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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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Cell and gene therapy development moves into cardiac indications

Drug Target Review

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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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. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications.

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Transforming cancer treatment for greatest impact

Drug Target Review

The preclinical results demonstrated significant immune responses and tumour regression. Could you elaborate on the mechanisms by which these responses are initiated within cancer cells? This induced anti-tumour immunity is also detectable by increased innate and adaptive immune cells in the tumour, spleen and blood.

Treatment 106
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Merck Strikes $1B+ Deal to Leverage Janux’s T Cell Engager Program Against Cancer

The Pharma Data

“At Janux, we have developed a technology to engineer best-in-class T cell engagers that are potent and highly tumor-specific, which is essential for an immune response that kills tumor cells but spares healthy tissue,” David Campbell, president and chief executive officer of Janux Therapeutics said in a statement.

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Cancer immunotherapy candidate provokes powerful dual response in cancer and immune cells

Broad Institute

The molecule’s dual mechanism of action — targeting both tumor and immune cells — is unique compared to other cancer immunotherapies including PD-1 drugs, and the researchers think it could explain why the molecule was so effective on its own in animal models and may not even need to be used in combination with other drugs such as anti-PD-1 therapy.

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Clover Biopharmaceuticals Announces Positive Phase 1 Data for its Adjuvanted S-Trimer COVID-19 Vaccine Candidates

The Pharma Data

Strong Th1 cell-mediated immune responses were also observed for the vaccine candidates with either adjuvant. We are encouraged by the high level of neutralizing antibodies in combination with the strong Th1 response which we believe could play an important role in controlling infection.

Vaccine 52