J&J bets $785 million on Sail Biomedicines and the future of in vivo CAR-T
Johnson & Johnson is making another major move into the rapidly developing in vivo CAR-T space, entering into a strategic collaboration with Sail Biomedicines that could ultimately see the pharmaceutical giant acquire the company for an additional $2.58 billion. The deal includes an upfront payment of $785 million, comprising a $465 million equity investment, alongside up to $140 million in development milestones. J&J will also have an exclusive option to acquire Sail.
The partnership will focus initially on Sail’s in vivo CAR-T programme, led by SAIL-0839, which is being developed for autoimmune diseases and is designed to target CD4 and CD8 T cells. Rather than extracting and genetically engineering a patient’s T cells outside the body, in vivo CAR-T approaches aim to deliver the necessary genetic instructions directly to T cells within the patient. Sail’s platform uses nanoparticles and circular RNA technology to drive targeted and durable CAR expression.
The deal is another indication of the growing strategic interest from major pharmaceutical companies in moving CAR-T beyond traditional ex vivo manufacturing. In vivo approaches could potentially address some of the key limitations associated with conventional CAR-T, including complex manufacturing, lengthy vein-to-vein times and the need for lymphodepleting conditioning. The possibility of an off-the-shelf treatment that can be administered directly to patients could therefore significantly alter the scalability and accessibility of cell therapy.
The scale and frequency of these transactions suggest that in vivo CAR-T is increasingly moving from an experimental concept towards a strategically important modality for the biopharma industry. For developers, however, the challenge will be translating promising preclinical delivery and targeting technologies into safe, controllable and durable clinical treatments. As investment accelerates, the ability to demonstrate precise delivery, appropriate cell targeting and consistent manufacturing of the underlying delivery platforms will become increasingly important.
Read more via First Word Pharma and Fierce Biotech
Death of six-year-old following experimental gene-editing treatment raises questions over clinical oversight in China
A six-year-old girl in China has died following an experimental gene-editing treatment, with an investigation by Science and Retraction Watch raising serious questions about the clinical, ethical and regulatory processes surrounding the treatment. The child, referred to as “Mei”, received an experimental base-editing therapy intended to address a rare genetic condition affecting her cognitive development. She died approximately seven days after receiving an infusion of trillions of viral particles designed to deliver the gene-editing machinery into her brain.
The case has attracted particular concern because the treatment was undertaken despite safety signals emerging from preclinical testing. According to the investigation, all four monkeys included in a subsequent toxicology study developed moderate to severe liver damage following treatment. Experts cited by Science questioned whether these findings should have prompted further dose-ranging and safety studies before the therapy was administered to a child.
Shanghai Jiao Tong University School of Medicine, which was affiliated with the research, has since established a working group to investigate the circumstances surrounding the case. The investigation comes against the backdrop of China’s efforts to strengthen oversight of gene-editing research following the 2018 scandal involving the creation of gene-edited babies.
The case is a stark reminder that the rapid development of gene-editing technologies must be matched by equally rigorous approaches to preclinical safety, patient selection, informed consent and regulatory oversight. As the field moves towards increasingly personalised and potentially curative interventions, the consequences of inadequate translation from animal models to humans can be profound.
Read more via Science and NY Post
FDA advisory committee votes against Capricor’s Duchenne cell therapy
Capricor Therapeutics has suffered another regulatory setback after an FDA advisory committee voted 9-3 against the effectiveness of its investigational cell therapy deramiocel for Duchenne muscular dystrophy-associated cardiomyopathy. The vote followed a contentious meeting in which regulators and the company disagreed over the interpretation of clinical trial data and the statistical analysis used to demonstrate efficacy.
The vote does not represent a final rejection: advisory committees provide recommendations rather than binding decisions, and the FDA remains able to approve the therapy. The agency is expected to make its final decision on deramiocel by 22 August 2026.
The outcome reinforces the increasingly high bar for demonstrating clinical benefit in advanced therapies, particularly where trials involve small patient populations and complex endpoints. For cell and gene therapy developers, the Capricor case highlights the importance of agreeing statistical methodologies and endpoint strategies with regulators early, ensuring that clinical evidence is sufficiently robust to withstand scrutiny at the point of approval. It also illustrates the challenges of generating definitive evidence in rare diseases, where patient numbers are limited but the unmet need is substantial.
Read more via Biopharma Dive and Fierce Biotech
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