AstraZeneca, 4D team up to develop lung disease gene therapies

Research & development (Image: AstraZeneca)
The collaboration is underpinned by the progress of AAV vector technology. (AstraZeneca)

AstraZeneca and 4D Molecular Therapeutics have joined forces to develop gene therapies against chronic lung diseases. The collaboration will use 4D’s adeno-associated virus (AAV) vectors to create therapies that insert genes that code for therapeutic proteins into human cells.

Knowledge of the genetic roots of many chronic lung diseases, such as chronic obstructive pulmonary disease (COPD), has fueled interest in the use of gene therapies for years. However, while the interest has led to research into targets including alpha-1-antitrypsin, it has yet to translate into gene therapies with validated clinical efficacy.

AstraZeneca’s MedImmune and 4D are setting out on a path they hope will lead to such therapies. 4D is taking the lead on early steps in the process that are in its wheelhouse, such as vector discovery and engineering, optimization and process development. MedImmune will take over early in clinical development, enabling it to leverage AstraZeneca’s vast experience in respiratory diseases to power the programs forward.

The partners have yet to talk about the collaboration in much detail. We know the plan is to equip AAV vectors to target dividing and nondividing cells in accessible tissue. Without integrating genetic material into the host genome, the vectors will drive production of proteins that interact with disease pathways. 4D and MedImmune have yet to share details such as the targets and indications.

AstraZeneca’s extensive interest in chronic lung diseases such as COPD means several indications could be on its hit list. In some cases, knowledge of the genetic roots of these indications has been available for years. What has been missing, as MedImmune sees it, is a vector to safely and effectively get genetic material to target cells. That barrier may now be coming down.

“Rapid advances in AAV therapy make this a promising tool to advance innovation in chronic lung disease, particularly in areas of high unmet need,” Roland Kolbeck, Ph.D., MedImmune’s VP of respiratory R&D, said in a statement.