AskBio's Dual Breakthroughs Gain Japan's Prized SAKIGAKE Status

RESEARCH TRIANGLE PARK, NC – December 09, 2025

In a move that signals a significant acceleration for advanced therapeutics, AskBio Inc., a gene therapy subsidiary of Bayer AG, announced that two of its investigational programs have received the prestigious Pioneering Regenerative Medical Product (SAKIGAKE) designation from Japan’s Ministry of Health, Labour and Welfare (MHLW). The dual designations for AB-1005, a potential treatment for Parkinson’s disease, and AB-1002, targeting congestive heart failure, place these therapies on an expedited regulatory track in one of the world's most advanced healthcare markets.

This recognition is more than just a procedural step; it represents a major validation of AskBio’s scientific platform and a strategic victory for Bayer's growing ambitions in gene therapy. For the millions of patients in Japan and globally suffering from these debilitating chronic conditions, it offers a tangible beacon of hope, potentially shortening the timeline to access treatments that aim to address the root causes of their diseases.

Japan's Regulatory Gambit: The SAKIGAKE Advantage

The SAKIGAKE designation is Japan's answer to accelerating the development of truly innovative medicines. Established in 2015, the system is designed to identify and fast-track breakthrough therapies that demonstrate prominent efficacy in early clinical trials and target severe diseases with high unmet medical needs. It is a core component of Japan’s strategy to position itself as a global leader in the field of regenerative medicine.

For companies like AskBio, the benefits are substantial. The designation provides access to priority consultations, a dedicated review manager from the Pharmaceuticals and Medical Devices Agency (PMDA), and a significantly compressed review timeline, targeting an approval decision in as little as six months, compared to the standard one-year process. This streamlined pathway is critical for therapies like AB-1005 and AB-1002, which have already garnered similar fast-track designations from the U.S. FDA and UK's MHRA, highlighting their global potential.

“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “This recognition not only accelerates regulatory review but also reaffirms our commitment to delivering advanced treatments to those living with serious chronic diseases that lack therapies targeting root causes.”

Targeting the Root Causes of Chronic Disease

The true innovation behind AskBio's candidates lies in their approach. Rather than managing symptoms, these one-time gene therapies are designed to intervene at a fundamental biological level.

For Parkinson’s disease, which affects an estimated 150,000 people in Japan and over 10 million worldwide, the current treatment paradigm is limited to symptomatic relief. AB-1005 aims to change that. It uses a harmless adeno-associated viral (AAV) vector to deliver the gene for glial cell line-derived neurotrophic factor (GDNF) directly to the brain. The goal is to promote the survival and restoration of dopamine-producing neurons, the very cells that are lost in Parkinson's, thereby potentially slowing or even reversing disease progression. Early results from a Phase Ib trial were encouraging, showing the therapy was well-tolerated and led to clinically meaningful improvements in motor function for patients with moderate-stage disease. The therapy is now being evaluated in a larger, global Phase II trial, REGENERATE-PD.

Similarly, AB-1002 addresses the dire unmet need in congestive heart failure, a condition affecting 1-2 million people in Japan and over 64 million globally. This investigational gene therapy is administered directly to the heart and is designed to inhibit an enzyme called protein phosphatase 1, which is linked to poor cardiac function. By blocking this enzyme, the therapy intends to improve the heart's ability to pump blood. Data from the Phase I trial, published in the prestigious journal Nature Medicine in October 2025, showed a favorable safety profile and clinically meaningful improvements in heart function, including left ventricular ejection fraction (LVEF). The therapy is now advancing in a Phase II trial known as GenePHIT.

A Strategic Win for Bayer's Gene Therapy Ambitions

The dual SAKIGAKE designations are a significant milestone not just for AskBio, but for its parent company, Bayer. The German pharmaceutical giant made a bold move into the gene therapy space with its 2020 acquisition of AskBio in a deal worth up to $4 billion. This latest regulatory success serves as a powerful validation of that high-stakes investment.

For Bayer, this is the first time any of its products have received the SAKIGAKE designation, marking a pivotal moment in its efforts to build a world-class cell and gene therapy platform. AskBio operates as an independent subsidiary, a strategy designed to preserve its innovative culture while leveraging Bayer's global scale. The progress of AB-1005 and AB-1002 strengthens Bayer's pipeline in neurology and cardiology, two of its core focus areas.

Christian Rommel, Global Head of Research and Development for Bayer's Pharmaceuticals Division, commented on the achievement: “Receiving the designation in Japan, which is a first for Bayer, marks an important milestone in expanding global access to pioneering therapies and reinforces our shared commitment to delivering breakthrough science to improve outcomes for patients worldwide.”

This progress, combined with Bayer's other assets like BlueRock Therapeutics—which is also developing a cell therapy for Parkinson's—cements the company's position as a formidable competitor in the rapidly evolving field of regenerative medicine. While the path to final approval is still long and contingent on the success of ongoing Phase II trials, the accelerated pathway in Japan provides a clear line of sight to the market. For the millions of patients in Japan and worldwide, the progress of these trials represents a tangible source of hope in the fight against two of modern medicine's most formidable diseases.