AskBio's Gene Therapies AB-1005 and AB-1002 Receive Japan's Pioneering Regenerative Medical Product Designation
核心洞察
Japan's Ministry of Health, Labour and Welfare granted Pioneering Regenerative Medical Product designation to AskBio's AB-1005 for Parkinson's disease (搜索) and AB-1002 for heart failure (搜索), marking the first such recognition for Bayer.
AB-1005 is an investigational GDNF (搜索) gene therapy currently in Phase II trials for moderate-stage Parkinson's disease (搜索), aiming to restore neuronal function in patients with limited treatment options.
AB-1002 targets protein phosphatase 1 (搜索) inhibition for NYHA Class III heart failure (搜索) patients, with Phase I trial results published in Nature Medicine showing no related adverse events.
AskBio Inc., a gene therapy company wholly owned by Bayer AG, announced that Japan's Ministry of Health, Labour and Welfare has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two investigational gene therapy programs: AB-1005 for Parkinson's disease (搜索) and AB-1002 for non-ischemic heart failure (搜索). This marks the first time Bayer has received this designation in Japan.
The SAKIGAKE designation is awarded to products demonstrating innovativeness, prominent efficacy or safety data, and potential to address severe diseases, especially when submitted first or simultaneously with other countries. The recognition offers priority consultations and accelerated review timelines, facilitating earlier patient access to transformative treatments.
AB-1005: Targeting Parkinson's Disease
AB-1005 is currently being evaluated in the Phase II REGENERATE-PD trial, an investigational gene therapy using adeno-associated viral vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF (搜索)) gene for participants with moderate-stage Parkinson's disease (搜索). The therapy aims to restore neuronal function and potentially slow disease progression for people with limited treatment options.
The investigational treatment is based on an adeno-associated viral vector containing the human GDNF (搜索) transgene, allowing for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with convection-enhanced delivery. In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons (搜索), which could aid in the preservation and restoration of dopaminergic neuronal circuitries normally lost in the disease.
AB-1005 has previously received multiple regulatory designations, including US FDA Regenerative Medicine Advanced Therapy (RMAT), FDA Fast Track, and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport designations.
REGENERATE-PD Trial Details
REGENERATE-PD is a Phase II, randomized, double-blind, surgery controlled trial evaluating the efficacy and safety of intraputaminal AB-1005 in adults aged 45-75 years with moderate-stage Parkinson's disease (搜索). The trial will include an estimated 87 participants with trial sites located in Germany, Poland, the United Kingdom, and the United States.
In the completed Phase Ib trial, participants were administered AB-1005 to the putamen via one-time bilateral convection-enhanced delivery. Participants were enrolled into two cohorts - mild (6 participants) and moderate (5 participants) - based upon the duration and stage of their Parkinson's disease (搜索). The outcomes assessed at 36 months included incidence of treatment-emergent adverse events, motor symptoms via the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS), and brain dopaminergic network integrity as measured by DaTSCAN.
AB-1002: Addressing Heart Failure
AB-1002 is an investigational one-time gene therapy based on an adeno-associated viral vector administered to the heart to promote production of a modified version of the therapeutic inhibitor 1 (I-1c) protein designed to block the action of protein phosphatase 1 (搜索), which is linked to congestive heart failure (搜索). The therapy previously received FDA Fast Track designation and is designed as a one-time gene therapy targeting protein phosphatase 1 inhibition, with the intention of improving cardiac function.
Phase I Trial Results
The AB-1002 Phase I trial was a non-randomized, sequential dose-escalation study evaluating the safety and preliminary efficacy of the investigational gene therapy in 11 participants with New York Heart Association (NYHA) Class III non-ischemic heart failure (搜索) with reduced ejection fraction. No adverse events were deemed related to AB-1002 in this trial, and clinically meaningful improvements were recorded across several efficacy assessments. The data further support that AB-1002 may be highly cardiotropic when administered as a single intracoronary injection.
Full results were published in October 2025 in Nature Medicine and are available via open access. The therapy is now being evaluated in GenePHIT, a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial conducted at 46 locations across the United States, Austria, Germany, the Netherlands, Spain, and the United Kingdom.
Disease Burden and Unmet Need
Parkinson's disease (搜索) is a progressive neurodegenerative disease affecting more than 10 million people worldwide, making it the world's second most prevalent neurodegenerative disease and the most frequent movement disorder. The prevalence of Parkinson's disease has doubled over the past 25 years. In the disease, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function, with symptoms including tremors, muscle rigidity, and slowness of movement. At present there is no cure, and current treatment options lack the holistic management of symptoms.
Heart failure (搜索) affects more than 64 million people worldwide. The condition occurs when the heart cannot pump blood efficiently enough to meet the body's needs, including providing sufficient oxygen to the organs. Congestive heart failure (搜索) results in the slowing of blood flow out of the heart, causing blood returning to the heart through the veins to back up and create congestion in the body's tissues.
Company Perspective
"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."
Christian Rommel, PhD and Global Head of Research and Development for Bayer's Pharmaceuticals Division, added: "Bayer and AskBio's collaboration continues to drive progress in gene therapy with a robust pipeline targeting central nervous system, cardiovascular, and other disease indications. 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."