AskBio

World Parkinson's Day
#AskBio #Blood brain barrier #FDA #Gene therapy #Parkinson's #Regulatory

How AskBio is pioneering ‘transformative genetic therapies’ in Parkinson’s disease

Bruno Quinney speaks to Adrian Kells, Senior Vice President and Integrated Product Team Lead PD at AskBio, about the company’s gene therapy programme and pioneering treatment for Parkinson’s. The drug discovery landscape for Parkinson’s disease is becoming one of the most exciting frontiers in the industry. It is unsurprising to… Read More

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Human heart
#AskBio #Gene therapy #Heart failure

Gene therapy could treat congestive heart failure

Gene therapy company AskBio has announced encouraging results from the Phase I trial of gene therapy AB-1002 in participants with congestive heart failure.  The 12-month data set, revealed in Nature Medicine, confirms that meaningful improvements were recorded across several efficacy assessments and no adverse events… Read More

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Genetic engineering concept
#AskBio #FDA #Gene therapy #Neuroscience #Parkinson's

Further FDA support for Parkinson’s gene therapy

AskBio’s investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA).  The FDA decision follows a review of information and data provided by AskBio, including clinical evidence from the… Read More

#News #Regulatory

Cell and gene therapies
#AskBio #Gene therapy #Neurodegenerative disorders #Parkinson's #Phase II trial

Gene therapy for Parkinson’s disease progresses to Phase II

A new Phase II trial, REGENERATE-PD, will study AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. The trial will include an estimated 87 subjects with study sites located in the US, UK and… Read More

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Parkinson's disease
#AskBio #Cell and gene therapy #Gene therapy #Neurodegenerative disorders #Parkinson's #Phase II trial

Gene therapy proved to be safe in patients with Parkinson’s disease

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado,… Read More

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#AAV #AskBio #Duchenne muscular dystrophy #Gene editing #Giant axonal neuropathy #Haemophilia #Pompe disease

Podcast: The evolution and future of gene therapy

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business.    In this episode, Megan Thomas is in conversation with Dr… Read More

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