- |||||||||| 4D-150 / 4D Molecular Therap
Enrollment change, Trial completion date, Trial primary completion date, Gene therapy: PRISM: 4D-150 in Patients with Neovascular (Wet) Age-Related Macular Degeneration (clinicaltrials.gov) - Nov 5, 2024
P1/2, N=215, Recruiting,
Clinical trials are underway to establish the safety and efficacy of 4D-150 in individuals with wet AMD and DME. N=150 --> 215 | Trial completion date: Nov 2025 --> Nov 2026 | Trial primary completion date: Nov 2024 --> Nov 2025
- |||||||||| 4D-710 / 4D Molecular Therap
Enrollment change, Trial completion date, Gene therapy: AEROW: 4D-710 in Adult Patients with Cystic Fibrosis (clinicaltrials.gov) - Oct 22, 2024
P1/2, N=40, Recruiting,
N=150 --> 215 | Trial completion date: Nov 2025 --> Nov 2026 | Trial primary completion date: Nov 2024 --> Nov 2025 N=24 --> 40 | Trial completion date: Jan 2027 --> Jan 2030
- |||||||||| Review, Journal, Gene therapy: Gene Therapy for Non-Hereditary Retinal Disease: Age-Related Macular Degeneration, Diabetic Retinopathy, and Beyond. (Pubmed Central) - Jun 27, 2024
These therapies offer potential advantages of reduced treatment frequency and enhanced safety profiles, representing a paradigm shift in management towards durable and efficacious cellular-based biofactories. These advancements in gene therapy hold promise for improving outcomes in AMD and addressing the complex challenges of DME and DR, providing new avenues for the treatment of diabetic eye diseases.
- |||||||||| 4D-310 / 4D Molecular Therap
Trial completion date, Trial primary completion date, Gene therapy: 4D-310 in Adults With Fabry Disease and Cardiac Involvement (clinicaltrials.gov) - Apr 8, 2024
P1/2, N=18, Active, not recruiting,
These advancements in gene therapy hold promise for improving outcomes in AMD and addressing the complex challenges of DME and DR, providing new avenues for the treatment of diabetic eye diseases. Trial completion date: May 2028 --> Jan 2030 | Trial primary completion date: May 2024 --> Jan 2026
- |||||||||| 4D-310 / 4D Molecular Therap
Trial completion date, Trial primary completion date, Gene therapy: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov) - Apr 7, 2024
P1/2, N=18, Active, not recruiting,
Trial completion date: May 2028 --> Jan 2030 | Trial primary completion date: May 2024 --> Jan 2026 Trial completion date: Jun 2027 --> Jun 2030 | Trial primary completion date: Jun 2023 --> Jan 2026
- |||||||||| 4D-710 / 4D Molecular Therap
Trial completion date, Trial primary completion date, Gene therapy: AEROW: 4D-710 in Adult Patients with Cystic Fibrosis (clinicaltrials.gov) - Jan 8, 2024
P1/2, N=24, Recruiting,
Enrolment has been initiated for cohort 3 (5x10 14 vg). Trial completion date: Aug 2025 --> Jan 2027 | Trial primary completion date: Aug 2024 --> Dec 2026
- |||||||||| 4D-150 / 4D Molecular Therap
Enrollment change, Trial completion date, Trial primary completion date, Gene therapy: PRISM: 4D-150 in Patients with Neovascular (Wet) Age-Related Macular Degeneration (clinicaltrials.gov) - Aug 31, 2023
P1/2, N=150, Recruiting,
Trial completion date: Aug 2025 --> Jan 2027 | Trial primary completion date: Aug 2024 --> Dec 2026 N=65 --> 150 | Trial completion date: Sep 2026 --> Nov 2025 | Trial primary completion date: May 2026 --> Nov 2024
- |||||||||| 4D-150 / 4D Molecular Therap
Enrollment open, Gene therapy: 4D-150 in Patients With Diabetic Macular Edema (clinicaltrials.gov) - Aug 31, 2023
P2, N=72, Recruiting,
N=65 --> 150 | Trial completion date: Sep 2026 --> Nov 2025 | Trial primary completion date: May 2026 --> Nov 2024 Not yet recruiting --> Recruiting
- |||||||||| 4D-310 / 4D Molecular Therap
Enrollment closed, Trial primary completion date, Gene therapy: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov) - Jan 31, 2023
P1/2, N=18, Active, not recruiting,
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting | Trial primary completion date: Feb 2024 --> Jun 2023
- |||||||||| 4D-125 / 4D Molecular Therap
Enrollment closed, Enrollment change, Gene therapy: 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - Jan 31, 2023
P1/2, N=21, Active, not recruiting,
Recruiting --> Active, not recruiting | Trial primary completion date: Feb 2024 --> Jun 2023 Recruiting --> Active, not recruiting | N=43 --> 21
- |||||||||| 4D-110 / 4D Molecular Therap, Roche
Enrollment closed, Trial completion date, Trial primary completion date: Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia (clinicaltrials.gov) - Jan 19, 2023
P1, N=13, Active, not recruiting,
Recruiting --> Active, not recruiting | N=43 --> 21 Recruiting --> Active, not recruiting | Trial completion date: May 2023 --> Jun 2027 | Trial primary completion date: Nov 2021 --> Jun 2024
- |||||||||| 4D-125 / 4D Molecular Therap
Trial completion date, Gene therapy: 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - Jun 8, 2022
P1/2, N=43, Recruiting,
Recruiting --> Active, not recruiting | Trial completion date: May 2023 --> Jun 2027 | Trial primary completion date: Nov 2021 --> Jun 2024 Trial completion date: Sep 2026 --> May 2029
- |||||||||| 4D-125 / 4D Molecular Therap
Trial completion date, Trial primary completion date, Gene therapy: 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - Mar 4, 2022
P1/2, N=43, Recruiting,
Trial completion date: Sep 2026 --> May 2029 Trial completion date: Sep 2024 --> Sep 2026 | Trial primary completion date: Mar 2024 --> Apr 2026
- |||||||||| 4D-125 / 4D Molecular Therap
Trial completion date, Trial primary completion date, Gene therapy: 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) (clinicaltrials.gov) - Dec 8, 2021
P1/2, N=43, Recruiting,
Trial completion date: Sep 2024 --> Sep 2026 | Trial primary completion date: Mar 2024 --> Apr 2026 Trial completion date: Sep 2023 --> Sep 2024 | Trial primary completion date: Mar 2023 --> Mar 2024
- |||||||||| 4D-310 / 4D Molecular Therap
[VIRTUAL] Development of Novel AAV-Based Gene Therapy for Cardiac Disease () - Mar 14, 2021 - Abstract #ATS2021ATS_4734;
After completing all preclinical and IND-enabling studies in normal and Fabry mice, we performed a dose-ranging study in NHPs using C102 carrying GLA (4D-310)...The tropism of this novel AAV variant, C102, represents a significant advance over conventional AAVs and is well suited for the development of gene therapies for diseases where cardiac involvement is primary or syndromic, such as FD. Since the therapeutic transgene can readily be switched (modularity), the C102 capsid could be the gene therapy vehicle to treat any number of cardiovascular diseases.
- |||||||||| 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-310 / 4D Molecular Therap
Enrollment open, Gene therapy: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (clinicaltrials.gov) - Sep 29, 2020
P1/2, N=18, Recruiting,
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. Not yet recruiting --> Recruiting
- |||||||||| 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.
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