Recent Advances and News in In Vivo Therapeutic Technologies (2025)

Major pharmaceutical firms are investing heavily in in vivo CAR-T therapies, which modify patients’ T cells directly within the body, bypassing complex and expensive ex vivo cell manipulation1 9 10.

AbbVie's $2.1 billion acquisition of Capstan Therapeutics and Bristol Myers Squibb's $1.5 billion acquisition of Orbital Therapeutics underscore strong financial and strategic support for in vivo cell and gene therapy platforms, especially for targeting autoimmune and hematologic diseases1 7 8 10 11.

Recent clinical data on in vivo CAR-Ts—such as Capstan’s CPTX2309 (targeting B cell-mediated autoimmune diseases) and EsoBiotec’s therapy for multiple myeloma—showed encouraging responses, including complete cancer resolution in some patients, but also revealed immune-related side effects requiring further study with larger cohorts1 5.

The FDA has granted fast track designation to Umoja Biopharma’s UB-VV111, an in vivo CAR-T therapy for relapsed/refractory B-cell malignancies, recognizing its potential to fill significant unmet needs and to streamline the therapy development process4.

In vivo approaches are transforming therapy logistics by enabling off-the-shelf, scalable, and more accessible treatments compared to traditional ex vivo CAR-T, reducing manufacturing costs, time, and complexity, and potentially broadening patient access1 2 5 6.

Technological advances showcased in 2025 include targeted lipid nanoparticles (tLNPs) for RNA delivery, lactate-sensitive nanoparticle drug delivery, bioink-targeted therapeutics, and click-chemistry protodrugs—all designed to activate or convert into effective therapies only inside the patient for improved safety and potency1 3 6.

Clinical and scientific consensus highlights ongoing challenges such as establishing optimal dosing regimens, avoiding immunogenicity, and managing adverse effects; more data from ongoing trials will be critical to inform future clinical practice5.

There is a notable expansion in the therapeutic scope for in vivo platforms, extending beyond cancers into autoimmune diseases and potentially other indications, cementing their role as a disruptive force in regenerative and precision medicine7 8 9 11.