Lilly's gene therapy restores hearing in a child

A gene therapy made by Eli Lilly has allowed an 11-year-old boy with a genetic form of deafness to hear sound for the first time. Aissam Dam's inability to hear is caused by a mutation in a single gene, called otoferlin. The gene therapy is delivered directly to the ear, which is a closed system — so it won't affect other cells in the body. There are about 200,000 people with this form of deafness around the world, the New York Times writes.

The FDA mandated that researchers testing out the gene therapy begin with older children, not infants, and treat only one ear. The researchers, who were at Children's Hospital of Philadelphia, were "flying blind," they said — unsure how effective the treatment would be. But now that it's been shown to be safe, the treatment is being tested on more children — including 2- and 3-year-olds in the U.S.

The technology was developed by Akuous, a small biotech that Lilly acquired in 2022. And this isn't the only company developing gene therapies for hearing loss: China-based Otovia Therapeutics and Refreshgene Therapeutics, as well as Regeneron and Decibel Therapeutics, are testing out their respective treatments in this space.

obesity

Novo Nordisk invests in a new obesity approach

Novo Nordisk is investing in another obesity treatment: It will pay Swiss biotech EraCel Therapeutics some $255 million for a medicine that has a novel approach to controlling appetite and body weight, FierceBiotech writes. Novo Nordisk began collaborating with EraCel two years ago, with the companies studying potential obesity candidates in zebrafish larvae.

The result is a small molecule drug that has demonstrated potency in weight loss and could potentially work in tandem with drugs like Wegovy and Ozempic, but little is known of it outside of that. It targets an "undisclosed novel protein that derives peripheral liver-brain signaling and acts in concert with GLP-1-targeting agents in vivo," the company said.

trends

Biopharma accounts for majority of stock sales this year

More than half of all the stock sales this year thus far have been in biopharma, a new Stifel report shows. At 55%, this outpaces prior years pretty dramatically, particularly if the trend continues as the year progresses. In 2023, for instance, 35% of U.S. equity IPOs and follow-ons were in biopharma, whereas in 2014 only 15% were. "This is unprecedented," Stifel writes.

Another interesting trend: The number of venture deals made by investors outside of the U.S. has dropped pretty dramatically: It's down 52% since 2016, Stifel writes. Europe and China had fewer large venture financings in 2023, whereas the U.S. venture ecosystem seems to be strengthening in comparison.

cancer

Large pharma backs Accent's RNA approach

Accent Therapeutics, a startup developing pills that modify RNA to treat cancer, just raised a $75 million Series C round — with backing from both Bristol Myers Squibb and the venture arm of Johnson & Johnson. The financing will help Accent push its candidates into clinical trials, which is projected for early 2025. These aren't the first large pharmaceutical companies to eye Accent; in 2020, AstraZeneca forged a cancer-focused deal with the company.

One of the company's lead programs targets a stretch of RNA called DHX9, which can be found in many tumor types, BioPharmaDive writes. Another is a KIF18A inhibitor, which could potentially treat several cancers, including ovarian and triple-negative breast tumors.

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