AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s disease meets primary endpoint

Berlin, Germany/Research Triangle Park, North Carolina, USA, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Not intended for UK Media

  • AB-1005 (AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no attributed serious adverse events in all 11 patients at 18 months.
  • 18-month assessment of all patients demonstrated feasibility of administration of AB-1005 to the putamen via one-time bilateral convection-enhanced delivery.
  • AskBio plans to present 18-month study data, including secondary endpoints, at a scientific meeting in Q2 of 2024.
  • Planning is underway for a Phase II trial to be initiated in the first half of 2024.

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2

The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based upon the timing from a PD diagnosis and the severity of their PD symptoms at screening.1 Neurosurgical delivery of AB-1005 was well tolerated by all patients with target putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. No serious adverse events have been attributed to AB-1005, with continued clinical follow-up for up to 5 years post administration ongoing.2

“We are encouraged by these early data, which show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Although there is still much to learn about this early-stage investigational gene therapy, these first findings will inform our work in this space and have the potential to contribute to the clinical advancement of AB-1005 for the treatment of Parkinson’s disease.”

Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD. Additionally, brain imaging was performed to longitudinally assess safety and potential changes in dopamine handling or abnormal metabolic patterns associated with PD.1

AskBio is planning to present the 18-month study data, including secondary endpoints, at a scientific meeting in Q2 2024. Planning is underway for a Phase II trial that is expected to begin screening patients in the first half of 2024. The trial design has been harmonized with feedback from U.S. and European health authorities.

“People living with Parkinson’s disease deserve options to address their unmet medical need,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The positive outcome of the AB-1005 Phase Ib clinical trial is an important step forward in our goal to deliver much-needed treatments in areas where innovation has the potential to make a tremendous impact.”


About AB-1005

AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery.3,4 GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake. GDNF has long been evaluated as a potential treatment for diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons.5


About the AB-1005 Phase Ib trial

In this Phase Ib, multi-center, multi-site, parallel assignment, non-randomized trial, 11 patients were administered AB-1005 to the putamen via one-time bilateral convection-enhanced delivery. Patients were enrolled into two cohorts, Mild (6 patients) and Moderate (5 patients), based upon the duration and stage of their PD. The objective of this investigation was to evaluate the safety and potential clinical effect of AB-1005 delivered to the putamen in patients with either a recent or a long-standing diagnosis of PD. The outcomes assessed at 18 months were incidence of Treatment-Emergent Adverse Events (TEAEs) as reported by the patients or assessed clinically by physical and neurological examinations, motor symptoms including the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and PD Motor Diary self-assessments, non-motor symptoms of PD and brain dopaminergic network integrity as measured by DaTSCAN.1 These assessments will continue for up to five years. Brain Neurotherapy Bio, Inc., an AskBio subsidiary, is the sponsor of this Phase Ib trial. For more information, visit (NCT#04167540), visit, or send an email to


About Parkinson’s disease

Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons and at diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons.6 Parkinson’s disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping, and slowness of movement (bradykinesia).7 According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease.8


About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at or follow us on LinkedIn.


About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its 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. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to

Find more information about AskBio at
Find more information about Bayer at
Follow Bayer on Facebook:
Follow Bayer on Twitter: @BayerPharma

naf                  (2024-0002e)


Bayer Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


AskBio Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.




[1] GDNF Gene Therapy for Parkinson’s Disease. Available at: Accessed December 2023.
[2] Asklepios BioPharmaceutical. Data on file.
[3] Heiss JD, Lungu C, Hammoud DA, Herscovitch P, Ehrlich DJ, Argersinger DP, Sinharay S, Scott G, Wu T, Federoff HJ, Zaghloul KA, Hallett M, Lonser RR, Bankiewicz KS. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson’s disease. Mov Disord. 2019 Jul;34(7):1073-1078.
[4] Kells AP, Eberling J, Su X, Pivirotto P, Bringas J, Hadaczek P, Narrow WC, Bowers WJ, Federoff HJ, Forsayeth J, Bankiewicz KS. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul 14;30(28):9567-77.[5] Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 1993;260(5111):1130-1132.
[6] NIH – Parkinson’s disease. Available at: Accessed December 2023.
[7] Medline Plus. Parkinson disease. Available at: Accessed December 2023.
[8] AlMahadin, G., Lotfi, A., Zysk, E. et al. Parkinson’s disease: current assessment methods and wearable devices for evaluation of movement disorder motor symptoms - a patient and healthcare professional perspective. BMC Neurol 2020 20, 419.


Contact Data