Japan Grants Pioneering AAV Gene Therapy Designations to AskBio for Parkinson’s and Heart Failure Programs

BERLIN, Germany, and DURHAM, N.C., USA – December 9, 2025 — AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio’s investigational AAV (adeno-associated virus) gene therapy programs.

This prestigious Japanese designation, which expedites regulatory review and consultation, was awarded to:

1. AB-1005: An AAV gene therapy for the treatment of Parkinson’s disease (PD).

2. AB-1002: An AAV gene therapy for the treatment of non-ischemic heart failure with reduced left ventricular ejection fraction (NYHA Class III).

Accelerated Path for Central Nervous System AAV Therapy

AB-1005, an investigational AAV gene therapy, utilizes an adeno-associated viral (AAV) vector for the delivery of the glial cell line-derived neurotrophic factor (GDNF) gene. The therapy, currently in the Phase II REGENERATE-PD trial, aims to restore neuronal function and potentially slow the progression of moderate-stage PD by providing stable and continuous expression of GDNF in the brain.

This is the latest in a series of key designations for this AAV therapeutic, which previously received FDA Regenerative Medicine Advanced Therapy (RMAT), FDA Fast Track, and UK MHRA Innovation Passport designations, underscoring its significant global potential as an AAV treatment for this progressive neurodegenerative disease.

Heart Failure AAV Program Recognized

AB-1002 is an investigational AAV gene therapy designed as a one-time treatment for adults with NYHA Class III non-ischemic heart failure. The AAV gene therapy aims to improve cardiac function by targeting protein phosphatase 1 inhibition.

• Clinical Status: AB-1002 is currently being evaluated in the Phase II GenePHIT trial and previously received FDA Fast Track designation.

• Mechanism: The one-time AAV gene therapy is administered directly to the heart via intracoronary artery infusion, promoting the production of a modified protein designed to block the action of a protein linked to congestive heart failure (CHF).

“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative AAV gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.

The SAKIGAKE designation reinforces the shared commitment of AskBio and Bayer to delivering breakthrough AAV science to patients worldwide, facilitating earlier participant access to these potentially transformative treatments.