Gene Therapy
Regulatory & Standards

FDA and European Commission Both Grant Orphan Designation to AviadoBio’s Gene Therapy Product Targeting Frontotemporal Dementia

Anna Osborne
27 April 2022

The pre-clinical stage gene therapy company AviadoBio has announced both the FDA and European Commission have granted orphan drug designation to its one-time gene therapy – AVB-101 – for the treatment of frontotemporal dementia (FTD).

AVB-101 is an investigational AAV gene therapy for FTD patients who experiences mutations in the Progranulin (GRN) gene. AVB-101 works by delivering a functional GRN copy throughout the CNS, allowing normal levels of proganulin to be restored and disease progress to be arrested or slowed.

“Achieving orphan designations in both the US and EU represents an important milestone in bringing AVB-101 to FTD patients who are living with a debilitating disease without treatment options […] We look forward to initiating clinical trials for AVB-101 later in 2022,” commented Lisa Deschamps, CEO, AviadoBio.

The orphan drug designation’s received from both the FDA and EU will help support AviadoBio develop AVB-101 to treat this serious life-debilitating and life-threatening rare condition.

Focusing specifically on transforming the lives of those living with neurodegenerative disorders through gene therapies, AviadoBio’s platform combines: neuroscience expertise, next-generation gene therapy technology – based on pioneering research from King’s College London and UK Dementia Research Institute -, with a novel neuroanatomical approach to drug delivery.

Frontal lobe dementia affects around 55,000 patients in the U.S., and over 100,000 in the E.U. As it stands there are no approved treatments for this debilitating disease. FTD patients suffer from personality changes, loss of empathy and disinhibition. With time patients often require significant care and will typically survive seven to ten years from disease onset.

“FTD is a devastating condition, for patients, their families and friends, for which there is no effective treatment. A one-time treatment of AVB-101 has the potential to supplement the missing gene on a long-term basis, thereby stopping FTD in its tracks. As a clinician who has been treating this patient group for many years, I am looking forward to seeing how this therapeutic candidate performs in clinical studies,” concludes Prof. Christopher Shaw, Co-founder and Chief Scientific and Clinical Advisor, AviadoBio.

Source: AviadoBio Press Release