Defining Target Product Profiles (TPPs) for Bacteriophage Therapies to treat CF lung infections
The CF AMR Syndicate has joined forced with the TRAILFINDER CF Innovation hub to deliver TPPs to catalyse and guide the development of phage therapies to treat lung infections in CF.
These TPPs are based around the needs and priorities of people with CF and are intended to outline the necessary characteristics for phage-based therapies to treat CF lung infections. They will serve as guiding documents for the development of such therapies and provide insights into the development of both conventional and modified phage products. It is anticipated that this work will help drive new research projects and investment into phage therapies to treat chronic lung infections in CF and beyond.
The Need
Lung infections remain a cause of morbidity and mortality in CF. Despite major advances in CF care with the introduction of CFTR modulators, lung infections remain a clinical challenge driving disease progression and treatment burden. Problematic pathogens such as Mycobacterium abscessus, Pseudomonas aeruginosa, and Burkholderia cepacia complex continue to be associated with worse outcomes and increased treatment complexity. Treatment options for pathogens like M. abscessus in particular are limited, often involving lengthy, complex, and toxic multidrug regimens.
Antibiotics remain a key therapeutic for managing infections, but prolonged and repeated use contributes to antimicrobial resistance, drug toxicity, and intolerable side effects- issues that are becoming increasingly important in an aging CF population.
Phage-based therapeutics offer a promising and targeted approach to tackling these infections. Phages are viruses that specifically infect and lyse (or breakdown) bacteria, including antibiotic-resistant strains. They can penetrate biofilms offering a mechanism to restore treatment options where antibiotics fail.
Compassionate-use experience supports their safety and biological activity, however access is currently restricted to unlicensed, individualised preparations. Phage products developed under standard drug authorisation pathways could provide a scalable and regulated means to meet this unmet medical need ensuring consistent manufacturing, quality control, and wider clinical availability for CF and other chronic lung infections.
Our Approach
The approach followed the CF AMR Syndicate’s established patient-centric framework for TPP development, previously used for antimicrobial therapeutics and diagnostics for CF. The process included three key phases:
Development of TPPs
01
Scoping phase:
We conducted a systematic review of the preclinical and clinical pipeline for phage therapeutics targeting lung infections in CF and other chronic respiratory conditions. During this phase, we also identified key stakeholders to engage with.
02
Stakeholder engagement and drafting phase:
We carried out focus groups and one-to-one interviews with people living with CF, their families and loved ones, clinicians, industry partners, and other experts. These discussions helped identify unmet needs and define the key characteristics of the TPPs.
03
Consensus-building, publication and dissemination phase:
Draft TPPs were evaluated through a Delphi-style survey process to build consensus among stakeholders. Findings were then discussed during a virtual symposium, which helped refine and finalise the documents before publication and dissemination.
Access the TPPs for Phage
Please complete the form to request a free copy of the TPPs and a digital copy will be sent to you when it is available.