Phase I
With one disease-modifying therapy already reaching patients and another expected to soon, several biopharma companies anticipate key data for novel assets in the coming 12 months.
Biotech companies are exploring multiple targets in the fight against neuroinflammation. Experts say these projects could yield the next breakthrough in treating Alzheimer’s disease.
After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.
While decentralized trials are expediting innovation and promoting diversity, challenges remain, including complex stakeholder ecosystems, skills gaps and regulation.
In a Phase I/II trial, an 11-year-old boy gradually regained hearing—eventually reaching normal range for some frequencies at 30 days—following treatment with Eli Lilly’s dual adeno-associated viral vector-delivered gene therapy.
From ALS to depression to Huntington’s disease, many neuroscience-focused companies are anticipating key data over the next few months.
In a major pivot, Allogene Therapeutics will no longer focus on two of its studies testing blood cancer therapy cema-cel in an effort to extend its financial runway into 2026.
Over the past year, obesity treatments have become the focus of intense media attention and discussion in healthcare — with increased focus on Wegovy and Zepbound. This attention has highlighted the need to address the global obesity epidemic.
Patients treated with Longboard Pharmaceuticals’ bexicaserin saw a 32.5% drop in the frequency of countable motor seizures relative to placebo in a Phase 1b/IIa study. The company’s shares soared 316% Tuesday on the news.
Gene therapy company uniQure announced Tuesday “ongoing evidence” of a clinical benefit for its Huntington’s disease treatment. However, investors were not impressed as the stock dropped nearly 20%.
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