Intellia Therapeutics reports positive Phase III data for in vivo gene editing therapy
Intellia therapeutics has announced that its in vivo CRISPR candidate Ionvo-z has met its primary endpoint in the global Phase III HAELO trial for hereditary angioedema (HAE). The therapy demonstrated strong efficacy and a favourable safety profile, reinforcing confidence in systemic gene editing approaches. Intellia has initiated a rolling Biologics Licence Application to the FDA, with a potential launch anticipated in 2027. This milestone represents a significant step forward for in vivo therapies which have long promised greater scalability compared to ex vivo approaches. Positive late-stage data at this level helps de-risk the modality and could accelerate both regulatory pathways and investment into similar platforms. It also raises the bar for competitors, particularly around delivery technologies and long-term durability.
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Regeneron secures first FDA approval for gene therapy in inherited deafness
Regeneron has received FDA approval for the first gene therapy targeting genetic hearing loss, intended for individuals with congenital deafness caused be a deficiency of the otoferlin protein. According to Regeneron, 80% of trial participants achieved at least some significant hearing restoration and 42% ended up with normal hearing. Regeneron has confirmed that the therapy will available for free to patients in the United States and has plans to expand into other forms of hearing loss using different modalities. Investigators noted that Otarmeni is also the first gene therapy to have caused no serious adverse events in a clinical trial, with adverse events limited to those related to the surgical procedure.
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Spain launches $200M biotech fund to strengthen ties with Boston
The Spanish government has launched a venture capital fund with a goal of raising $200 million to invest in biotechnology companies from Spain and Massachusetts, anchored by a $57 million government commitment. The initiative is designed to help Spanish biotech companies scale within one of the world's most competitive life sciences ecosystems and to foster structured collaboration between both regions. The fund could help European companies overcome historical barriers to growth, particularly in accessing late-stage funding. For the advanced therapies space, increased capital flow and cross-border partnerships are likely to accelerate development timelines while intensifying global competition.
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