After being given more than $3 million from the Cystic Fibrosis Foundation (CFF) in 2017 to expand the development of viral vectors for gene delivery in the lungs, 4D Molecular Therapeutics (4DMT) now will receive up to $14 million from CFF to support the development of a customized gene carrier for the treatment of cystic fibrosis (CF).
The new gene carrier, called 4D-710, is a harmless adeno-associated viral vector (AAV) designed to deliver a functional copy of the CFTR gene — the faulty gene in people with CF — directly to patient lung cells.
“Today’s announcement reaffirms our commitment to invest aggressively to draw the best scientific minds and technology into CF and deliver the next generation of transformative therapies for people with CF,” Michael P. Boyle, MD, president and CEO of the CFF, said in a press release.
“Despite decades of progress in gene therapy, delivery to the lung remains a key challenge and one we are committed to solving,” Boyle said.
4DMT selected this particular vector from its library of viral vectors due to its ability to specifically target cells in the lungs. This key property may potentially enable researchers to overcome one of the major hurdles of developing an effective CF gene therapy — the therapy’s inability to reach the lungs due to their strong protection against foreign substances.
With the new funds, 4DMT plans to test if an inhaled formulation of 4D-710 is able to restore the levels of the CFTR protein and improve lung function in CF patients.
To reach its goal of advancing 4D-710 into clinical testing, the company will have to first develop specific manufacturing procedures to produce the new version of the carrier, and evaluate its safety and tolerability.
The CFF’s funding is part of its Path to a Cure, a $500 million initiative begun by the foundation on October 2019 to promote the development of new treatments tackling the underlying causes of CF, with the final goal of finding a cure for the disease.
The initiative is currently centered on three approaches to tackle the underlying causes of CF: restoring the levels of CFTR protein when none or few exist; repairing the faulty CFTR protein; and replacing the mutated CFTR gene with a functional one.
More information about CFF’s funding programs for projects centered on CF research is available here.
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