NEW YORK – The National Institutes of Health's All of Us research program said this week that it has started limited enrollment of children between the ages of 0 and 4 years at five partner healthcare organizations in Pennsylvania, Arizona, Michigan, California, and Connecticut. To do so, the program needed to develop protocols that are specific to children and update its technology platforms. All of Us started enrolling kids in late 2023 as part of a pilot phase and modified the enrolling process after gaining feedback from its partners and parents. The program plans to enroll a few hundred children and their families this year. Pending available funding, it hopes to increase pediatric enrollment to include older children and additional sites.
The CMT Research Foundation, a nonprofit that funds investigations into treatments for Charcot-Marie-Tooth (CMT) disease, this week announced that it has invested into a research project at Nationwide Children's Hospital to develop vehicles for delivering gene therapies more efficiently into peripheral nerves. The project is led by Afrooz Rashnonejad, a principal investigator at the Center for Gene Therapy at Nationwide Children's Abigail Wexner Research Institute. Nerea Zabaleta, a researcher at the Grousbeck Gene Therapy Center at Mass General Brigham's Mass Eye and Ear, is a collaborator on the project.
Taiba Healthcare Group said this week that it has invested $12 million in Immuneel Therapeutics to advance CAR T-cell research and access in developing countries, especially in the Middle East and Africa. Taiba intends for the strategic investment to go toward making CAR T-cell therapy commercially available and affordable as well as starting a Phase II clinical trial of Immuneel Therapeutics' CAR T-cell therapy IMN-003A in patients with certain leukemias and lymphomas.
The special committee of 23andMe's board of directors this week responded to an offer by CEO Anne Wojcicki earlier this week to take the company private by purchasing all outstanding shares for $.40 per share in cash. In a letter to Wojcicki, the committee said it is "disappointed" by the proposal because it provides no premium to the company's share closing price on July 31, lacks committed financing, and is conditional in nature. "Accordingly, we view your proposal as insufficient and not in the best interest of the non-affiliated shareholders," it wrote, adding that it is "not prepared to move forward under the terms provided" and that it plans to assess whether there is interest in the company from third parties. That said, the committee said it is willing to give Wojcicki and her potential investors "a limited amount" of additional time to submit a revised proposal that meets its expectations and requirements. In the meantime, the committee intends to hire a consultant to work on a revised business plan to help the company achieve "a more sustainable financial profile" and profitability.
Redwood City, California-based Adverum Biotechnologies this week said it has received regenerative medicine advanced therapy designation from the US Food and Drug Administration for ixoberogene soroparvovec (Ixo-vec), a gene therapy candidate it's developing for wet age-related macular degeneration. Adverum is testing the gene therapy in the ongoing Phase II LUNA trial and Phase I OPTIC extension study. The company said it plans to share updates from regulatory discussions with the FDA and European Medicines Agency on a pivotal clinical trial in Q4.
Germany's Federal Institute for Drugs and Medical Devices (BfArM) has announced six genome data centers (GRZs) and seven clinical data nodes (KDKs) for the country's new national precision medicine model project, following the passage of an ordinance by the Bundesrat, one of Germany's federal legislative chambers, earlier this month. The GRZs are located at Max Delbrück Center Berlin, Technical University of Dresden, German Cancer Research Center (DKFZ) Heidelberg, University of Cologne, Technical University of Munich, and University of Tübingen. The KDKs are located at University Hospital Dresden, National Center for Tumor Diseases Heidelberg, University Hospital Cologne, University of Leipzig (two data nodes), and University Hospital Tübingen (two data nodes).
Boston Oncology and King Fahd Medical City have signed an agreement to establish local access to cell and gene therapy in Saudi Arabia including research and development and clinical trials. Boston Oncology will contribute advanced technologies in cell and gene therapy, while KFMC will provide clinicians and infrastructure, the company and KFMC said this week in a statement.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.