This Week’s Biopharma News: Roche’s $50 Billion Investment in the US and More
Our latest news roundup reports on Roche’s US investment, a lawsuit against Vertex Pharmaceuticals, research into GLP-1s and the risk of Alzheimer’s disease, and more.
| 4 min read | News
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Roche strengthens US presence
Roche says it will invest $50 billion over the next five years to expand operations across the US. This strategic move includes expanded and upgraded manufacturing and distribution capabilities in Kentucky, Indiana, New Jersey, Oregon and California; a new gene therapy manufacturing facility in Pennsylvania; a center in Massachusetts focused on cardiovascular, renal and metabolism R&D; and a manufacturing center dedicated to weight loss medicines (location to be announced).
Roche emphasized that this investment reflects its confidence in the US market and its commitment to being a long-term partner in the country’s healthcare ecosystem. Thomas Schinecker, Roche Group CEO, said: “We are proud of our 110 year legacy in the United States which has been a key driver for jobs, innovation and the creation of intellectual property in the US, across both our Pharmaceutical and Diagnostics Divisions. Our investments of USD 50 billion over the next five years will lay the foundation for our next era of innovation and growth, benefiting patients in the US and around the world.”
CRISPR court case for Vertex
South Korean company ToolGen has filed an IP lawsuit in the UK against Vertex Pharmaceuticals and its manufacturing partners, Lonza and RoslinCT. The company claims that the Vertex therapy Casgevy infringes on ToolGen’s patents for CRISPR ribonucleoprotein complex (RNP) delivery technology. According to a statement from ToolGen, “CRISPR RNP represents a groundbreaking method that delivers the Cas9 protein directly into cells in its protein form. This approach avoids the cellular toxicity often associated with DNA- and mRNA-based systems and eliminates the risk of foreign DNA integration into the genome. With its significantly lower off-target effects providing superior safety profiles, the technology is now widely adopted in therapeutic development as well as in plant and animal genome editing.”
The company alleges that Casgevy was “born” from ToolGen’s CRISPR RNP technology. ToolGen CEO Jong Sang Ryu said, "This lawsuit is not aimed at restricting patient access to CASGEVY in the United Kingdom. Rather, it seeks to ensure ToolGen is properly acknowledged and rewarded through a fair licensing agreement."
Boehringer Ingelheim targets ALT tumors
Boehringer Ingelheim and precision oncology company Tessellate Bio have entered into a research collaboration and global license agreement to focus on the development of first-in-class, oral precision treatments targeting tumors dependent on alternative lengthening of telomeres (ALT) for their growth.
According to a statement, “Tessellate Bio has developed inhibitors of an undisclosed target that plays a key role in enabling the uncontrolled growth of ALT positive cancer cells. Blocking this target has been shown to lead to increased DNA damage, replication stress and ultimately tumor cell death, specifically in ALT positive tumor cells. A clear benefit is that healthy cells are not affected because they have no dependency on this mechanism.”
The deal is valued at over $500 million.
Research news
GLP-1 and the risk of Alzheimer’s disease
A study from the University of Florida, suggests that certain diabetes medications, including GLP-1s, may also offer protection against Alzheimer’s disease and related dementias in older adults with Type 2 diabetes. The research analyzed Medicare claims data from nearly 1.9 million patients aged 65 and older, assessing the impact of three classes of antihyperglycemic drugs: GLP-1 receptor agonists, SGLT2 inhibitors, and DPP-4 inhibitors.
After controlling for a wide range of variables, the researchers found that GLP-1 receptor agonists were associated with a 30 percent reduction in the risk of developing Alzheimer’s, while SGLT2 inhibitors correlated with a 23 percent lower risk. DPP-4 inhibitors, by contrast, showed no significant effect. The research was supported by funding from the National Institute on Aging and the National Institute of Diabetes and Digestive and Kidney Diseases.
Machine learning to optimize gene editing
Researchers at Mass General Brigham and Beth Israel Deaconess Medical Center have developed a machine learning-based method for designing custom enzymes intended for gene editing applications. The approach uses deep learning algorithms to generate enzyme variants that can be programmed to act on specific DNA targets.
The machine learning model was trained on large datasets containing protein sequences and structural features, allowing it to predict new enzyme designs with desirable editing properties. After computational generation, selected enzyme candidates were experimentally validated to assess their performance in targeting genomic sequences. The research team focused on tailoring enzymes to improve both target-site recognition and catalytic efficiency, with the goal of minimizing unintended genetic changes and maximizing editing effectiveness.
The researchers emphasize that this computationally guided design process could streamline the development of genome editing reagents, especially for targets that are difficult to access using existing enzyme libraries. They have also made a web tool to allow others to use their model.
