AviadoBio’s Gene Therapy Candidate Receives Orphan Designation from Food and Drug Administration and European Commission in Frontotemporal Dementia

April 27, 2022

• FDA and EC decisions reinforce severity of unmet medical need with this rare, aggressive form of dementia

• AviadoBio to start clinical trials with its gene therapy candidate later this year

London, UK, April 27, 2022 — AviadoBio, a pioneering, pre-clinical stage, gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted orphan designation to AVB-101, a one-time gene therapy for the treatment of frontotemporal dementia (FTD).

AVB-101 is an investigational, adeno-associated virus (AAV) gene therapy designed for FTD patients with mutations in the Progranulin (GRN) gene. AVB-101 is designed to slow or arrest disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore normal progranulin levels. FTD is a debilitating form of early onset dementia that currently has no approved therapies.

“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 are committed to advancing our proprietary gene therapy platform and working with regulatory authorities and the FTD community to bring a desperately needed option to patients. We look forward to initiating clinical trials for AVB-101 later in 2022,” said Lisa Deschamps, CEO, AviadoBio.

“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,” said Prof. Christopher Shaw, Co-founder and Chief Scientific and Clinical Advisor, AviadoBio.

Orphan drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition that affects fewer than 200,000 individuals in the U.S. The EC grants orphan designation to encourage the development of medicines intended for the treatment of a seriously debilitating or life-threatening rare condition, defined as one that affects fewer than five in 10,000 people in the EU.

About AviadoBio

At AviadoBio our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.

About Frontotemporal Dementia and AVB-101

Frontotemporal dementia (FTD) is one of most common forms of dementia in individuals under the age of 65 (second after Alzheimer’s disease). It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U and is characterized by a rapid decline in behaviour, executive function and/or language with an average of onset in the mid-50s. Patients experience a change in personality, apathy, loss of empathy, inappropriate social behaviour, and disinhibition. Unfortunately, with time, patients require significant supportive care and survive seven to ten years after disease onset. Currently there are no approved medicines for the treatment of frontotemporal dementia.

Approximately 30-40% of FTD cases are familial and linked to autosomal dominant mutations in three genes including GRN (progranulin). AviadoBio’s AVB-101 is an investigational, one-time, adeno-associated virus (AAV) gene therapy for patients with Frontotemporal Dementia (FTD) with mutations in the GRN gene. AVB-101 is designed to slow or arrest disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore normal progranulin levels.


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