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AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that it has introduced its proprietary, commercially ready manufacturing process to supply ametefgene parvec (AB-1005), an investigational gene therapy for the treatment of Parkinson’s disease (PD) and multiple system atrophy-parkinsonian type (MSA-P), following submission of a United States Food and Drug Administration (FDA) Investigational New Drug Application (IND) amendment.
This enables AskBio to now advance its REGENERATE-PD phase II clinical trial in the United States with the material produced at the commercial manufacturing facility of AskBio’s wholly owned subsidiary, Viralgen, with other trial sites to follow, using an intensified, high-efficiency, next-generation large-scale suspension manufacturing process that provides a consistently high purity product.
“Today’s news marks another important advancement in our Parkinson’s programme,” said Canwen Jiang, MD, PhD, chief development officer and chief medical officer at AskBio. “We are initiating supply with our manufacturing platform, to deliver our investigational gene therapy to participants in our ametefgene parvec trials, including our REGENERATE-PD trial, which recently saw the randomization of our first participants in Germany. The trial is currently enrolling participants in Poland, the United Kingdom, and the United States.”
“Manufacturing efficiency of our AAV gene therapy-based drug product is mission critical for our phase II Parkinson’s disease trial,” said Christian Rommel, PhD, global head of research and development for Bayer’s pharmaceuticals division. “It underscores the innovation of our production technology and reflects our continued commitment to meet high regulatory standards.”
In December 2025, AskBio was granted Pioneering Regenerative Medical Product designation (SAKIGAKE) in Japan for ametefgene parvec, and in February 2025 the company was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Prior to that, AskBio received FDA Fast Track designation and the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation. All designations were for PD.
AskBio is also exploring ametefgene parvec in participants in the United States with the parkinsonian subtype of MSA-P in a fully enrolled phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing neurodegenerative condition.
Ametefgene parvec is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.
Parkinson’s disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person’s daily life. In PD, the progressive death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks increasingly demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms, so there is an urgent need for new therapies.
The parkinsonian subtype of multiple system atrophy (MSA-P) can initially be difficult to distinguish from Parkinson’s disease and is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting, among other symptoms. Individuals experience increasing difficulty with movement and autonomic dysfunction as a result of progressive loss of nerve cells in the brain and spinal cord. Affecting approximately 400,000 people worldwide, MSA-P is a rare disease, and its exact cause is unknown. Symptoms usually start to develop around the age of 50, followed by rapid progression within 5–10 years. At present there is no cure and no approved treatments for MSA-P, so there is an urgent need for new therapies.
REGENERATE-PD is a phase II, randomized, double-blind, surgery controlled trial of the efficacy and safety of intraputaminal ametefgene parvec (AB-1005) in the treatment of adults (45–75 years) with moderate-stage Parkinson’s disease. The trial will include an estimated number of 127 participants with trial sites located in Germany, Poland, the United Kingdom, and the United States.
Ametefgene parvec (AB-1005) is an investigational gene therapy intended to slow disease progression and improve motor outcomes in people with Parkinson’s disease (PD). Composed of an adeno-associated viral vector containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, ametefgene parvec allows 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, which could aid in the preservation and restoration of dopaminergic neuronal circuitries normally lost in the disease. GDNF has long been evaluated as a potential disease-modifying treatment for diseases, such as PD which are marked by progressive degeneration of midbrain dopaminergic neurons. Through a combination of an investigational gene therapy and innovative neurosurgical delivery approach, building on the promising outcomes of the open-label phase 1b trial, we can now test the GDNF hypothesis by getting this neurotrophic factor to these degenerating nigrostriatal neurons in a well-controlled clinical study to understand the effects of GDNF in a moderate PD population.
AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases.
Viralgen is a leading contract development and manufacturing organization (CDMO) specializing exclusively in adeno-associated virus (AAV)–based gene therapies. Founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, Viralgen provides end-to-end support from early development through large-scale commercial production.
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.
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