Passage Bio, a company that specializes in developing genetic therapies, administered the first dose in a clinical trial evaluating a potential treatment for people diagnosed with frontotemporal degeneration associated with a variation in the GRN gene, or FTD-GRN.  

Variations in the GRN gene can result in an inability for the body to produce the protein progranulin (PGRN). Insufficiencies in PGRN have been linked to hereditary FTD. 

The PBFT02 gene therapy is a single-dose injection that delivers a working, un-mutated copy of the GRN gene into the body of the person diagnosed through an engineered virus known as a vector.  

Mark Forman, MD, Passage Bio’s chief medical officer, said that this approach “provides a potential opportunity to achieve higher than normal levels of PGRN in the [central nervous system], thereby overcoming the PGRN deficiency in GRN mutation carriers with a diagnosis of early symptomatic FTD-GRN.” 

The first dose administered in this trial represents “an important milestone for the Passage Bio team and for the PBFT02 program,” Edgar (Chip) Cale, interim CEO of Passage Bio, said in a press release. “We look forward to continuing our important work to develop PBFT02 as a potential treatment option for the thousands of people living with FTD-GRN. We are grateful for the support from the families and clinical trial investigators who have chosen to participate in our studies.” 

“FTD-GRN is a devastating disease with no approved disease-modifying therapies, and we are hopeful this trial will provide evidence that PBFT02 could become a meaningful treatment option for adults living with FTD-GRN,” Dr. Forman added. “We look forward to building on our preclinical data with this Phase 1/2 trial.” 

Passage Bio is still recruiting participants for the clinical trial, known as upliFT-D. People with an FTD-GRN diagnosis who are interested can find more information on clinicaltrials.gov.  

A list of other clinical trials that are actively recruiting can be found here.