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SpliceBio lands $135M for a new kind of eye gene therapy

BioPharma Drive: Drug Pricing

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to help overcome the packaging constraints of current genetic medicines.

Packaging 245
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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies 137
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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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Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy

Broad Institute

Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy By Allessandra DiCorato August 7, 2024 Breadcrumb Home Machine learning approach helps researchers design better gene-delivery vehicles for gene therapy The method could accelerate the development of more effective adeno-associated viruses (AAVs).

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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo. By Sarah C.P.

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Delivering gene-based therapies

Broad Institute

To be effective, these therapies must deliver the new genes, or the gene-editing machinery, to enough of the right cells in the body to have a therapeutic effect. But researchers have struggled to design molecular vehicles that can deliver gene-based therapies safely and effectively to the right cells.