A year removed from its initial grant of $525,000, Cystic Fibrosis Foundation Therapeutics (CFFT) is providing $3 million in additional funding to 4D Molecular Therapeutics (4DMT).
The recent grant is intended to continue the development of 4D-710, a gene therapy and an investigational new drug (IND) candidate for cystic fibrosis (CF).
CF is a life-threatening inherited disorder caused by a faulty or missing a cystic fibrosis transmembrane conductance regulator (CFTR) protein that results in lung and digestive system damage. Common symptoms include frequent lung infections, heavy coughing and difficulty breathing.
Approximately 30,000 people in the United States are affected by CF, and currently-approved treatments by Vertex only help a portion of CF patients. Gene therapy could potentially cure patients of the condition via a proprietary AAV vector that delivers a functional copy of the CFTR gene to the airway and lungs.
It is expected that the new funding, in addition to matching funds from 4DMT, will allow for the completion of remaining studies necessary to file an IND application with the U.S. Food and Drug Administration (FDA). If the application is accepted, clinical trials of 4D-710 will begin.
“We are very pleased and honored to have been selected to receive additional IND candidate development funding from Cystic Fibrosis Foundation Therapeutics Inc., a world leader in CF research and development,” said David Kirn, MD, co-founder and CEO of 4DMT in a press release.
“The CF Foundation’s support for our CF product development further validates the potential of our Therapeutic Vector Evolution platform, and the products engineered from our optimized and proprietary AAV vectors. This additional investment will accelerate IND enabling studies and initiation of clinical testing in CF patients with high unmet medical needs.”
Previously, 4DMT has also partnered with Pfizer, Roche, uniQure, AGTC, and Benitec.
CFFT is a non-profit drug discovery and development affiliate of the CF Foundation. The CF Foundation has a long and successful history of funding companies to develop CF treatments. As an early investor of Vertex Pharmaceuticals, the foundation later sold their future royalty rights of Vertex’s Kalydeco for $3.3 Billion in 2015.