Overview of Cystic Fibrosis
Cystic fibrosis (CF) is a rare, potentially fatal, genetic disease impacting close to 40,000 people in the U.S. and at least 105,000 globally.1 In people with CF, the cystic fibrosis transmembrane conductance regulator (CFTR) protein is dysfunctional due to mutations in the CFTR gene. This reduces chloride transport to the cell surface leading to reduced hydration and thick and sticky mucus in multiple organs. The thickened mucus in the lungs can cause breathing difficulties and recurrent infections, leading to lung inflammation, tissue damage, and respiratory failure.2
Current Treatments and Unmet Needs
Management of this progressive disease includes several interventions, including CFTR modulators to correct the malfunctioning protein made by the CFTR gene, mucolytics to thin and loosen mucus in airways, and antibiotics to treat frequent lung infections.3
Although highly effective CFTR modulators have recently changed the outlook for patients and their caregivers, up to 10% of patients have mutations not known to be responsive to CFTR modulators (genotype ineligible).4
TAVT-135 is an inhaled peptide-conjugate being explored as a treatment for cystic fibrosis. If successful, TAVT-135 could benefit all CF patients, independent of CFTR mutation. TAVT-135 has a unique mechanism of action as an artificial chloride ion transporter. Restoring chloride ion balance in the airways is expected to prevent mucus thickening and accumulation, and to slow the progressive deterioration of lung function.
Initial in-vitro and ex-vivo experiments have successfully demonstrated transport of chloride ions without any cell toxicity. Tavanta is conducting IND-enabling studies to bring TAVT-135 into the clinic as soon as possible.
1 Guo J, Garratt A, Hill A. Worldwide rates of diagnosis and effective treatment for cystic fibrosis. J Cyst Fibros. 2022 May;21(3):456-462.
2 Almughem FA, Aldossary AM, Tawfik EA, et al. Cystic Fibrosis: Overview of the Current Development Trends and Innovative Therapeutic Strategies. Pharmaceutics. 2020 Jul 2;12(7):616.
3 Cystic Fibrosis Foundation Drug Development Pipeline: https://apps.cff.org/trials/pipeline/
4 Allen L, Allen L, Carr SB, et al. Future therapies for cystic fibrosis. Nat Commun. 2023 Feb 8;14(1):693.