Emily’s Entourage awards $220K toward nonviral CF gene therapy

OS001 is designed to deliver a full-length, working copy of CFTR gene to lungs

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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A hand holds up a coin against a backdrop of dollar signs and packets of money.

The nonprofit Emily’s Entourage has awarded $220,000 to a project to develop a nonviral and inhalable gene therapy for cystic fibrosis (CF).

The grant was awarded to Lorraine Martin, PhD, professor at Queen’s University Belfast, U.K. Martin is developing the therapy in collaboration with OmniSpirant Therapeutics. The Cystic Fibrosis Trust is also funding the project with a grant from its Venture and Innovation Award fund.

“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,” Martin said in a press release.

OmniSpirant CEO Gerry McCauley said the first-in-class regenerative gene therapy OS001 has already shown “promising results in preclinical disease models.”

OS001 uses extracellular vesicles (tiny sacs) produced from stem cells to deliver a full-length, working copy of the CFTR gene to the lungs of CF patients. Mutations in the CFTR gene, leading to the protein’s impairment or absence, are the root cause of CF.

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By restoring the production of full-length CFTR, the therapy may treat CF patients regardless of their mutation, including those with nonsense mutations, which account for about 10% of all CF cases and result in a shortened CFTR protein that’s degraded by the cell.

“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,” McCauley said. “We are excited to progress the development of OS001 with [Emily’s Entourage] 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.

Current CFTR modulator therapies work by increasing the protein’s function, helping to ease CF symptoms. Their effectiveness depends on the type of underlying mutation, however.

Gene therapies traditionally use viruses modified in the lab to be harmless as vehicles to deliver a working version of a gene to targeted cells. However, these viral vectors can trigger an immune response that weakens the therapy’s effectiveness.

“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, chief scientific officer of Emily’s Entourage.

“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,” said Lucy Allen, director of research and healthcare data at the Cystic Fibrosis Trust.

The funding will allow Martin’s team to conduct more preclinical studies. “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,” she said.

Besides CF, OmniSpirant is also using its OmniSome platform to develop a gene therapy for lung cancer, along with treatments for acute respiratory distress syndrome and idiopathic pulmonary fibrosis.

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