Business
4D Molecular Therapeutics Announces FDA Clearance of IND Application for 4D-710, an A101 Vector-based, Aerosol-delivered Gene Therapy for the Treatment of Cystic Fibrosis Lung Disease
Initiation of 4D-710 Phase 1/2 clinical trial sites expected before year-end EMERYVILLE, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics
About this update from 4d Molecular Therapeutics, Inc.
[{"type":"text","content":"Initiation of 4D-710 Phase 1/2 clinical trial sites expected before year-end\nEMERYVILLE, Calif., Oct. 06, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for 4D-710 for the treatment of patients with cystic fibrosis. The active IND enables the initiation of 4D-710 Phase 1/2 clinical study sites, which is expected before year-end. “4D-710 is an aerosol-delivered gene therapy that has promise as a mutation agnostic treatment for patients with cystic fibrosis lung disease,” said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT. “4D-710 is designed to express high levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein directly within target cells lining the airway, enabling CFTR mutation-independent activity, and was also invented for resistance to pre-existing antibodies in humans. We plan to focus initially on the approximately 10-15% of all patients whose disease is not amenable to existing modulator medicines that target the CFTR protein. Ultimately, we believe 4D-710 has the potential to treat a broad cystic fibrosis patient population, including those patients treated with current CFTR modulators, all of which require daily dosing over the patient’s lifetime and generally result in only partial correction of lung function.” “4D-710 is comprised of our targeted and evolved vector, A101, and a microCFTR transgene,” said Robert Fishman, M.D., Chief Medical Officer of 4DMT. “A101 was invented not only for aerosol delivery diffusely throughout the lung airways and alveoli, but also for penetration through the mucus barrier and for resistance to pre-existing antibodies, both of which are potentially key attributes for successful treatment of these patients. 4D-710 has the potential to be a differentiated therapy for the treatment of cystic fibrosis lung disease due to its potentially corrective mechanism, expected ability to treat patients independent of CFTR mutation and resistance to AAV antibodies.” The Phase 1/2 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion trial of 4D-710 in patients (n=~18) with cystic fibros...