AskBio Inc., a gene therapy company fully owned and independently operated as a subsidiary of Bayer AG, announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has awarded the prestigious Pioneering Regenerative Medical Product designation (SAKIGAKE) to two of its investigational gene therapy candidates—AB-1005 for Parkinson’s disease and AB-1002 for non-ischemic heart failure. This designation underscores Japan’s commitment to accelerating breakthrough therapies that demonstrate novel mechanisms of action, strong preliminary data, and meaningful potential for addressing severe, underserved conditions.

The SAKIGAKE designation offers several advantages, including priority consultation and significantly accelerated regulatory review, enabling earlier participant access to potentially transformative treatments. According to AskBio’s chief development and medical officer, Dr. Canwen Jiang, the dual recognition reaffirms the company’s mission to advance next-generation gene therapy products that target the root causes of chronic and progressive diseases.

AB-1005, currently in the phase II REGENERATE-PD trial, uses an adeno-associated viral (AAV) vector to deliver the GDNF gene, with the aim of restoring neuronal function and slowing the progression of Parkinson’s disease. The product has previously received multiple global designations, including FDA RMAT, FDA Fast Track, and the UK MHRA Innovation Passport—highlighting its global development momentum. Early clinical results suggest the potential to preserve dopaminergic neurons and address the growing global burden of Parkinson’s disease, which affects more than 10 million people worldwide.

AB-1002, designed as a one-time AAV gene therapy for adults with NYHA Class III non-ischemic heart failure, previously earned FDA Fast Track designation. Its mechanism targets inhibition of protein phosphatase 1 to improve cardiac performance. A phase I/II study reported encouraging safety and clinically meaningful improvements, supporting further development through the ongoing GenePHIT phase II trial across multiple global sites. Heart failure remains one of the world’s largest chronic health challenges, impacting more than 64 million people.

Bayer’s R&D head Christian Rommel emphasized that this is the first SAKIGAKE designation for the company—marking a major milestone in expanding its global gene therapy portfolio while strengthening future pharma manufacturing pipelines for regenerative products.

Both gene therapy candidates remain investigational and unapproved, with safety and efficacy under evaluation. AskBio is also advancing AB-1005 in multiple system atrophy (MSA-P), further broadening its neuroscience pipeline.

Together, these designations reinforce Japan’s leadership in accelerating advanced biopharmaceutical and next-generation therapeutic products, while supporting global innovation in gene therapy manufacturing and development.