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

Gene therapy
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Ferring ramps up bladder cancer drug launch with Royalty deal worth up to $500M

Aug. 24, 2023
By Caroline Richards
Ferring Pharmaceuticals A/S has struck a deal with Royalty Pharma for $500 million, capital it plans to plough into achieving a successful launch of its approved bladder cancer gene therapy, Adstiladrin (nadofaragene firadenovec), in the U.S., as well as enabling it to upgrade and expand its manufacturing sites.
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Astellas returns two gene therapies to Modalis

Aug. 15, 2023
By Marian (YoonJee) Chu
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
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Liver and DNA
Endocrine/Metabolic

FDA grants orphan drug designation to liver-targeted gene therapy, BGT-OTCD

Aug. 14, 2023
Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).
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Astellas returns two gene therapies to Modalis

Aug. 10, 2023
By Marian (YoonJee) Chu
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
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Drug R&D concept image.
Immune

City of Hope receives CIRM funding to support cell and gene therapy clinical trials

Aug. 10, 2023
Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia (AML) and severe aplastic anemia.
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Regeneron brings hearing loss gene therapy programs in-house in $213M Decibel buy

Aug. 9, 2023
By Jennifer Boggs
Six years of collaboration is ending in a buyout, as Decibel Therapeutics Inc. agreed to be acquired by Regeneron Pharmaceuticals Inc. in a deal valued at up to $213 million, including $109 million in equity, with additional payments via contingent value rights linked to clinical and regulatory milestones for DB-OTO, the lead gene therapy program targeting hearing loss.
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Cancer cell, dropper, test tubes
Immuno-oncology

INT-2104, newly engineered lentiviral vector platform to treat B-cell malignancies

Aug. 3, 2023
Researchers from Interius Biotherapeutics Inc. presented the development and preclinical evaluation of a novel gene therapy candidate, INT-2104, as potential candidate for the treatment of B-cell malignancies.
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Art concept for gene therapy research
Drug Design, Drug Delivery & Technologies

Alexion, Astrazeneca Rare Disease agrees to acquire Pfizer’s preclinical rare disease gene therapy portfolio

July 31, 2023
Alexion, Astrazeneca Rare Disease, part of Astrazeneca plc, has entered a definitive purchase and license agreement for a portfolio of preclinical gene therapy programs and enabling technologies from Pfizer Inc.
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Bladder
Newco news

EG 427 advances toward clinic with HSV-based gene therapy for overactive bladder

July 13, 2023
By Nuala Moran
EG 427 SAS has added a further a further €5 million (US$5.6 million) to its series A, closing the round at €18 million and setting the stage for the first clinical trial of a gene therapy in the treatment of a chronic disorder.
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Bladder
Urology

EG 427 announces final series A closing to fund gene therapy to treat neurogenic bladder overactivity

July 13, 2023
EG 427 has announced the final closing of a series A financing, bringing the total raised in the...
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