player in areas ranging from biomedical and clinical studies to sustainability, and from pharmaceuticals to creative writing.

About OmniSpirant Therapeutics

OmniSpirant is an Irish biotech founded in 2016 to develop first-in-class inhaled regenerative gene therapies. OmniSpirant received EU funding in 2018 via the Horizon 2020 SME Instrument to develop advanced therapies for the treatment of cystic fibrosis. OmniSpirant also received the EU seal of excellence in 2020 and 2022. The company’s new gene therapies are based on its proprietary OmniSome platform technology, which utilises tiny particles, known as extracellular vesicles (EVs), secreted by stem cells carrying gene therapy cargoes. The OmniSome platform of products are currently being developed as first-in-class inhaled regenerative gene therapies for cystic fibrosis, and lung cancer via the Horizon Europe €12.8M INSPIRE consortium. The company also has plans to use this platform to develop treatments for other lung diseases including alpha-1 antitrypsin deficiency (AATD), acute respiratory distress syndrome (ARDS) and idiopathic pulmonary fibrosis (IPF).

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