RARE Daily

4D Molecular Signs New Agreement with Cystic Fibrosis Foundation

June 17, 2020

Rare Daily Staff

One day after closing a $75 million financing round, 4D Molecular Therapeutics announced a new agreement with Cystic Fibrosis Foundation to develop precision gene medicines for cystic fibrosis.

An investor in the financing, the Cystic Fibrosis Foundation’s investment builds upon previous work with 4D Molecular to advance new therapies for cystic fibrosis.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease caused by mutations in the CFTR gene that lead to a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein. There are approximately 2,000 known mutations in the CFTR gene, some of which lead to CF by creating non-working or too few CFTR proteins at the cell surface that results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

4DMT’s gene therapy approach holds promise for the treatment of CF by using a proprietary and optimized AAV vector to deliver a functional copy of the CFTR gene to the airway and lungs to restore function and alleviate disease symptoms. 4DMT’s wholly owned experimental candidate for the treatment of CF, 4D-710, is comprised of a transgene insert encoding for the CFTR gene and 4DMT’s proprietary vector 4D-A101, a vector that is designed for an efficient, single dose aerosol delivery to the lung airways and with resistance to pre-existing antibodies.

Under their agreement, the CF Foundation will support the completion of research and development activities needed to advance to clinical trials, and the planned phase 1/2 clinical study of 4D-710, 4DMT’s wholly-owned product candidate for the aerosol treatment of CF lung disease.

The CF Foundation has also committed $14 million in new funding, consisting of the $10 million equity investment in 4DMT’s recently completed financing and additional funding contingent upon achievement of a pre-clinical development milestone. 4DMT will match the funds provided and allocate these funds to the ongoing development of 4D-710. 4DMT retains worldwide rights to develop and commercialize 4D-710 and any other potential medicines.

The CF Foundation previously awarded 4DMT more than $3 million in funding to support discovery and development of AAV gene delivery to the lung in 2016.

The CF Foundation’s investment in 4DMT falls under its $500 million Path to a Cure initiative to accelerate development of treatments that address the underlying cause of the disease and a cure for CF.

“This investment will accelerate development activities for 4D-710, our AAV gene therapy product candidate designed to deliver a functional copy of the CFTR gene via one-time aerosol administration,” said David Kirn, co-founder, chairman, and CEO of 4DMT.

Photo: David Kim, co-founder, chairman, and CEO of 4DMT





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