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AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced the completion of enrollment for REGENERATE MSA-101, its phase 1 clinical trial of AB-1005, an investigational gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).
The completion of enrollment marks a significant milestone in the development of AB-1005, an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, and it brings this potential treatment option one step closer to reaching the many patients affected by MSA-P.
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. This is a result of progressive loss of nerve cells in the brain and spinal cord. Affecting approximately 400,000 people worldwide, MSA is a rare disease that in most cases seems to occur for unknown reasons. Symptoms tend to develop in people over 50 years of age, followed by rapid progression within 5–10 years.
“There is currently no disease-modifying treatment to stop or slow the progression of MSA, which makes the completion of enrollment in REGENERATE MSA-101 an important step in AskBio’s efforts to advance this clinical program and potentially bring a safe treatment to patients” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA at AskBio. “This latest milestone follows recent additional AB-1005 announcements highlighting meaningful progress in our Parkinson’s disease program, including phase 1b favourable safety outcomes.”
AskBio is also exploring AB-1005 beyond MSA-P in participants living with Parkinson’s disease in REGENERATE-PD, the company’s randomized, double-blind, surgically controlled phase 2 clinical trial evaluating the safety and efficacy of AB-1005 delivered to the putamen in the brain of adult participants aged 45–75 years with moderate-stage Parkinson’s.
In January, AskBio announced that the first participants had been randomized in REGENERATE-PD. In February, AskBio received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AB-1005 for Parkinson’s disease, following the presentation of phase 1b 36-month data that demonstrated a favorable safety profile and continued positive trends in assessed clinical outcome measures with no product-related serious adverse events.
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.
REGENERATE MSA-101 is a randomized, double-blind, placebo-controlled phase 1 trial designed to determine the safety of AB-1005, an investigational gene therapy delivered to the putamen in patients with multiple system atrophy-parkinsonian type (MSA-P). The trial will include adults aged 35 to 75 years, with a clinical diagnosis of MSA-P (as defined by current consensus criteria), who are suitable surgical candidates. The trial enrolled 11 participants who were randomized on the day of surgery to receive either AB-1005 or a non-invasive control surgery without any infusion to the putamen. The trial has a three-year follow-up period. Initial results will be available after all participants have completed one year of blind clinical assessments and the trial has been unblinded. The first participant in the REGENERATE MSA-101 trial was randomized at the Ohio State University Wexner Medical Center, Ohio, with participants enrolled by investigators based at University of California Irvine, California; Quest Research Institute, Michigan; Vanderbilt University, Tennessee; and New York University Langone, New York. Brain Neurotherapy Bio, Inc., an AskBio subsidiary, is the sponsor for REGENERATE MSA-101.
Glial cell line-derived neurotrophic factor (GDNF) is a homodimer that is a distantly related member of the transforming growth factor-ß superfamily. In midbrain cell culture experimental models, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake. GDNF has long been suspected to be a potential treatment for neurodegenerative diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons. AB-1005 is an adeno-associated viral vector serotype 2 (AAV2) containing the human 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.
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.
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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