AB-1005 has also been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (
AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy being studied for the treatment of moderate Parkinson's disease. Earlier this year, AskBio presented the 18-month Phase Ib clinical trial results for AB-1005, which met its primary objective of evaluating the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.
These designations clearly underscore the importance of developing innovative therapies for those living with Parkinson's disease, where a significant unmet need still exists. They further highlight the willingness of key regulatory bodies to support the accelerated development of AB-1005 with a focus on the potential benefit to patients.
Scientific Chair, Parkinson's and MSA, AskBio
The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.1 The purpose of the Program is to get important new therapeutics to patients earlier.1 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.
The
'The FDA Fast Track and the
'We are excited about the opportunity to potentially accelerate the development of AB-1005, leveraging the frequent interaction with relevant regulatory bodies,' said
AB-1005 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.
AskBio is also exploring GDNF therapy beyond Parkinson's disease and is currently enrolling patients in the
About Parkinson's disease
Parkinson's disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels.5 At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons.6 The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement.7 Even with medication, the symptoms of Parkinson's disease can fluctuate during the course of the day. According to the
About REGENERATE-PD
REGENERATE-PD is a Phase II, randomized, double-blind, surgery-controlled trial of the efficacy and safety of intraputaminal AB-1005 in the treatment of adults (45-75 years) with moderate stage Parkinson's disease. Subjects will receive either bilateral image-guided infusion of AB-1005 into the putamen, single dose (active treatment arm), or bilateral partial burr/twist holes (control arm). The trial will include an estimated 87 subjects with trial sites located in the
About AB-1005
AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-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.10,11 GDNF is a homodimer that is a distantly related member of the transforming growth factor- 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 disease modifying neurorestorative treatment for diseases, such as Parkinson's, marked by progressive degeneration of midbrain dopaminergic neurons.12
About AskBio
About Bayer
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. 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 2023, the Group employed around 100,000 people and had sales of
Contact:
Tel: +1 (984) 5207211
Email: pmcnamara@askbio.com
Dr.
Tel: +49 (214) 60001275
Email: imke.meyer@bayer.com
Web: www.bayer.com
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