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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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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: How does SNK01 work? RA: What are some of the unmet needs in current treatments for Alzheimer’s?

Therapies 306
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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 Casgevy, a cell-based gene therapy, is approved for the treatment of SCD in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs).

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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
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Positive results announced for COPD therapy

Drug Discovery World

Biopharmaceutical company EpiEndo has completed a Phase IIA clinical trial for its therapy addressing chronic obstructive pulmonary disease (COPD). Damage of this barrier is implicated in several chronic inflammatory diseases, including COPD. ” 1: The Global Economic Burden of Noncommunicable Diseases.

Therapies 130
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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. Specific numerical values (steps) denote the severity of NPDR, with 47-53 describing the moderately severe disease.

Disease 162