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BioWorld - Sunday, November 9, 2025
Home » Topics » Drugs » Gene therapy

Gene therapy
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Astellas stops dosing gene therapy for rare neuromuscular disease after trial's fourth SAE

Sep. 1, 2021
By Michael Fitzhugh
Astellas Pharma Inc. has halted further dosing of the experimental X-linked myotubular myopathy (XLMTM) gene therapy AT-132 (resamirigene bilparvovec) after one participant in the ongoing Aspiro study experienced a serious adverse event (SAE) of abnormal liver function.
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Person holding knee, joint pain

Non-viral gene therapy developer Xalud brings in a $30M series C

Aug. 27, 2021
By Lee Landenberger
Xalud Therapeutics Inc. raised an oversubscribed $30 million series C financing to continue advancing its lead candidate, XT-150, for regulating interleukin-10 in order to treat pathologic inflammation. The injectable, plasmid DNA gene therapy expresses IL-10v, a modified version of the cytokine IL-10, and is in a phase IIb study for treating moderate to severe pain caused by osteoarthritis (OA) of the knee.
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Blood sample, DNA

Belief Biomed’s BBM-H901 wins first Chinese IND approval in gene therapy for hemophilia B

Aug. 17, 2021
By Doris Yu
China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
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Blood sample, DNA

Belief Biomed’s BBM-H901 wins first Chinese IND approval in gene therapy for hemophilia B

Aug. 12, 2021
By Doris Yu
China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
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BIO Asia-Taiwan: Cell and gene therapy development changing biopharma ecosystem

July 27, 2021
By Doris Yu
Cell and gene therapy have seen much progress in recent times, with the product pipelines in those areas bursting with more than 1,200 therapies. Naturally, the challenges, opportunities and essential development strategies in those fields were the focus on the last day of the BIO Asia-Taiwan Conference 2021.
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Woman taking Amsler grid eye exam

Through Infinity and beyond, Adverum gene therapy keeps buzz alive in AMD

July 23, 2021
By Randy Osborne
Adverum Biotechnologies Inc. CEO Laurent Fischer said the firm chose the more prudent route in scrapping development of gene therapy ADVM-022 (AAV.7m8-aflibercept) for diabetic macular edema (DME) as a result of dose-limiting toxicity in the phase II Infinity trial.
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Pill with British pound sign

NHS England tackles pricey gene therapies with £340M fund

July 23, 2021
By Richard Staines
NHS England has announced a new £340 million (US$481 million) Innovative Medicines Fund, as the country grapples with the issue of bringing ultra-pricey specialist medicines to patients on the country’s taxpayer-funded health care system.
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BIO Asia-Taiwan: Cell and gene therapy development changing biopharma ecosystem

July 23, 2021
By Doris Yu
Cell and gene therapy have seen much progress in recent times, with the product pipelines in those areas bursting with more than 1,200 therapies. Naturally, the challenges, opportunities and essential development strategies in those fields were the focus on the last day of the BIO Asia-Taiwan Conference 2021.
Read More
DNA illustration

No drama as Horama raises $25M, becomes Coave Therapeutics to reflect wider focus

July 21, 2021
By Cormac Sheridan
DUBLIN – Coave Therapeutics unveiled a new identity and a new gene therapy platform, as it closed a €21.2 million (US$25 million) extension to its long-running series B round, which takes the total raise to €33 million.
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RNA strand

Shape of RNA to come: Series Bs boost gene therapy companies

July 15, 2021
By Lee Landenberger
Private biopharma investment in 2021 continues to outpace that of the past two years as two gene therapy companies, Shape Therapeutics Inc. and Kriya Therapeutics Inc., posted series B financings this week totaling more than $200 million.
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