Emily’s Entourage Awards $38,500 Research Grant to Dr. Luka Clarke of the University of Lisboa to Advance Research and Development of Novel Antisense Oligonucleotides (ASOs) for the Treatment of CF

The research is focused on the development of novel splice-switching antisense oligonucleotides (ASOs) for one of the most common CFTR variants in people of African heritage.

Luka A. Clarke, PhD University of Lisboa

Lower Merion, PA – November 20, 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), today announced it has awarded $38,500 in funding to Dr. Luka Clarke, PhD of the University of Lisboa to support his team’s antisense oligonucleotides (ASO) based strategy to correct a population specific CFTR splicing variant.

Despite significant advances in CF research and several approved CFTR modulators, there are still no therapies for individuals with nonsense and certain rare CFTR variants. Dr. Clarke’s research is focused on investigating novel strategies for the correction of one particular rare CFTR gene variant in CF. The project, titled, “CF-Splice: an ASO based strategy to correct a population specific CFTR splicing variant,” aims to develop ASOs to treat individuals with CF who have the rare variant c.2988+1G>A, which is a “splicing” variant that is very commonly found in individuals of African ancestry, including black South Africans, African Americans, Brazilians of African origin, and many others.

The variant c.2988+1G>A causes the cell machinery to leave out a part of the genetic message in the intermediate molecule between gene and protein, termed “messenger RNA”, so that the whole CFTR protein cannot be made. With funding from the grant, Dr. Clarke and his team will design ASOs that mask regions of the message so that the cell’s machinery is tricked into leaving the missing part in, allowing for the full protein to be made, and the CF defect to be bypassed.

“The CF-Splice project represents a crucial step forward in our mission to support the development of innovative treatments for the CF community, particularly those in the final 10% who have not yet seen the benefits of current therapies,” said Chandra Ghose, PhD, Chief Scientific Officer of EE. “Nonwhite individuals are disproportionately represented in the final 10%. Dr. Clarke’s innovative approach holds promise for addressing the unique challenges faced by individuals of African ancestry with CF, and we are excited to support this important work.”

As part of this grant, Dr. Clarke and his team will conduct a series of experiments to determine the most promising strategy by testing ASOs in fluorescent cell models until they find the best one(s). They will explore various different methods of ASO delivery and evaluate the effectiveness of ASOs in organoids/nasal cells from individuals with CF who have the specific variant.

“I’m thrilled to receive this grant from Emily’s Entourage to support the CF-Splice project,” said Dr. Clarke. “This research has the potential to pave the way for treating additional CFTR variants, particularly those of African ancestry, offering hope to those in the final 10% of the CF population who do not benefit from currently available mutation-targeted therapies.”

About Emily’s Entourage’s (EE’s) Grant Program 

Emily’s Entourage’s (EE’s) Grant Program provides grant funding to accelerate research and therapeutic development for people with cystic fibrosis (CF) who do not benefit from existing mutation-targeted therapies. EE provides funding through several grant funding mechanisms, including translational grants, collaborative grants, and preclinical exploratory grants, as well as through venture philanthropy investments.

To date, EE has awarded millions of dollars to multi-disciplinary teams around the world and helped to secure millions of dollars in follow-on funding. To view awarded grants, visit https://www.emilysentourage.org/awarded-grants/. To learn more about EE’s funding opportunities, visit https://www.emilysentourage.org/funding-opportunities/. 

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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