Lotus Neuro launches to deliver therapies across blood-brain barrier

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Lotus Neuro, a clinical-stage biotechnology company pairing focused ultrasound with leading therapeutic modalities to treat focal neurologic disease, has announced its public launch.

Spun out in December 2025 from Insightec, Lotus Neuro is built on a clinically validated platform for precision modulation of the blood-brain barrier (BBB) and is dedicated to solving the delivery problem that has stalled decades of promising neuroscience.

The blood-brain barrier protects the brain, but it also keeps the vast majority of drugs from reaching it in effective concentrations. Many therapies that should work in the brain never get the chance, and a delivery failure is often read as a failure of biology. Lotus Neuro’s platform uses the most clinically validated focused ultrasound technology in combination with circulating microbubbles to modulate the barrier across precisely targeted brain volumes, from a few millimetres to a large hemispheric region, in combination with therapeutic administration. The drug itself does not require modification, which makes the approach modality-agnostic: small molecules, antibodies and antibody-drug conjugates, enzymes, gene therapies, oligonucleotides and cell therapies can all be paired with the technology.

The platform has been used in approximately 600 treatment sessions across brain tumours, Alzheimer’s disease, Parkinson’s disease and ALS, including delivery across four classes of therapeutics. In glioblastoma, a multicentre phase 1/2 trial published in The Lancet Oncology in 2025 reported median overall survival of 31.3 months when the barrier was opened during standard chemotherapy, versus 19.3 months in a matched control cohort, with no treatment-related deaths. In Alzheimer’s disease, a study published in The New England Journal of Medicine in 2024 found greater amyloid plaque reduction in regions where the barrier was opened alongside aducanumab than in the matching untreated regions. In Parkinson’s disease, a phase 1 study delivered an enzyme replacement therapy repeatedly to the putamen, and in HER2-positive brain metastases a radiolabelled antibody roughly doubled its uptake in sonicated lesions. Gene therapy vectors and oligonucleotides have been delivered to targeted brain regions in non-human primates, and the barrier has been opened safely over the motor cortex in ALS.

“Lotus Neuro exists because science is not the problem, delivery is,” said Arjun “JJ” Desai, founder and chief executive officer of Lotus Neuro.

“Brain disease is complex and variable, demanding an answer that is both personalized and precise. Through extensive pre-clinical and clinical testing, a global network of the leading neuroscience centres has demonstrated that focused ultrasound can safely pair with therapeutics and reach anywhere in the brain, delivering medicines where and when they are needed most.”

Lotus Neuro is launching with a partnership-first strategy, working with pharmaceutical and biotech companies and with academic centres to bring promising therapeutics precisely to their brain targets. With a globally established clinical network and leading technology safety profile, Lotus Neuro is focused on near-term clinical development, using advances in blood, tissue and imaging analysis to characterise the most promising combination therapies.

Through its Proof of Pharmacology (POP) Challenge, launched in June 2026, Lotus Neuro is funding investigators at leading neuroscience institutions to add focused ultrasound study arms to existing investigational drug programs, generating clinical evidence of drug delivery and target engagement in the brain.

“Too many CNS drug trials have failed without first confirming that the therapy reached its intended target in the brain at a meaningful concentration,” said Ajay Verma, founding scientist of Lotus Neuro.

“Without that evidence, a negative result cannot distinguish between an ineffective target and inadequate drug delivery. Focused ultrasound enables us to address this question with precision, tailoring blood-brain barrier modulation to a specific brain region and neurological indication. This approach allows us to assess earlier, directly in patients, whether a promising therapy is reaching its target and producing the intended biological effect.”

“Because we don’t alter the drug, we can move at the speed of our partners’ science, whether that is a small molecule, an antibody, an enzyme or a gene therapy,” said Junghae Suh, chief technology officer of Lotus Neuro.

“Our work is to ensure that targeted delivery to the brain is as rigorous and repeatable as the medicines it carries.”

Insightec continues to advance and commercialise the core focused ultrasound platform, while Lotus Neuro applies it to new therapeutic frontiers in the brain, supported by shared translational data and close scientific collaboration across the two organizations.

“Brain health is the last frontier in medicine, and for a long time the tools simply weren’t there,” said Maurice R. Ferré, chairman and CEO of Insightec and a member of the Lotus Neuro board of directors.

“Insightec proved that focused ultrasound could safely and precisely reach the brain without a single incision. Lotus Neuro is the natural next chapter: taking that platform from treating symptoms to delivering the medicines that can change the course of disease, across brain cancer and neurodegeneration alike. Together we’ve built a complete ecosystem, from device to drug delivery, and JJ Desai is exactly the person to lead it.”