CGTLive™'s Clinical Trials in Progress feature series offers a look into ongoing clinical trials of various novel cell, gene, regenerative, and engineered medicines that are being evaluated across the spectrum of disease.
Tenaya's Phase 1b MyPeak-1 Trial is Testing the Waters in Gene Therapy for Hypertrophic Cardiomyopathy
TN-201 uses an AAV vector-based approach to deliver a functional copy of the disease-targeted MYBPC3 gene to cardiomyocytes.
REGENXBIO’s ABBV-RGX-314 Trials Aim to Reduce Treatment Burden in Inherited Retinal Diseases
The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.
bluebird's HGB-210 Trial Hopes to Support First SCD Gene Therapy Approval
Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.
Precigen Trying Its Hand at CAR-T for Ovarian Cancer in Phase 1/1b Trial
Notably, the trial is assessing intravenous administration of PRGN-3005 against intraperitoneal administration in 2 separate cohorts.
Genprex Takes a Three-Pronged Approach to Assessing Reqorsa for Lung Cancer
Acclaim-1, Acclaim-2, and Acclaim-3 will evaluate Reqorsa in combination with osimertinib, pembrolizumab, and atezolizumab, respectively.
First in Human: PROCLAIM and upliFT-D Trials Bring Gene Therapy to Frontotemporal Dementia
With up to 20% of incidences of FTD driven by genetic mutations, multiple gene therapy companies have initiated clinical trials of AAV-mediated treatments for a patient population with little to no options.
Imagine-01 Aiming to Evaluate Safety and Efficacy of GM1 Gangliosidosis Gene Therapy
The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.
PR001 Hopes to Address Underlying Mutations in Neuronopathic Type 2 Gaucher Disease
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
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