An AI agent performed brain surgery on an epileptic mouse, picking the drug, the brain target, and the injection coordinates on its own. According to lead researcher Ocana-Santero, it's the first time...

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved a gene-editing treatment for severe sickle-cell disease and transfusion-dependent β-thalassemia for patients aged five…

Get instant access to a concise analysis of 22 BioPharma (+ Google) CVCs and their portfolios. In minutes, you’ll see where strategic capital is flowing across Technology and Therapy Areas—and how 2025 trends compare to 2023. What’s inside: Clear mapping of CVC focus by Technology and Therapy Areas Trend shifts vs. 2023 to validate your thesis Actionable insights to spot momentum early How it works: Fill out the form to receive your report immediately. Questions or feedback? info@lqventures.com LucidQuest empowers BioPharma executives and Life Sciences investors with up-to-date analysis. Stay tuned for 2025 deep dives on Cell & Gene Therapy, Digital Health, Oncology & Hematology, and more.

Discover in-person and virtual events to connect with the bleeding disorders community. Stay informed about upcoming gatherings, workshops, and conferences.

Source: BioPharma Dive

Source: STAT News

Discover in-person and virtual events to connect with the bleeding disorders community. Stay informed about upcoming gatherings, workshops, and conferences.

Strategic insights for gene and cell therapy from R&D through commercialization. Primary KOL research, competitive analysis, and investment diligence in rare diseases.

The U.S. Food and Drug Administration has published draft guidance aimed at streamlining approvals for individualized gene therapies, a move welcomed by rare disease advocates who have long criticized the agency's previous approach. Under the current system, companies must submit separate drug approval applications for each patient's customized gene therapy, even when using fundamentally the same method to target different genes. This process has been deemed prohibitively expensive by advocates, who argue it creates unnecessary regulatory burdens. The new draft guidance formalizes a 'plausible mechanism pathway,' offering clearer rules for licensing and updating gene editing treatments. Separately, Missouri Attorney General Catherine Hanaway has sent a cease-and-desist letter to Eli Lilly, demanding the company stop requiring hospitals to provide claims data to receive discounted medicines under the federal 340B Drug Pricing Program. Hanaway claims Lilly has violated its promise to sell medicines under the 340B program to eligible hospitals in Missouri, making 'false and misleading statements' and breaking state law. The letter, dated October 26, 2026, marks a growing trend of states pushing back against drugmakers' data collection practices tied to the 340B program. The 340B program, designed to help safety-net hospitals stretch scarce resources, has faced increasing scrutiny as pharmaceutical companies seek more data from participating hospitals. Meanwhile, the article also mentions that Lilly has released new weight loss drug data, though the details are not elaborated in the provided text. The STAT+ subscription model is referenced throughout the article, with multiple calls to subscribe to access full content. The piece is part of Ed Silverman's Pharmalot column, which focuses on prescription drug pricing, affordability, and access issues, as well as patents, litigation, and legislation. The article's tone is professional and informative, typical of STAT's coverage of pharmaceutical industry developments.

Application of the high-precision technique is part of a clinical trial to treat a genetic disease that can cause liver and nerve damage.

Get instant access to a concise analysis of 22 BioPharma (+ Google) CVCs and their portfolios. In minutes, you’ll see where strategic capital is flowing across Technology and Therapy Areas—and how 2025 trends compare to 2023. What’s inside: Clear mapping of CVC focus by Technology and Therapy Areas Trend shifts vs. 2023 to validate your thesis Actionable insights to spot momentum early How it works: Fill out the form to receive your report immediately. Questions or feedback? info@lqventures.com LucidQuest empowers BioPharma executives and Life Sciences investors with up-to-date analysis. Stay tuned for 2025 deep dives on Cell & Gene Therapy, Digital Health, Oncology & Hematology, and more.