Remove 2021 Remove Disease Remove Therapies
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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. alone, according to the Centers for Disease Control and Prevention.

Therapies 118
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With new IPO pitch, Artiva touts ‘natural killer’ cell therapy for autoimmune disease

BioPharma Drive: Drug Pricing

Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here.

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Rare autoantibody diseases: an innovative targeted pathway

Drug Target Review

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

Disease 98
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Gamma delta T cells: a rising star in cancer therapy

Drug Target Review

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105
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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111
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Improved prime editing system makes gene-sized edits in human cells at therapeutic levels

Broad Institute

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

DNA 144