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- 4D-710 / 4D Molecular Therap
Transgene expression and CFTR channel function in human bronchial epithelial cells after addition of CFTR modulators and 4D-710 () - Sep 4, 2024 - Abstract #NACFC2024NACFC_995;
Addition of 4D-710 to ETI results in robust transduction and transgene expression in HBE cells and confers an additive effect on CFTR channel function in CF HBE ALI cultures. These findings support further evaluation of 4D-710 as a therapeutic option for PwCF who have a suboptimal response to CFTR modulators.
- 4D-710 / 4D Molecular Therap
CFTR transgene expression in airway epithelial cells following aerosolized administration of the AAV-based gene therapy 4D-710 to adults with cystic fibrosis lung disease (Clyde Auditorium) - Mar 22, 2024 - Abstract #ECFS2024ECFS_279;
CFTR protein levels exceeded levels in normal lung samples, supporting exploration of lower doses. Enrolment has been initiated for cohort 3 (5x10 14 vg).
- 4D-710 / 4D Molecular Therap
AAV mediated gene therapy for cystic fibrosis (4D-710) (Petree Hall D) - Mar 9, 2023 - Abstract #ASGCT2023ASGCT_237;
- 4D-710 / 4D Molecular Therap
AAV mediated gene therapy for cystic fibrosis: interim results from a phase 1/2 clinical trial (Hall E) - Feb 27, 2023 - Abstract #ECFS2023ECFS_220;
P1/2
Initial data from a Phase 1/2 clinical trial evaluating 4D-710 adults with CF lung disease demonstrated evidence of tolerability and successful transgene delivery and expression in the lung. Additional data from cohort 2 will be presented.
- 4D-710 / 4D Molecular Therap
Update on AAV-mediated CFTR gene delivery (4D-710) (108 AB) - Jul 8, 2022 - Abstract #NACFC2022NACFC_36;
- || 4D-710 / 4D Molecular Therap
New P1/2 trial, Gene therapy: 4D-710 in Adult Patients With Cystic Fibrosis (clinicaltrials.gov) - Feb 20, 2022
P1/2, N=21, Recruiting, Sponsor: 4D Molecular Therapeutics
- 4D-710 / 4D Molecular Therap
[VIRTUAL] Identification and Characterization of a Novel AAV Capsid and Product for the Treatment of Cystic Fibrosis () - Mar 14, 2021 - Abstract #ATS2021ATS_3078;
We showed that our gene replacement therapy A101-CFTR∆R (4D-710) is capable of safe, robust, and widespread transduction and transgene expression throughout the primate lung with minimal systemic exposure. The tropism profile after aerosol delivery in NHPs, coupled with its resistance to human Abs and transduction efficiency in human lung cells, represents a significant advance over existing AAV serotypes for the development of gene therapies for cystic fibrosis and other lung diseases.
- 4D-710 / 4D Molecular Therap
[VIRTUAL] 4D-A101 IDENTIFIED BY DIRECTED EVOLUTION IN NON-HUMAN PRIMATES DEMONSTRATES ROBUST GENE DELIVERY BOTH IN VITRO AND IN VIVO () - Aug 26, 2020 - Abstract #NACFC2020NACFC_281;
We have identified a novel AAV vector, 4D-A101, that when delivered by aerosolization is capable of safe, robust and widespread transduction and transgene expression throughout the primate lung with minimal systemic exposure. The tropism profile of this novel AAV vector after aerosol delivery in NHPs, coupled with its resistance to human Abs and transduction efficiency in human lung cells, represents a significant advance over existing AAV serotypes for the development of gene therapies for CF.