Developing a one-time gene editing treatment for cystic fibrosis

Project Duration – 2 years

Dr Fatwa (Adi) Adikusuma from the University of Adelaide is leading a new gene-editing project that could help everyone with cystic fibrosis (CF), no matter their mutation. Using a new gene-editing tool, the team hopes to insert a healthy copy of the CFTR gene into its natural place in the DNA.  By delivering this through tiny particles, called lipid nanoparticles (LNPs), the goal is to restore normal CFTR function in the lungs, with just a single treatment.

Cure4CF’s funding will support Dr Adikusuma to test and refine this approach using pre-clinical models to:

• Develop and optimise the editing tools for targeted CFTR gene integration
• Optimise the LNP delivery and test two delivery systems in human CF airway cells
• Develop a CFTR model to test the ability of the particles to deliver the gene and restore CFTR function
• Evaluate treatment delivery in the model by inhalation and injection and examine the ability of the treatment to impact CFTR expression and activity.

Why is gene editing needed?

While CFTR modulators have improved outcomes for many people with cystic fibrosis, they are ineffective or unavailable for up to 30% of patients, particularly those with nonsense or rare mutations. Even for responders, these drugs are not curative, requiring lifelong treatment and offering only partial protection against progressive lung disease. Gene editing offers the potential for a one-time, broadly applicable therapy that corrects the underlying genetic defect, restores natural CFTR function, and could halt or reverse disease progression, providing a transformative solution for all individuals with CF.

What is unique about this project?

This project has the potential to develop a universal, mutation-independent approach to correct the CFTR gene, making it effective for all CF patients.  In addition, the team hopes to be able to develop and test the technology to allow a single administration and durable treatment that will use the latest technology in gene-editing science.  A successful gene-editing tool would provide improved health equity and broad impact for people with CF including children.

What will be the pathway to moving this therapy into the clinic?

The team has completed its pre-clinical validation experiments and developed a carefully considered translational pathway. Following this project, the team will commence expanded safety and efficacy studies with regulatory engagement to ensure any resulting treatments are developed in a safe and approved way. 

Once all the safety and efficacy studies have been completed, the team will plan its first in human clinical trials.

About Dr. Fatwa (Adi) Adikusuma

Dr. Fatwa Adikusuma is a molecular biologist and genome engineer dedicated to transforming the treatment of genetic diseases through precise and effective CRISPR-based therapies. His research focuses on harnessing and refining gene editing technologies to correct mutations that cause severe inherited conditions. Dr. Adikusuma’s work aims to uncover the mechanisms that influence CRISPR efficiency and precision, and to translate these advances into therapies for diseases such as Duchenne Muscular Dystrophy (DMD), Retinitis Pigmentosa, Spinal Muscular Atrophy, and Cystic Fibrosis. By developing innovative strategies for gene correction, his team strives to restore health and improve quality of life for patients. He leads the Gene Editing Technology and Therapeutics Laboratory at the University of Adelaide and the South Australian Health and Medical Research Institute (SAHMRI). His research is supported by an NHMRC Emerging Leadership Investigator Grant, and he currently holds an ARC DECRA Fellowship. He was previously awarded a CSIRO Synthetic Biology Future Science Platform Fellowship (2018–2021). Dr. Adikusuma’s contributions have been recognised with multiple Researcher Awards, highlighting his leadership in CRISPR innovation. Beyond academia, he co-founded Gene Editing Therapeutics (GETx), a biotechnology company focused on translating CRISPR research into clinical solutions. The discoveries from Dr. Adikusuma’s team are paving the way for a new generation of genetic medicines, with applications in treating previously incurable diseases, advancing genome engineering technologies, and shaping the future of precision medicine.