CF Foundation adds to investment in 4DMT’s CF gene therapy

Funding supports ongoing study testing inhaled treatment in adults with CF

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Cystic Fibrosis Foundation is making a new investment of up to $11 million in 4D Molecular Therapeutics (4DMT) to support the development of 4D-710, the company’s inhaled gene therapy for cystic fibrosis (CF) that is currently in clinical development.

The funding, which builds on earlier support, brings the foundation’s investment in 4DMT to nearly $32 million, according to a company press release.

“We are honored to receive the CF Foundation’s continued support, which underscores our shared mission to bring transformative new treatments to people with CF,” said David Kirn, MD, co-founder and CEO of 4DMT.

As part of the investment, the CF Foundation and 4DMT are forming a joint steering committee of experts to help guide development of 4D-710.

The funding and steering committee will help support an ongoing Phase 1/2 study called AEROW (NCT05248230), which is testing 4D-710 in adults with CF who are ineligible for or intolerant to CFTR modulator therapy. Some of the funding will support a redosing study, in which patients initially given a single dose of 4D-710 in the Phase 1 part of the trial will receive a second dose at least one year later.

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Phase 2 recruiting, Phase 3 planned

The investment will also support the Phase 2 part of the trial, which is recruiting participants at sites across the U.S. All participants in the Phase 2 portion will receive a single dose of 4D-710 at a set dose, with the main goal of evaluating safety. 4DMT aims for the dose tested in the Phase 2 study to be suitable for future testing that would support an eventual regulatory approval. The company noted that the new funding will also support plans for a future Phase 3 study.

“This funding and strategic support are critical as we advance 4D-710 through Phase 2, with the goal of delivering a durable, redosable, and variant-agnostic [suitable for patients with any disease-causing mutation] genetic medicine with the potential to become a foundational treatment for individuals with CF with remaining unmet pulmonary needs,” Kirn said.

Interim data from AEROW announced last year showed that the therapy may at least stabilize lung function in adults with CF.

“We look forward to sharing interim Phase 1 data from the AEROW clinical trial, including functional durability results one to three years post-dosing, and providing a program update by year-end,” Kirn said.

CF is caused by mutations in the CFTR gene, which encodes a protein called CFTR that’s important for regulating mucus production. In CF, the CFTR protein doesn’t work correctly or is entirely absent, leading to the production of thick and sticky mucus that builds up in the lungs and other organs to drive most CF symptoms.

4D-710 is designed to deliver a healthy copy of the CFTR gene to lung cells, allowing production of functional CFTR protein to normalize mucus production and ease respiratory symptoms. This approach is designed to be equally effective in all CF patients. By contrast, CFTR modulators are medicines that can improve the function of the CFTR protein, but not all disease-causing mutations respond to these therapies.

The gene therapy is designed to deliver its genetic payload using a viral vector — a virus that’s been modified so it won’t cause infection — called A101.

“Our next-generation A101 vector utilized in 4D-710 was invented for efficient aerosol delivery and transduction throughout the lung airways and was designed to enable repeat dosing to maintain clinical benefit over time,” Kirn.

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