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BioWorld - Tuesday, December 30, 2025
Home » Topics » Drugs » Gene therapy

Gene therapy
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DNA in drug capsules

BioFuture 2024: FDA eases the way for cell and gene therapy companies

Nov. 6, 2024
By Lee Landenberger
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.
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Ring Therapeutics, ASTAR and SERI MOU

Ring Therapeutics expands Singapore footprint with new deals

Nov. 5, 2024
By Tamra Sami

Ring Therapeutics Inc. has joined forces with Singapore’s Agency for Science, Technology, and Research and with the Singapore Eye Research Institute to advance the first new class of viral vectors in more than 50 years, Ring CEO Tuyen Ong told BioWorld.


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PMDA U.S. office in Washington D.C.

Japan PMDA opens second overseas office in Washington

Nov. 5, 2024
By Marian (YoonJee) Chu
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) set up its second overseas regulatory office in Washington, four months after the drug and med-tech regulator opened its first Asia base in Bangkok, Thailand, in July 2024.
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Pancreas illustration
Endocrine/metabolic

Fractyl Health nominates GIP/GLP-1 dual agonist as pancreatic gene therapy candidate for obesity

Nov. 5, 2024
Fractyl Health Inc. has nominated RJVA-002, a GIP/GLP-1 dual agonist, as its first pancreatic gene therapy candidate for obesity.
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AI-generated digital horse illustration
Cancer

Gene editing is Trojan horse of cancer immunotherapy

Nov. 4, 2024
By Mar de Miguel
Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.
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Cardiovascular

AAV-driven gene therapy shows promise for PLN-R14del cardiomyopathy

Nov. 4, 2024
The PLN-R14del mutation is often tied to dilated or arrhythmogenic cardiomyopathy, which can progress to end-stage heart failure. Cardiac phospholamban (PLN) plays a crucial role in calcium modulation in cardiomyocytes, which is disrupted by the PLN-R14del mutation.
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DNA in drug capsules

BioFuture 2024: FDA eases the way for cell and gene therapy companies

Oct. 31, 2024
By Lee Landenberger
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York.
Read More
Amyloid plaques on nerve cell

How now, tau? ‘Newer science’ shines in Lexeo Alzheimer’s data

Oct. 30, 2024
By Randy Osborne
The prospect of a gene therapy for Alzheimer’s disease has kept Wall Street steadily interested in Lexeo Therapeutics Inc., and another increment of intrigue was added when the New York-based firm  offered positive interim results from the 52-week, 15-subject phase I/II study of LX-1001 for the treatment of the condition when APOE4-associated.
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DNA in drug capsules

BioFuture 2024: FDA eases the way for cell and gene therapy companies

Oct. 30, 2024
By Lee Landenberger
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of  Ray Therapeutics Inc.
Read More
Brain cancer illustration
Cancer

ESGCT 2024: Steps forward in gene and cell therapies for brain tumors

Oct. 29, 2024
By Mar de Miguel
Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome.
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