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Cell therapy limitations with iPS-based multi-cell therapeutics

Drug Target Review

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103
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Researchers reprogram gene therapy viral vectors to bind specific protein targets

Broad Institute

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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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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Targeting a human protein may stop Ebola virus in its tracks

The Pharma Data

To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase. Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Drugs that target polymerase could potentially treat Ebola virus infections and save lives.

Virus 52
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Recent Advances in Gene Therapy

DrugBank

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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mRNA Therapeutics: Revolutionizing Treatment Beyond Vaccines

DrugBank

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system.

Vaccine 97