Clinical Trials in Progress

Following up on Liver Cancer Awareness Month, which is observed annually in October by the patient and clinician communities, CGTLive has decided to take a closer look at this ongoing study.

The REVEAL adolescent and adult trial and the separate phase 1/2 REVEAL Pediatric Study are both evaluating the gene therapy TSHA-102 in Rett syndrome populations.

Following up on World Lung Cancer Day, observed annually on the first of August, CGTLive® has decided to take a closer look at this novel cell therapy.

BriaCell initiated the pivotal phase 3 BRIA-ABC trial in October 2023.

In honor of World Brain Day, observed annually on July 22 by patient and clinician communities, CGTLive is taking a closer look at this ongoing study.

The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.

With the company having recently announced that it intends to expand the clinical trial with a new cohort, CGTLive® has decided to take a closer look at this ongoing study.

The phase 1/2 trial is proceeding to its phase 2 trial after safety was demonstrated in its first phase.

In honor of Healthy Vision Month, CGTLive® took a closer look at the clinical trial design for this novel treatment.

The trial is set to dose its second cohort following a positive DSMB recommendation in March.

In anticipation of Bladder Cancer Awareness Month, CGTLive® has decided to take a closer look at this noninterventional real-world study.

The more recent data were presented at the 2022 ASGCT Meeting.

In observance of Parkinson's Awareness Month, held annually in April, CGTLive® took a closer look at REGENERATE-PD, a new trial for PD gene therapy AB-1005.

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a closer look at the clinical evaluation of hearing loss gene therapy DB-OTO.

AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.

The trial is assessing satri-cel CAR T-cell therapy, which is currently on clinical hold due to CMC concerns.

Exa-cel was recently approved for the treatment of SCD in the US, but data from the pivotal clinical trial continues to be collected.

December is HIV/AIDs Awareness Month and CGTLive is taking a look at Excision's first-in-human CRISPR therapy trial for people with HIV-1.

In observance of Crohn and Colitis Awareness Week, observed from December 1 to 7, CGTLive™ took a closer look at the clinical evaluation of the MSC-derived therapy, ExoFlo.

The PDUFA date for Iovance’s lead TIL lifileucel for melanoma was recently pushed back to February 24, 2024.

TN-201 uses an AAV vector-based approach to deliver a functional copy of the disease-targeted MYBPC3 gene to cardiomyocytes.

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.

Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.

Notably, the trial is assessing intravenous administration of PRGN-3005 against intraperitoneal administration in 2 separate cohorts.

Acclaim-1, Acclaim-2, and Acclaim-3 will evaluate Reqorsa in combination with osimertinib, pembrolizumab, and atezolizumab, respectively.

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.

The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.

CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.