Clinical Trials in Progress

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.