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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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Stem cell therapy to slow progression of multiple sclerosis (MS)

Drug Target Review

This study sought to shed light on the safety and effectiveness of aHSCT in routine healthcare settings, moving beyond the confines of clinical trials. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.

Therapies 116
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The rising demand for lentiviral vectors for in vivo gene therapy

Drug Target Review

What trends are driving the increased use of in vivo delivery methods in gene therapy? Being a specialist lentiviral vector CDMO, at VIVEbiotech, this annual growth rate has been higher with a CAGR from 2020 to 2023 of almost a 50 percent. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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Improving Study Activation Time for Gene Therapy Research

Advarra

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027. An IRB tends to get the most attention since all human trials need IRB approval.

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Vertex’s and CRISPR Therapeutics’ Gene Therapy Shows Positive Efficacy | 2020-12-08

The Pharma Data

Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. Source link.

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Regulatory Trends in Cell and Gene Therapies

Advarra

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.