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Gene therapy for Parkinson’s disease progresses to Phase II

Drug Discovery World

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 Europe.

Therapies 147
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Diabetic eye disease: The urgent need for early, non-invasive treatments

Drug Discovery World

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Diabetic retinopathy – current treatments The journey for patients with DR is one of increasing severity and loss of vision.

Disease 162
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Gene therapy proved to be safe in patients with Parkinson’s disease

Drug Discovery World

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. mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

Therapies 130
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The killer instinct: Using investigational natural killer therapy to treat Alzheimer’s

Drug Discovery World

DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. RA: What are the clinical benefits of using the patient’s own cells as a treatment?

Therapies 306
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Webinar: Transforming disease treatment with CGT

Drug Discovery World

Register to attend Cell and gene therapy: sustaining growth and maximising opportunities, a free-to-attend DDW webinar, supported by Benchling and BPS Bioscience, on February 17, 2023 at 8am PST / 11am EST / 4pm GMT / 5pm CET. Cell and gene therapies (CGT) are transforming the way diseases are treated.

Disease 130
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FDA approves first gene therapies for sickle cell disease (with a warning)

Drug Discovery World

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Reflections from Advanced Therapies Week 2023 

Drug Discovery World

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

Therapies 246