Tag: Cognitive focus and executive control

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FcRn Inhibition in Autoimmune Disease

Eric Venker,
Eric Venker, MD, PharmD
CEO, Immunovant

Although immunoglobulin G (IgG) normally protects the body against pathogens, it can become problematic in many autoimmune diseases like lupus, rheumatoid arthritis, Graves’ disease, myasthenia gravis, and Sjögren’s disease.

“In these conditions, the immune system is creating defective IgGs—called autoantibodies—that are no longer fighting infections,” explained Eric Venker, MD, PharmD, CEO of Immunovant. “Instead, they are attacking a part of your normal functioning body and causing dysfunction.”

Leonard L. Dragone
Leonard L. Dragone, MD, PhD
Disease Area Leader
Johnson & Johnson Innovative Medicine.

Historically, autoimmune conditions have been challenging to treat because therapies like steroids rely on broad immune suppression, noted Leonard L. Dragone, MD, PhD, disease area leader of autoantibody and rheumatology at Johnson & Johnson Innovative Medicine. These non-specific approaches are often inconsistently effective and lead to adverse side effects.

“For many autoimmune diseases, there is a need for more targeted strategies that address disease-causing autoantibodies directly, rather than broadly suppressing the immune system,” emphasized Dragone. Beginning in 1998, the U.S. Food and Drug Administration (FDA) approved infliximab, a tumor necrosis factor (TNF)-α inhibitor, for the treatment of Crohn’s disease. This marked the first approval of a monoclonal antibody for the treatment of a chronic condition. Since then, targeted therapies for autoimmune diseases have expanded to address cytokine signaling pathways (TNF-α, IL-6, IL-17, IL-23), Janus kinase (JAK–STAT) signaling, and immune cell surface markers (CD20).

Another such targeted strategy involves an emerging drug class called FcRn blockers, which are now showing considerable promise in the treatment of certain autoimmune diseases.

FcRn blockers, which typically consist of monoclonal antibodies or antibody fragments, work by blocking the function of a protein receptor called FcRn (neonatal Fc receptor). This prevents IgG recycling, thereby reducing IgG levels in the body.

Immunovant
FcRn maintains levels of IgG in circulation by preventing IgG degradation in the lysosomes of cells. However, FcRn drugs block this pathway.

Venker compares FcRn inhibitors to cholesterol-lowering drugs such as statins. “LDL is the disease-causing agent that healthcare providers target to prevent many cardiovascular diseases. Likewise, in the case of FcRn blockade, we are aiming to lower IgG. We believe that deeper IgG reduction may provide improved results.”

As of early 2026, the FDA has approved three FcRn inhibitors for the treatment of myasthenia gravis, a chronic autoimmune disorder affecting up to 100,000 people in the U.S. Efgartigimod (approved in 2021), rozanolixizumab-noli (approved in 2023), and nipocalimab-aahu (approved in 2025) all work by reducing pathogenic IgGs associated with the disease.

Myasthenia gravis results
Myasthenia gravis results from harmful antibodies (anti-AChR or anti-MuSK) produced by the immune system that interfere with signaling in the neuromuscular junction.

“With FcRn blockers, it is exciting to know that there is now a targeted mechanism for patients around the world with autoimmune diseases caused by an IgG autoantibody,” said Venker.

Tackling Graves’ disease

In Graves’ disease, an IgG autoantibody called thyrotropin receptor antibody (TRAb), which targets the thyroid-stimulating hormone (TSH) receptor of the thyroid, is produced. The condition, which is the most common cause of hyperthyroidism, causes elevated heart rate, shakiness, irritability, muscle weakness, and weight loss.

“TRAb is an IgG antibody, but it is a badly behaving one that is basically hijacking the thyroid system,” noted Venker. “It doesn’t serve any purpose that is normal at all.”

Mark A. Lupo
Mark A. Lupo, MD
Founder and Medical Director
Thyroid & Endocrine Center of Florida

Unfortunately, the toolkit for treating Graves’ disease hasn’t changed much since 1950, when the FDA approved the drug methimazole, said Mark A. Lupo, MD, founder and medical director of the Thyroid & Endocrine Center of Florida.

Methimazole is an anti-thyroid drug that slows down the production of thyroid hormones. Although Graves’ patients benefit from anti-thyroid drugs, Lupo estimates a 50% relapse rate within two years of discontinuing these drugs.

Other options for treating Graves’ disease include surgical removal of the thyroid or the use of radioactive iodine to induce destruction of the thyroid gland. However, these approaches result in permanent hypothyroidism, and patients typically require lifelong thyroid hormone replacement after treatment.

Because TRAb is an IgG, FcRn drugs represent a potential autoimmune solution for Graves’ disease. Like all FcRn blockers, they may work by decreasing TRAb recycling and lowering TRAb levels.

Lupo highlights Immunovant’s recent proof-of-concept study of an FcRn inhibitor for Graves’ disease, the first such study for the condition. “Despite the small number of patients (around 25), the results from this study suggest a potential, durable remission six months off treatment,” said Lupo.

While study participants experienced an increase in total IgG levels following treatment, TRAb levels remained low over a six-month period. The thyroid also decreased in size. “To see TRAb levels down six months off the study drug caught the attention of the endocrine thyroid community,” noted Lupo.

“What was unexpected was that TRAb, the disease-causing antibody, stayed down for many months after stopping the investigational therapy,” added Venker.

“I think we are overdue for a new option in Graves’ disease that could help break some of these methimazole cycles and potentially address not the innocent thyroid gland but the underlying immune system issues,” concluded Lupo.

But are they safe?

Venker recalls that safety was an initial concern with FcRn inhibition. After all, these drugs work by reducing IgG, an essential part of the immune system. “Any time you are using an autoimmune drug that potentially suppresses your immune system, you have to think about going too far. Am I going to cause an infection or weaken the immune system?

“So far, this investigational drug has demonstrated a safety profile we expected, and appears positive,” noted Venker. “That makes sense mostly because FcRn blockade is pretty targeted.”

“Although there are no head-to-head comparative safety trials yet, most clinicians and principal investigators view FcRn blockers as relatively safe,” added Lupo. “There are FcRn blockers on the market, and they have demonstrated a good safety record in patients.” The most common side effect tends to involve injection site reactions with either intravenous or subcutaneous delivery.

Preventing fetal exposure

During pregnancy, maternal antibodies—called alloantibodies—can cross the placenta and attack the organs and tissues of the fetus, explained Dragone.

A distinguishing feature of Johnson & Johnson’s nipocalimab is its pH-independent binding to FcRn. This allows it to bind with high affinity in the placenta, a low-pH environment.

The drug is currently showing potential in the treatment of two alloimmune diseases of pregnancy: hemolytic disease of the fetus and newborn (HDFN) and fetal and neonatal alloimmune thrombocytopenia (FNAIT), said Dragone. These conditions can arise during alloimmunized pregnancies, when the pregnant person’s immune system forms alloantibodies against fetal red blood cells (HDFN) and/or fetal platelets (FNAIT). Importantly, published data on nipocalimab suggest minimal transfer of the drug to the fetus or infant. “Therapies like nipocalimab offer a blueprint for how precision medicine can expand to include pregnant people, a population that has historically been excluded from drug development,” noted Dragone. “Our approach with nipocalimab has the potential to change how we think about treating autoantibody-driven diseases in people of childbearing age.”

FcRn blockers bind to FcRn receptors
Nipocalimab (IMAAVY®) and other FcRn blockers bind to FcRn receptors and reduce levels of both normal and harmful IgG antibodies.

The FDA has granted a fast track designation to nipocalimab for both FNAIT and HDFN, and Phase III studies are underway to further investigate the drug in both diseases.

Expanding indications

“There are probably 20 trials out there for FcRn blockers, and many are likely to work,” noted Venker. “There are a ton of potential new indications under investigation, including rare diseases that have been ignored historically.”

He notes that Immunovant’s pipeline alone includes potential indications in endocrinology (Graves’ disease), rheumatology (rheumatoid arthritis, Sjögren’s disease, and cutaneous lupus erythematosus), and neurology (myasthenia gravis and chronic inflammatory demyelinating polyneuropathy).

Venker stresses that no FDA-approved solutions exist for Sjögren’s disease, which affects as many as four million Americans. The condition causes severe dry eyes and mouth, fatigue, and joint and muscle pain. Immunovant and Johnson & Johnson are conducting clinical trials to evaluate FcRn blockers for the disease.

Hani Houshyar
Hani Houshyar, PhD
Strategy Team Lead
argenx

Meanwhile, argenx’s FcRn inhibitor efgartigimod has been used in 19,000 people worldwide for myasthenia gravis and other autoimmune conditions, said Hani Houshyar, PhD, FcRn asset strategy lead for argenx.

“However, we believe myasthenia gravis is just the beginning,” she said. As of 2026, the company has active clinical trials to test the drug’s effectiveness in additional autoimmune diseases with high unmet medical need, like myositis, Sjögren’s disease, ocular myasthenia gravis, systemic sclerosis, Graves’ disease, and autoimmune encephalitis.

UCB’s rozanolixizumab was the first FcRn blocker to be approved for the treatment of generalized myasthenia gravis in adults who are positive for anti-AChR or anti-MuSK antibodies, who together account for approximately 90% of cases, said Omar Sinno, MD, UCB’s U.S. medical strategy lead of rare disease. So far, the drug has been approved in the U.S., Canada, the EU, Australia, Switzerland, China, Turkey, and Korea.

Omar Sinno
Omar Sinno, MD
Medical Strategy Lead, UCB

Rozanolixizumab is administered via a convenient subcutaneous infusion rather than intravenously. The company’s long-term studies demonstrate robust IgG reductions (up to 75%) with sustained benefit across multiple treatment cycles. UCB is also investigating rozanolixizumab as a potential treatment for a rare autoimmune condition called myelin oligodendrocyte glycoprotein antibody-associated disease.

Finally, Johnson & Johnson’s nipocalimab is in mid-to-late-stage studies for Sjögren’s disease, lupus, warm autoimmune hemolytic anemia, and chronic inflammatory demyelinating polyneuropathy.

Drugs in development

Viridian Therapeutics is currently investigating two FcRn inhibitors, VRDN-006 and VRDN-008, said Steve Mahoney, president and CEO. Both candidates are designed as subcutaneous products that can be conveniently self-administered by the patient.

Steve Mahoney
Steve Mahoney
President and CEO
Viridian Therapeutics

VRDN-006 is an Fc fragment in Phase I trials, while VRDN-008 is made up of an Fc fragment and an albumin-binding domain designed to prolong IgG suppression. Mahoney notes that VRDN-008 showed a longer half-life and more sustained IgG reduction than efgartigimod in a high-dose, head-to-head study in non-human primates.

Clinical trial results of VRDN-008 in healthy volunteers are expected later in 2026. “What we believe differentiates VRDN-008 from other FcRn inhibitors is a longer half-life, which has the potential to support less frequent dosing for patients to enhance convenience,” said Mahoney.

Although three FcRn blockers are currently FDA-approved to treat myasthenia gravis in the U.S., Venker notes that Immunovant is continuing to investigate the condition with the company’s follow-on FcRn candidate, imeroprubart (IMVT-1402).

In Immunovant’s proof-of-concept study for Graves’ disease, TRAb stayed low even six months after the investigational treatment was discontinued. But how long will this effect last? “We don’t know that yet because our randomized trials with IMVT-1402 are ongoing,” Venker said. “However, Graves’ disease has given us the first hint that FcRn drugs may be able to put certain autoimmune conditions into permanent remission.”

Viridian’s VRDN-006 illustration
Viridian’s VRDN-006 (top) is an Fc fragment, whereas VRDN-008 (bottom) is made up of an Fc fragment and an albumin-binding domain designed to prolong IgG suppression.

“A key question for autoimmune disease, the holy grail, so to speak, is whether we can reset the immune system so the person can function normally without medication for the rest of their lives,” he added.

Finally, argenx is developing ARGX-213, a next-generation FcRn inhibitor engineered to extend half-life and sustain IgG reduction.

“Looking ahead, FcRn inhibition represents an increasingly important approach across IgG-driven disease,” noted Sinno. “By selectively reducing pathogenic IgG, these agents enable more targeted autoimmune care. And as clinical experience with FcRn inhibition grows, treatment paradigms may shift toward earlier intervention.”

 

Tiffany Yesavage, PhD is a freelance writer from Denver, Colorado.

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In Conversation with Haijiao Liu, PhD

Haijiao Liu, PhD
Haijiao Liu, PhD

As a postdoctoral researcher at the University of Pennsylvania, Haijiao Liu, PhD, helped advance tumor-on-a-chip technology, a feat of bioengineering that mimics the microenvironment of malignant human tumors. Led by Dan Dongeun Huh, PhD, a Penn Engineering professor and trailblazer of organ-on-a-chip technology, Liu and his team explanted lung adenocarcinoma tumors onto the transparent chips to test their perfusion with chimeric antigen receptor (CAR) T cells. Their findings were published in October in Nature Biotechnology, with Liu as first author.

Now on paternity leave in Toronto, Liu spoke with Lindsey Leake about the implications of this work, the challenges inherent to tumor-on-a-chip studies, and his plans to launch a lab of his own this fall.

Q: Walk me through the creation of the tumor-on-a-chip. What went into its design?

Haijiao Liu: It’s essentially inspired by the need for alternative tumor models. This is speaking to the traditionally used animal tumor models and some existing in vitro tumor models, especially for the study of immunotherapies.

For example, when I started at Penn around 2018, Penn Medicine was pioneering this immunotherapy called CAR T-cell therapy, which is basically aiming to harness the patient’s own immune system, specifically the patient’s own T cells, to help fight the cancer. Penn Medicine was demonstrating huge clinical success using this CAR T therapy to treat blood cancers, such as leukemias and lymphomas. In a huge contrast to this, the solid cancer arena has seen a limited response from this new immunotherapy. So there’s this great need to study why this has not been successful, and that comes down to the consensus that the solid tumor has this really complex microenvironment.

In the category called tumor-on-a-chip, people try to control the cultural environment, the biochemical and biophysical environment of tumor cell cultures. We can use this for simple drug testing—see how the tumor growth will be affected or how effectively they can be killed. However, these existing tumor-on-chip or in vitro tumor models are still very simple. They don’t usually recreate or reproduce the complex structure of the human solid tumors that I described, like where they often include complex vessel networks.

I took the lead to address the need and the challenges of reproducing and then investigating, or probing, the dynamic interactions between those CAR T cells and human solid tumors entirely in vitro.

Q: How does the vascularization work on the chip?

Liu: It took several years to start, from the idea of building this more advanced tumor-on-a-chip technology toward proving it’s actually useful. I started by focusing on this one aspect, which is the CAR T-cell trafficking and their functions after they traffic to fight the tumors, and that will involve the recreation of the structural interface between the tumor and this complex vascular network that’s present in human tumors.

I was inspired by in vivo tumor transplantation, where traditionally, people take human tumors and then transplant them in a bulk, intact format into animal models. So my idea was, if we want to focus more on the human biology, if we want to engineer this entirely in vitro, how about we design a vascular bedding, like a miniature living model?

We basically took advantage of the self-assembly capability of human-sourced endothelial cells, combined with certain stromal fibroblasts, or stromal cells. With a bit of optimization, engineering, tweaking, then we can allow them to form capillary-like vascular networks in our engineered models.

Q: What are the advantages of recreating the tumor microenvironment in this way? That is, is the Petri dish becoming obsolete in cancer research?

Liu: The unique advantage of this way of engineering is to have a higher level of control over the structures of the tissue-tissue interface that we can build. For example, we can engineer different culture chambers. We can engineer different access windows with this model. That allows us to construct, step by step, the vascular bedding and then the tumor transplantation. Also, by forming these perfusable vessels—by the way, we can provide the infusion and flow of the CAR T cells, just like they are infused and flow in the patient—that gives us the leverage to reconstruct, probe, and then control these tissue functions in a highly precise manner.

Q: How did you and your colleagues at Penn explore CAR T-cell activity on the chip?

Liu: We first used different functional assays, like immunostaining and ELISA (enzyme-linked immunosorbent assay) assays, to characterize how the CAR T cells are doing and how they are interacting with the tumors in our engineered model. Then we disassembled this engineered tissue to extract all the cells for flow cytometry, to further characterize their functional phenotypes.

With the help of our collaborators and other people in the lab, I took advantage of this engineered model of vascular tumors interacting with CAR T cells for multi-omics analysis. For example, I was able to extract all the cells and send them for single-cell RNA seq[uencing]. We were able to look at the gene expressions of each individual cell from all the cell types that we included in this model. In this way, we have almost like a superpower to probe and read into how each cell—including the CAR Ts and tumors and the vessels—how they are responding and interacting with each other at the molecular gene levels. This is so powerful that it helped me to discover novel interactions between these parties and also new druggable targets.

The message is that through the development of this more advanced tumor-on-a-chip technology—combined with advanced multi-omics analytics and advanced computational analysis—we were able to provide this powerful in vitro technology to apply to accelerate the development of cell therapies, such as the CAR T immunotherapies for cancer, but also other complex diseases.

Q: What are the overall implications of this latest research?

Liu: With a growing understanding of human biology at the cellular and tissue levels, I think we’re seeing that our ability to engineer and design biological systems is also growing. More than ever, we have these advances in the ability to precisely construct, investigate, and then eventually control very complex tissue functions, and even organ functions. For example, our demonstrated tumor-on-a-chip technology is like presenting a miniature sandbox; we can literally see and predict the battlefield of CAR T therapy in cancer.

If we combine these advanced engineering technologies with emerging technologies in spatial multi-omics and the unprecedented productivity of the AI revolution, we will be able to accelerate the understanding of more complex human biology and extract more biological insights, and then apply that to accelerate the development of safer and more efficacious drugs and therapies, such as immunotherapies in cancer.

Q: What are the limitations of organ-on-chip technology that need to be overcome?

Liu: I think there are challenges on two fronts. The first limitation is the lack of complexity. We’re claiming that what we just published is a sufficiently complex system for us to deeply probe and understand the dynamics of CAR T tumor interactions. Still, if we’re speaking next level of translational power or potential, then we need to pursue a higher complexity that incorporates the missing but critical components from in vivo.

The other side of the coin is that if you make this engineered model more complex, you make it more challenging to reproduce or to scale up or to translate to other labs. But also, that points to an opportunity and growing room for translation, to standardize every single step, from the construction to the analysis of these engineered models, and to automate these processes as much as possible.

Q: What do you envision for your new lab?

Liu: I have a lot of things I want to do. I’m eager to establish my own team. The overarching and the unifying theme of the new lab will be to develop the next generation of in vitro complex tissue models, or I call it assembloid tissue models. Assembloid basically means there’s a stem cell-based, three-dimensional complex tissue model that intentionally incorporates different cell types, to emulate the critical tissue-tissue interactions that determine the tissue- and organ-level functions. I still need to make a big decision where the lab could be; it could be in Canada and it could also be in China.

Q: What impact might tumors-on-a-chip have on the future of precision medicine?

Liu: It’s attracting a lot of attention from biologists and clinicians who are heavily focused on using the traditional tissue models—animal models, for example, or the simple dish cultures—for their studies of interest. So the biggest impact I can foresee with our technology is that now it’s more mature. I can see it being gradually, and maybe quickly, adapted into more traditional biological labs, to help them dissect the complex biological questions they’re asking, or to accelerate the evaluation of the exciting new drugs or therapies they’re developing. Overall, I can see that accelerate this development pipeline of new drugs and therapies in precision medicine.

 

Lindsey Leake is an award-winning, independent health reporter based outside Washington, D.C. She spent 15 years as a staff journalist at outlets including Fortune, the USA TODAY Network and Sinclair Broadcast Group. She holds an MA in Science Writing from Johns Hopkins University, an MA in Journalism and Digital Storytelling from American University and a BA from Princeton University.

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Top 5 Firms Engineering Healthcare in the CNS Space

Central nervous system (CNS) treatments are having a major comeback. These five precision medicine players plan to ride the resurgence.

After a decade of stagnation, the CNS space is seeing a revival in sales and R&D spending as the market was last year projected to surpass $80 billion for the first time since 2013 and hit around $127 billion.

Recent landmark approvals have brought attention back to the CNS, including the U.S. Food and Drug Administration (FDA)’s greenlight of Eisai/Biogen’s lecanemab (Leqembi) for the treatment of Alzheimer’s disease in 2023, and the FDA approval of Bristol-Myers Squibb’s schizophrenia treatment xanomeline/trospium chloride (Cobenfy) in 2024.

At the same time, Johnson & Johnson’s depression treatment, esketamine (Spravato), is on its way to blockbuster status, showcasing the growth potential of the CNS market.

These successes accompany an emerging shift in psychiatry clinical trials from subjective rating scales to more objective endpoints, including digital and physiological measures, with the potential to better tailor treatments to a patient’s biological makeup.

Startups and scaleups are attracting increasing investor attention for their potential to change the way we treat CNS conditions. Check out our list of the most exciting companies that have netted the biggest investor dollars.

 

1. Aerska

Founded: 2025 | Headquarters: Dublin, Ireland

Aerska logo

Aerska’s name is derived from an Irish proverb stating that people survive in each other’s shelter, emphasising the strength of its team.

This team includes co-founder Jack O’Meara, previously co-founder of the liver-focused RNA interference (RNAi) biotech Ochre Bio, who is driven by the experience of loved ones suffering from Alzheimer’s disease.

Aerska is developing RNAi therapies for neurodegenerative conditions, including Parkinson’s and Alzheimer’s disease.

While there are already FDA-approved RNAi therapies on the market, such as Alnylam’s patisiran (Onpattro), these are typically focused on liver and cardiometabolic conditions rather than the CNS.

Aerska’s technology consists of antibody “brain shuttles” that bind to proteins on the blood-brain barrier (BBB). They then carry a payload RNA into the brain.

The payload, which is designed based on data-driven patient stratification and disease biomarkers, then silences specific genes driving the disease.

Aerska has already raised $60 million since its launch, including a $21 million seed round in October 2025 and a $39 million Series A round in February 2026, co-led by EQT Life Sciences and age1.

The company, which has research operations in the U.K., is using the latest funding to drive its pipeline programs toward clinical testing.

 

2. Beacon Biosignals

Founded: 2019 | Headquarters: Boston, Massachusetts, U.S.

Beacon Biosignals logo

Beacon Biosignals was co-founded by a team including its CEO—MIT neuroscientist Jacob Donoghue, MD, PhD—and its CTO, the machine learning researcher Jarrett Revels.

Boasting more than 100 employees, the company’s goal is to provide objective biomarkers in drug development that neurology and psychiatry have traditionally lacked compared with other areas of precision medicine.

Its FDA-cleared Waveband device measures the brain’s activity, known as electroencephalography (EEG), while patients sleep at home. The EEG data is then stored, quality-controlled, and fed into AI models that can guide the design of clinical trials.

For example, Beacon’s EEG data can identify patients with Alzheimer’s disease who have worse outcomes and might need a more targeted treatment or a different clinical trial than other patients.

Beacon raised $27 million in a Series A round in 2021 and an oversubscribed Series B round worth $86 million in November 2025.

The B round, which included investors such as Innoviva, Google Ventures, and Nexus NeuroTech, will help the startup to accelerate the discovery of neurobiomarkers and broaden clinical adoption of the technology.

Beacon acquired the French sleep monitoring company Dreem in 2023 to access its monitoring data and headband technology. Beacon then acquired the Ohio-based CleveMed in April 2025 to harness technology measuring breathing, oxygen, and other signals.

 

3. Brainomix

Founded: 2010 | Headquarters: Oxford, U.K.

Brainomix logo

Brainomix was founded by a team including CEO Michalis Papadakis, PhD, who was scientific director of the preclinical stroke lab at the University of Oxford.

Brainomix is dedicated to speeding up patient care in cases of stroke, where speedy treatment is key.

Brainomix’s flagship product, Brainomix 360 Stroke, is designed to harness AI to interpret brain scans and detect blood clots in patients with stroke, speeding up clinical decision-making.

The product involves a group of tools that automatically analyze images, including results from computed tomography (CT), CT angiography, magnetic resonance imaging (MRI), and CT perfusion.

Brainomix’s technology doubled the rate of thrombectomy treatment in patients with stroke and reduced hospital triage and transfer delays, according to a 2025 study.

The University of Oxford spinout is at a commercial stage, with operations in more than 20 countries, and is expanding into the U.S.

Brainomix raised a $21.2 million Series B round in 2021 and extended its Series C round from $6.5 million in March 2025 to $25.4 million in February 2026, with leading investors including Parkwalk Advisors and Hostplus. The proceeds will fuel the company’s expansion into the U.S. market.

Brainomix has also partnered with heavyweights, including Nvidia, Boehringer Ingelheim, Medtronic, and GE Healthcare.

Brainomix also has a product dedicated to disease monitoring in pulmonary fibrosis.

 

4. Circular Genomics

Founded: 2021 | Headquarters: San Diego, California, U.S.

Circular Genomics Logo

Circular Genomics was spun out of the University of New Mexico, with its founders including CSO Nikolaos Mellios, PhD, and Alexander Hafez, PhD.

The company later moved its headquarters from Albuquerque to San Diego in March 2025 to access scientific and operational know-how from Eli Lilly at Lilly Gateway Labs.

Circular Genomics aims to equip medical professionals with a blood test to detect CNS conditions early, in addition to stratifying and guiding the treatment of patients.

Its technology involves using a polymerase chain reaction (PCR) test of a patient’s blood sample to screen for specific circular RNA molecules produced in the brain that can cross into the blood and be measured as a biomarker of disease in the CNS.

Commercially launched in 2024, Circular Genomics’ MindLight SSRI Antidepressant Response Test predicts whether a patient will benefit from common antidepressants called SSRIs with around 77% accuracy. This is designed to predict a patient’s most suitable antidepressants without needing months of trial-and-error approaches.

The company is applying its technology in Alzheimer’s disease, where the approvals of disease-modifying therapies such as Leqembi have led to demand for tests that can detect the disease at earlier stages than traditional tests.

Circular Genomics raised $15 million in a Mountain Group Partners-led Series A round in December 2025 to finance the development of its technology and expansion of its technology in Alzheimer’s disease.

The company also has its sights on other CNS conditions, including multiple sclerosis and Parkinson’s disease.

 

5. Omniscient Neurotechnology

Founded: 2019 | Headquarters: Sydney, Australia

o8t logo

Omniscient (o8t)’s founders include CMO Michael Sughrue, MD, a neurosurgeon aiming to improve anatomy maps for other surgeons, and machine learning expert Stephane Doyen, PhD.

o8t’s FDA-approved product Quicktome involves using a patient’s MRI brain scans and AI models to map out a patient’s brain circuitry. These maps, accessible from an electronic tablet, can guide surgery to minimize the risk of brain damage compared to using a generalized anatomical diagram.

Quicktome is already in use at major hospitals around the world, including major centers in the U.S. Its partners include U.S. surgical support firm META Dynamic and the U.S. medical device innovation center, The Jacobs Institute.

o8t has raised more than $60 million, and bagged $14 million (AUD 20 million) in January 2026 as part of a Series D round targeted to reach $25 million (AUD 36 million). The round was led by Australia’s National Reconstruction Fund (NRFC) and OIF Ventures, with the aim of keeping the company based in Australia.

The funding is earmarked to fuel the development and commercialization of Quicktome, and grow o8t’s Australian workforce by more than 40. The company also has operations in Atlanta, Georgia, U.S.

o8t also plans to expand the technology into high-growth markets, including brain computer interface targeting, stroke and traumatic brain injury.

 

Jonathan Smith, PhD, is a freelance science journalist based in the U.K. and Spain. He previously worked in Berlin as a reporter and news editor at Labiotech, a website covering the biotech industry. Prior to this, he completed a PhD in behavioral neurobiology at the University of Leicester and freelanced for the U.K. organizations Research Media and Society of Experimental Biology. He has also written for medwireNews, Biopharma Reporter, and Outsourcing Pharma.

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How an Alzheimer’s Risk Gene Rewires the Brain Decades Before Symptoms

For millions of people worldwide, carrying the APOE4 gene variant means a significantly higher risk of developing Alzheimer’s disease. Yet one of the biggest unanswered questions has been when, and how, that risk begins to take hold in the brain.

New research from the Gladstone Institutes, published in Nature Aging, suggests that the effects of APOE4 emerge far earlier than previously understood. The study shows that subtle but important changes in brain activity occur long before memory loss begins, offering a potential window for early intervention.

Early changes in a seemingly healthy brain

Alzheimer’s disease is typically diagnosed after cognitive symptoms appear, but growing evidence suggests that the disease process begins decades earlier. The new study adds to this picture by demonstrating that brain circuits in young individuals carrying APOE4 are already functioning differently.

We found fundamental changes in brain circuits occurring in young mice that still had normal learning and memory, and importantly, that those changes predicted the development of cognitive deficits at older ages, ” said Misha Zilberter, PhD, principal staff research scientist at Gladstone and senior author of the study.

The researchers observed increased neuronal activity in the hippocampus, a brain region essential for learning and memory. Similar patterns of hyperactivity have been reported in human APOE4 carriers, even before clinical symptoms arise.

According to the scientists, this suggests that Alzheimer’s risk is not simply a matter of late-stage degeneration, but may instead involve long-term changes in how brain circuits are wired and function.

Smaller neurons, stronger signals

To understand what drives this early hyperactivity, the team examined individual brain cells. They found that neurons in key regions of the hippocampus were physically smaller in APOE4 carriers compared to those with the more common, lower-risk APOE3 variant.

While this might seem like a minor structural difference, it has functional consequences. Smaller neurons are more easily activated, meaning they fire more readily in response to stimuli. This heightened sensitivity can lead to persistent hyperactivity within neural circuits.

Over time, this imbalance may place stress on the brain and contribute to the gradual decline seen in Alzheimer’s disease.

A surprising source of dysfunction

For years, researchers believed that APOE4’s effects were primarily driven by astrocytes, support cells in the brain that produce most of the APOE protein. However, the new findings challenge this assumption.

The team discovered that the disruptive effects on brain activity were instead linked to APOE4 produced directly by neurons themselves. When APOE4 was removed from neurons, their size and activity returned to normal. Removing it from astrocytes, by contrast, had little effect.

This shift in understanding refocuses attention on neurons as key drivers of early disease processes, rather than passive victims of surrounding dysfunction.

A reversible pathway—and a new target

Perhaps the most striking finding of the study is that these early changes may not be permanent.

The researchers identified a protein called Nell2 as a central player in the process. Levels of Nell2 were elevated in APOE4 neurons and appeared to drive both the reduction in cell size and the increase in neuronal activity.

By reducing Nell2 levels in adult mice, the team was able to restore normal neuron structure and function—even after the changes had already occurred.

“What’s exciting about Nell2 is that we were able to reverse the disease manifestations in adult mice by lowering its level,” said Yadong Huang, co-senior author of the study. “That tells us the damage is not irreversible […].”

This raises the possibility of developing therapies that target Nell2, potentially slowing or preventing disease progression in individuals at high genetic risk.

Implications for early intervention

APOE4 is present in roughly one in four people and in the majority of Alzheimer’s patients. Despite this, current treatments largely focus on late-stage symptoms rather than early prevention.

The new findings suggest that intervening earlier, before cognitive decline begins, could be key. If brain circuit changes can be detected and corrected at an early stage, it may be possible to delay or even prevent the onset of Alzheimer’s disease.

The study also highlights the importance of understanding how genetic risk translates into functional changes in the brain. Rather than acting as a simple risk marker, APOE4 appears to actively reshape neural activity over time.

A shift in perspective

More broadly, the work reflects a growing shift in Alzheimer’s research, from focusing solely on hallmark features such as amyloid plaques and tau tangles to examining earlier, subtler changes in brain function.

By identifying a concrete pathway linking genetic risk to altered brain activity, the study provides a clearer framework for understanding how the disease develops.

“This study is a big breakthrough for the field of Alzheimer’s research,” Huang said. “It opens the door to a better understanding of how APOE4 alters the function of neurons at a young age to increase risk of cognitive decline, and to the development of therapies that could block the detrimental effects of APOE4 early on.”

While the findings are based on mouse models, they align closely with observations in humans and offer a strong foundation for future research. The next steps will involve determining whether targeting Nell2 or similar pathways can produce similar benefits in human patients.

If successful, such approaches could transform how Alzheimer’s disease is treated, not as an inevitable consequence of aging, but as a process that can be detected early and potentially reversed.

The post How an Alzheimer’s Risk Gene Rewires the Brain Decades Before Symptoms appeared first on Inside Precision Medicine.

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The Unspoken Toll: Why Exam Pressure Must Be Part of the Youth Mental Health Discussion

A Conversation with Tatum Redmond and Amanda van der Vyver-Anderson from Community Keepers, South Africa

By Mai El Shoush, Partnerships Campaign Manager, Stavros Niarchos Foundation (SNF) Global Center for Child and Adolescent Mental Health at the Child Mind Institute

Community Keepers is an award-winning organization based in Stellenbosch, South Africa, which works to improve the social and emotional well-being of learners and their caregivers. The SNF Global Center at the Child Mind Institute works with the organization to further advance the comprehensive mission of transforming schools into safe spaces where student well-being is prioritized alongside academic achievement. This includes strengthening the workforce to expand evidence-based support and brief interventions through low-intensity psychological therapy approaches.

While addressing the workforce gaps, the partnership has yielded valuable insight into the essential competencies front line workers require to effectively support young people experiencing mental health challenges. Together with other NGOs, Community Keepers has also been instrumental in strengthening the process of developing context-sensitive and culturally appropriate training materials scheduled for pilot implementation in South Africa later this year – representing an important step towards strengthening mental health care systems for underserved communities. The partnership also extends beyond training development, as the SNF Global Center at the Child Mind Institute continues to collaborate closely with Community Keepers on an upcoming randomized control trial (RCT). The scientific evaluation will assess both the feasibility of establishing a virtual clinic for young people and the effectiveness of remotely delivered cognitive behavioral therapy (CBT) interventions via video consultations. The research is intended to expand access to equitable and quality mental health care for young people across South Africa. Tatum Redmond has been a care facilitator in one of the Community Keepers’ high school-based offices, while Amanda van der Vyver-Anderson is an educational psychologist and heads the training and development of Mental Health First Aiders for internal and external staff.

Amanda van der Vyver-Anderson

How important is it to approach issues such as academic pressure within the wider conversation around youth mental health in South Africa, and beyond?

It is critical to integrate discussions of exam stress into the broader dialogue surrounding youth mental health, both here in South Africa and internationally. We see countless students under immense pressure to not only pass, but also secure their future prospects and meet family expectations. This is unfortunately often dismissed as “just school” or a “normal” experience. However, it impacts a substantial number of young people, often more severely than we acknowledge. And the level of support available is not equitable across the board. Addressing this is crucial because of the detrimental effects on core cognitive functions — and ultimately, academic performance — as well as the significant toll on mental health. This can manifest as anxiety, burnout, and even depression.

In what ways can exam-related stress connect to broader mental health challenges?

While a certain level of stress can serve as a beneficial motivator, severe distress can lead to cognitive shutdown. This specifically impacts the executive functions — planning, organizing, prioritizing, working memory, focus, and concentration — that are fundamental to preparing for exams. This shutdown can then create a detrimental, ongoing cycle of heightened stress about exams or the future, coupled with a decline in the ability to take effective action.

It’s vital to recognize that exam stress does not merely stay in the exam room — it can be a gateway to larger mental health challenges. Constant stress regarding school performance, marks, or the fear of failure can escalate into conditions like anxiety, chronic overwhelm, or depression. Students may experience sleep disruption, poor nutrition, and feelings of inadequacy. And these symptoms often persist long after the test is over. Compounding this is the reluctance of most students to seek help because they believe their feelings are normal or fear appearing weak. Yet, if left unaddressed, sustained pressure along with these symptoms can profoundly affect their psychological well-being.

Tatum Redmond

What role do community-focused organizations such as Community Keepers play in linking academic stress to systematic youth mental health support and improvement?

Organizations like Community Keepers play a truly pivotal role — not merely as emergency responders but as an integrated support system within educational institutions as well. Crucially, they move beyond immediate crisis response by collaborating with schools to develop long-term support and to provide safe spaces to engage in dialogue. They offer genuine attention and care when learners are struggling with school demands, exams, and family pressures.

The approach is not just “addressing stress today” but asking, “How can we create an enduring environment where young people feel safe, supported, and connected?” Doing this requires collaboration with the learners themselves, educators and school staff, as well as parents, caregivers, and community leaders.

What factors make schools uniquely positioned to be safe and supportive spaces?
Schools are exceptionally well-positioned to serve as safe and supportive spaces for students for several key reasons:

  • Learners spend a substantial portion of their day at school, making it a primary setting where adults can observe signs of distress, anxiety, or coping difficulties.
  • Schools have the opportunity to house critical personnel — teachers, counselors, and external partners like Community Keepers — who are on hand to offer support or a listening ear.
  • The curriculum can extend beyond academic skills and learning. It can include mental health and emotional literacy, stress management, and peer support.
  • When a school actively fosters an environment of safety, respect, and validation, it fundamentally alters how learners navigate pressure, stress, or complex personal problems. Having a guaranteed safe space at school is deeply stabilizing for the mind.

How can the goal of securing mental health support as a pillar of education be reached?
Achieving the goal of establishing mental health support as a solid, non-negotiable pillar of education requires several strategic commitments:

  • Schools must actively allocate resources for it, ensuring adequate numbers of support staff, rather than relying on minimal provision. Teachers need training to recognize signs of distress and respond helpfully and appropriately.
  • Mental health literacy must be integrated into the curriculum. Instead of only focusing on academic subjects, topics like stress management, emotional intelligence, and maintaining healthy relationships should be covered.
  • The government must demonstrate a serious commitment, including mental health support in education budgets, developing clear policies, and ensuring rigorous follow-through.

How have your practices and initiatives in promoting and supporting schools as safe spaces made meaningful change?
We’ve observed tangible change in the learners’ attitudes; those who feel comfortable expressing their emotions are generally happier and more resilient because they have established a safe, non-judgmental space where trust is built.

What role can teachers and school leadership play as partners in creating an evidence-based supportive learning environment? Where are the gaps in building capacity and how can they be better supported?
Educators and school leadership are essential partners in establishing an environment that successfully supports learner mental health and cultivates a culture of well-being. They can do so by:

  • Prioritizing both the physical space and curriculum time necessary for learners to engage with support services.
  • Serving as role models who embody and encourage emotional regulation and actively normalize help-seeking behaviour.
  • Remaining deeply cognisant of factors that contribute to learner distress so as to not inadvertently exacerbate it.

Investing in staff wellness and support, capacity building, and policy reform is not merely beneficial, but a foundational requirement to capacitate educators effectively. This allows them to sustainably support the mental health of their entire school community.

The SNF Global Center’s work in South Africa is carried out through the Child and Adolescent Mental Health Initiative (CAMHI South Africa). We are proud to expand the partnership with Community Keepers and value their collaboration towards co-creating scalable, school-centered mental health approaches that authentically respond to the diverse lived-experiences of young people.

The post The Unspoken Toll: Why Exam Pressure Must Be Part of the Youth Mental Health Discussion appeared first on Child Mind Institute.

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What The Atlantic Missed About OCD: There Is Hope

Dear Editor,

The Atlantic’s recent article, “When Mentally Ill Teenagers Ask to Be Put to Death,” brings needed attention to the profound and often misunderstood suffering caused by obsessive compulsive disorder (OCD). As the story illustrates, OCD can be severe, chronic, and life-threatening — so much so that individuals with OCD are at significantly elevated risk of suicide.

However, what is missing from this important conversation is an equally critical truth: effective, evidence-based treatments for OCD exist. Unfortunately, even after years in care, up to 98% of people never receive the many first, second, and third-line approaches that are available due to systemic breakdowns in proper screening, diagnosis, referral, and access to specialized care.

With appropriate care, many individuals who once felt trapped by their symptoms are able to reclaim their lives, pursue their goals, and thrive. At the International OCD Foundation, we regularly hear from people affected by OCD who have gone from a place of despair to one of hope because they were able to access evidence-based treatment, specifically Exposure and Response Prevention therapy (ERP). 

It is essential to raise awareness and acknowledge the devastating impact OCD can have. But it is equally important that people know there is hope. The tragic outcomes outlined in “When Mentally Ill Teenagers Ask to be Put To Death” are not inevitable. With continued efforts on raising accurate awareness of OCD and strengthening our systems of care around the globe, more people with OCD can access the effective, life-saving treatment they deserve.

For those seeking help, resources and treatment guidance are available at iocdf.org.

The post What The Atlantic Missed About OCD: There Is Hope appeared first on International OCD Foundation.

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Research on the Development, Implementation Effect and Neural Mechanism of a Physical Intervention Program for College Students With ADHD Based on the Characteristics of Balance Dysfunction

Conditions: ADHD – Attention Deficit Disorder With Hyperactivity; ADHD; ADHD – Combined Type; ADHD – Inattentive Type; ADHD Specifically With Executive Function Impairment

Interventions: Behavioral: Aerobic Treadmill Training; Behavioral: Progressive Balance Training; Behavioral: Cognitive-Balance Dual-Task Training

Sponsors: Wuhan Sports University

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