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BioWorld - Saturday, April 4, 2026
Home » Topics » Drugs » Gene therapy

Gene therapy
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University of Sydney to invest AU$478M into Sydney Biomedical Accelerator to translate research

Sep. 1, 2022
By Tamra Sami
The University of Sydney is investing AU$478 million (US$326.5 million) to build a leading biomedical precinct to fast-track research and shorten the time between discovery and development of transformative therapies.
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Gene editing illustration

Epigenic raises $20M to develop gene editing therapies

Aug. 23, 2022
By Doris Yu
Epigenic Therapeutics Co. Ltd., a company developing epigenomic-focused gene therapies in multiple indications, has raised $20 million across angel and pre-series A rounds to support preclinical studies, early stage research, and growth for its team.
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Gene editing illustration

Epigenic raises $20M to develop gene editing therapies

Aug. 18, 2022
By Doris Yu
Epigenic Therapeutics Co. Ltd., a company developing epigenomic-focused gene therapies in multiple indications, has raised $20 million across angel and pre-series A rounds to support preclinical studies, early stage research, and growth for its team.
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Red blood cells, DNA

Bluebird prepares to launch $2.8M beta-thalassemia drug

Aug. 18, 2022
By Lee Landenberger
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
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With $2.8M price tag, Bluebird’s Zynteglo first cell-based gene therapy for certain beta-thalassemia patients approved in US

Aug. 17, 2022
By Lee Landenberger
The U.S. FDA has approved the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia requiring frequent red blood cell transfusions. The $2.8 million wholesale acquisition cost for the one-time I.V. infusion will make it one of the most expensive drugs in the U.S.
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DNA illustration

Vector Biopharma closes $30M series A round to support new gene therapy vector

Aug. 10, 2022
By Cormac Sheridan
Vector Biopharma AG has secured a $30 million series A funding commitment from founding investor Versant Ventures to take forward a new gene delivery platform developed by Andreas Plückthun, of the University of Zurich, in Switzerland.
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Eye and DNA illustration

Frontera nets $160M in series B round to develop gene therapy candidate

July 26, 2022
By Doris Yu
Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
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DNA in test tubes
Newco news

Replay spins up genomic medicine companies with $55M seed round

July 25, 2022
By Nuala Moran
Increased payload capacity for gene therapies, off-the-shelf genome-engineered allogeneic cell therapies, reduced cost of goods and faster bioprocesses, are promised by “big DNA” specialist Replay Holdings LLC. The newco arrived with a $55 million seed round and having assembled a portfolio of technologies for writing and delivering large pieces of DNA.
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Eye and DNA illustration

Frontera nets $160M in series B round to develop gene therapy candidate

July 22, 2022
By Doris Yu
Frontera Therapeutics Inc. raised $160 million in a series B funding round to develop its lead gene therapy product candidate for retinal disease, FT-001, for which INDs have been approved by the U.S. FDA and China NMPA.
Read More
Ovarian cancer illustration

VBL’s phase III of ofra-vec misses in ovarian cancer, dragging the stock with it

July 20, 2022
By Lee Landenberger
VBL Therapeutics Inc.’s phase III study of its gene therapy, ofranergene obadenovec, in treating ovarian cancer missed its primary endpoints, prompting the company to discontinue the trial and investors to pull way back. Shares of the Tel Aviv, Israel, and New York-based company’s stock (NASDAQ:VBLT) plunged 79% on July 20 to close at 43 cents per share. The $1.62 per share drop in value made it a penny stock. VBL shares hit their high on Nov. 8, closing at $2.53 each. Top-line data for ofra-vec, also known as VB-111, showed no statistically significant improvement in progression-free survival or overall survival.
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