Lilly allies with gene-editing firm Seamless on hearing loss

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Black and white image of the side of a baby's face, focused on its ear
Laura Ohlman

Gene-editing specialist Seamless Therapeutics has attracted its first major pharma partner, Eli Lilly, with a $1.12 billion alliance that will seek out therapies for hearing loss.

Germany-headquartered Seamless, which was formed in Dresden three years ago, will work with Lilly on programmable recombinase-based treatments for a range of conditions associated with deafness, although the specific indications are yet to be divulged.

Seamless's gene-editing platform uses recombinase enzymes that can bind to any specific sequence in a genome, using a dual targeting mechanism that boosts specificity and, according to the company, all but eliminates off-target activity.

Unlike other gene-editing systems such as CRISPR, recombinases cut and rejoin DNA directly, rather than relying on cellular repair processes that may be more prone to resulting in mutations.

On behalf of Lilly, Seamless will design and programme recombinases that can correct alterations in multiple genes involved in hearing loss. Lilly will then receive a license to the recombinases and take responsibility for advancing them through development and onto the market.

The deal comes as various gene-directed therapies for hearing loss are making their way through the industry pipeline, including some focusing on mutations in the otoferlin (OTOF) gene. There are hundreds of other mutations in genes linked to deafness, including GJB2 – the most commonly encountered – as well as MYO15A, TMC1, and SLC26A4.

Lilly is among the companies already working on genetic therapies for deafness, as its acquisition of Akouos in 2022 for $487 million upfront gave it an OTOF-targeted therapy that is in phase 2 testing, having generated positive preliminary results in a phase 1/2 trial in 2024.

Others include Regeneron – which reported encouraging results from a phase 1/2 trial of its OTOF-directed candidate DB-OTO last year – and Sensorion, whose SENS-501 therapy is also in phase 1/2.

"Lilly is invested in advancing novel treatment approaches for genetic diseases and shares our vision of bringing genetic medicines to patients who currently have limited treatment options," commented Seamless's chief executive Albert Seymour.

"This collaboration is a validation of our gene editing platform and its broad disease-modifying potential," he added. "It's an exciting opportunity to apply our technology to bring treatments to patients with hearing loss and continue to expand the therapeutic potential for programmable recombinases through our proprietary pipeline."

Photo by Laura Ohlman on Unsplash