A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
Abbvie Inc. and Regenxbio Inc. have announced a partnership to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy and other chronic retinal diseases. Under the terms of the agreement, Abbvie will pay Regenxbio $370 million up front, plus up to $1.38 billion in additional development, regulatory and commercial milestones. The deal gives '314 – already the most advanced gene therapy in wet AMD – another potential edge against its nearest competitor, Adverum Biotechnologies Inc.’s ADVM-022.
The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
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.
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.
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.
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.
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.
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.
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.
RSS
