Avidity drug advances in muscular dystrophy; Syntis innovates weight loss method

Avidity Biosciences saw a significant increase in its shares, rising by nearly one-third after the company disclosed promising new clinical trial results for its drug del-brax. This drug is designed to treat facioscapulohumeral muscular dystrophy, a condition characterized by muscle function loss due to abnormal expression of the DUX4 gene. In a study involving eight patients, the company reported an average reduction of over 50% in a panel of genes regulated by DUX4. Additionally, participants exhibited signs of increased muscle strength, a positive indication for the drug's efficacy.

Meanwhile, Nodthera, based in Boston, announced favorable results for its leading drug candidate, which aims to inhibit a protein complex that triggers inflammation. In a small study involving obese patients with cardiovascular risks, those treated with Nodthera's drug showed reduced biological markers associated with inflammation after one month of treatment. The study also included a controlled daily calorie intake. While all participants lost weight, the most significant reductions were observed in high-risk subgroups who received the drug. Nodthera believes these findings underscore the potential anti-obesity benefits of its therapy.

Startup Syntis Bio launched on Tuesday, with a focus on advancing treatments for weight loss, other metabolic conditions, and rare pediatric diseases. Co-founded by MIT professor Robert Langer, the company is currently testing an oral medication named SYNT-101 in human trials. SYNT-101 is claimed to mimic the effects of gastric bypass surgery. Syntis Bio secured $15.5 million in a seed funding round in 2023 to support its research and development efforts.

QurAlis has made significant changes to its executive team. On Tuesday, the company announced the appointment of Douglas Williamson as the new Chief Medical Officer, succeeding Angela Genge. Williamson previously held positions at Avadel Pharmaceuticals and Acadia Pharmaceuticals. Additionally, Jason Brown will join QurAlis as the Chief Financial Officer. Brown was formerly the CFO at Karuna Therapeutics, which was acquired by Bristol Myers Squibb for $14 billion last year.

In other news, Japan's Kyowa Kirin announced plans to invest up to $530 million in a new production plant for biologic drugs in Sanford, North Carolina. The facility, covering approximately 170,000 square feet and housing two bioreactors, is expected to be completed by 2027. According to Kyowa Kirin's statement on Monday, the plant will focus on manufacturing next-generation antibodies.

These updates reflect significant strides and strategic moves within the biotech industry, showcasing the continuous efforts of companies to develop innovative therapies and expand their operational capacities.

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