EsoBiotec’s platform has the potential to eliminate the complex and time-consuming process for manufacturing, helping to make CAR-T cell therapies more accessible.
AstraZeneca is buying EsoBiotec for up to $1 billion for the company’s in vivo CAR-T cell drugs, or therapies that reprogram immune cells directly within patients' bodies. Analysts say the deal highlights the pharma industry’s growing interest in this technology.
Current CAR-T cell therapies have transformed the treatment landscape for certain blood cancers, but the drugs are complex and time-consuming to manufacture and carry high price tags. They also carry weeks-long post-treatment monitoring protocols and are mostly available only at major medical centers, limiting access. In vivo approaches aim to overcome these limitations and potentially extend cell therapy benefits to more patients.
EsoBiotec’s platform uses immune-shielded lentiviral vectors — HIV type 1 cells that have had their genetic information removed — to deliver genetic instructions directly to T cells inside the patient, eliminating the need for complex cell extraction, laboratory modification and reinfusion processes that traditional CAR-T cell therapies require. This allows for treatment in "just minutes" through a simple IV injection and without the need for immune cell depletion, AstraZeneca said in its announcement.
Susan Galbraith
“The platform has the potential to transform cell therapy and will enable us to scale these innovative treatments so that many more patients around the world can access them,” said Susan Galbraith, executive vice president, Oncology Hematology R&D at AstraZeneca, in a news release.
The acquisition represents a broader industry trend as pharmaceutical companies increasingly pursue in vivo cell therapy approaches, according to a research note from Jefferies. The note cited several companies developing similar technologies through different gene delivery strategies, including Interius, Umoja and Kelonia, which are employing lentiviral vectors to directly reprogram T cells in the body, and Capstan, Myeloid Therapeutics, Orna, Orbital and Carisma, which are using an mRNA-based approach to rapidly induce CAR expression in target cells.
“Key advantages of this platform include precise immune cell targeting, resistance to immune clearance, and modular design for flexible therapeutic applications,” Jefferies analysts wrote in a March 17 note. “Importantly, this approach eliminates the need for complex ex vivo T cell programming (faster turnaround times) and lymphodepletion, potentially making CAR-T more accessible and scalable.”
EsoBiotec's most advanced pipeline asset has already dosed its first multiple myeloma patient in a China investigator-initiated trial. Multiple myeloma is a cancer that forms in plasma cells in bone marrow. About 36,110 new cases will be diagnosed this year, and about 12,030 patients with multiple myeloma will die, according to the American Cancer Society.
According to Jefferies, the first patient received a single dose without prior lymphodepletion and showed "high levels of T cell reprogramming and CART expansion" with minimal residual disease in the bone marrow undetectable by day 28.
ESO-T01 is the first in vivo B-cell maturation antigen (BCMA) CAR-T candidate to be dosed in a human clinical trial, according to EsoBiotech in a news release. The therapy combines the immune-shielded cell-specific lentiviral vector platform, ENaBL, that reprograms immune cells inside the body with a CAR-T transgene developed by Pregene Biopharma.
“By combining our expertise and resources [with AstraZeneca}, we can accelerate the development of our in vivo platform, which has a novel delivery technology we believe will have broad therapeutic applicability,” said Jean-Pierre Latere, CEO, EsoBiotec, in a company news release.
AstraZeneca acquisition of EsoBiotec is expected to close in the second quarter of 2025, and EsoBiotec will become a wholly owned subsidiary of AstraZeneca.
Solid Biosciences Reports Positive Data from DMD Gene Therapy Trial
February 19th 2025Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.
Read More
Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates
January 31st 2025The majority are in the very early preclinical stage of development, but applications for regulatory approval have been submitted for 11 gene therapies worldwide, and 35 are in phase 3 trials, according to report delineating the gene therapy development landscape.
Read More