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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Unveiling neoantigen-directed cancer treatment

Drug Target Review

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

Treatment 105
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Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

The Pharma Data

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

FDA 52
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Europe: Where is the drug discovery innovation?

Drug Discovery World

Amsterdam: The centre of European life sciences As a result of the UK withdrawing from the EU, in 2019 the EMA headquarters was moved from London to Amsterdam. The company is now investigating a promising gene therapy for Huntingdon’s disease in a Phase I/II trial, with patients already showing signs of preserved neurological function.

Drugs 147
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AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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Revolutionary nanoparticles enable gene-editing in lungs

Drug Target Review

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. 1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis. The study appears in Nature Biotechnology.

RNA 98
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Tiziana Life Sciences announces collaboration with Parexel Biotech to conduct phase 1b/2 clinical trial in patients with Crohn’s Disease

The Pharma Data

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Oral and nasal administration routes are both physiologic approaches to stimulate the mucosal immune system to induce disease modifying benefits.”.