Orna Therapeutics brings Eli Lilly an in vivo cell therapy ready for Phase 1 testing as a potential treatment for autoimmune diseases. Lilly is now the latest pharma company to use M&A to enter this growing field, following deals made in the past year by AstraZeneca, AbbVie, Gilead Sciences, and Bristol Myers Squibb.
The growing use of advanced analytics and artificial intelligence (AI) is reshaping this landscape by enabling data-driven process control, predictive manufacturing, and greater transparency across the development lifecycle.
In a landscape where complexity has long been the norm, the power of one lies not just in unification, but in intelligence and automation.
Recent developments, including a high-profile acquisition and key regulatory changes, point to a paradigm shift. Cell and gene therapy is becoming increasingly commercially viable, with the potential to improve the lives of millions.
Non-binding offers for Galapagos’s cell therapies assets came mostly from financial investors, and none offered sufficient terms or financing to support the business, the company said. Galapagos’s exit from cell therapy work will lead to site closures in Europe, China, and the U.S.
Startup Orbital Therapeutics brings Bristol Myers Squibb an in vivo cell therapy in development for treating autoimmune disease. With the deal, BMS joins AstraZeneca, AbbVie, and Gilead Sciences in turning to M&A as a way to get in vivo cell therapy assets and technologies.
Gilead Sciences, whose Kite subsidiary was one of the pioneers of ex vivo cancer cell therapy, is joining the pursuit of in vivo therapies with the acquisition of startup Interius BioTherapeutics. The deal follows AstraZeneca and AbbVie acquisitions this year for in vivo cell therapy developers.
In a recent interview, Esperion president and CEO, Sheldon Koenig talks about the reality of statin intolerance and his company’s expansion into new diseases
Pharmaceutical tariffs are coming, part of President Trump’s stated goal of bringing drug manufacturing back to the U.S. Untangling the sprawling, global supply chain will be complicated and expensive, and could inadvertently compete with other administration goals regarding drug pricing and availability.
Capricor Therapeutics said the FDA asked for more data supporting the efficacy of Deramiocel, the biotech’s off-the-shelf cell therapy for treating the heart complications of Duchenne muscular dystrophy. Those data could come from an ongoing Phase 3 study expected to yield preliminary results soon.
The FDA has eliminated a safety monitoring requirement and eased certain restrictions that had been in place since CAR T-therapies first reached the market in 2017. The agency and financial analysts say the changes should improve patient access to these personalized cancer treatments.
In Phase 1/2 results for 12 patients at least one year after receiving the cell therapy, zimislecel, Vertex Pharmaceuticals reported all participants achieved blood glucose in the target range and 10 of the 12 were free of exogenous insulin as of the data cutoff. Vertex expects to complete enrollment in the pivotal portion of the study this summer, paving the way for potential regulatory submissions next year.
Greenway Health blends smart technology with human expertise to elevate revenue cycle.
Galapagos has dropped plans for a business separation that would have enabled the legacy company to focus exclusively on developing new cancer cell therapies. Instead, Galapagos said it will pursue strategic alternatives for its assets.
Abeona Therapeutics makes Zevaskyn by engineering a patient’s biopsied skin cells to express the collagen that they lack, then growing those cells into sheets that can be surgically applied to a wound. Abeona's cell-based gene therapy is just the third FDA-approved treatment for epidermolysis bullosa, a rare disease that leads to skin described as thin and fragile like a butterfly’s wings.
Caribou Biosciences’ restructuring narrows the biotech’s focus to two off-the-shelf cell therapies for blood cancers, whose key data readouts have been pushed out to the second half of 2025. It’s the CRISPR-editing company’s second cash-saving restructuring in the past year.
As the FDA’s top biologics official, Peter Marks oversaw regulation of cell and gene therapies. The departure of Marks and mass layoffs throughout the FDA raise concerns about the agency’s ability to review and potentially approve such treatments.
Vertex Pharmaceuticals is stopping development of VX-264, an experimental cell therapy/medical device treatment for type 1 diabetes, after an early look at Phase 1/2 data fell short of efficacy goals. This program came from a $950 million acquisition in 2019 that marked Vertex’s entry into type 1 diabetes.