Tag: United States

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The Download: Anthropic launches Claude Science, and California’s carbon manure math

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.

Claude Science is Anthropic’s newest flagship product

At an event for pharmaceutical executives, biotech founders, and researchers yesterday, Anthropic announced Claude Science, a major new product intended to support scientific research like Claude Code supports software engineering.

Like Claude Code, Claude Science can autonomously carry out meaningful work from concise, high-level instructions, with tools for computational biology and drug development. The launch signals that Anthropic is doubling down on AI for science, and the company will also use the product in its own research into drugs for rare, neglected diseases.

Discover why Anthropic is betting big on AI for scientific research.

—Grace Huckins

Why California’s carbon manure math doesn’t add up

Something stinks in California’s climate policies. 

Years ago, the state set up a system that pays cattle farmers to turn the methane emitted from cattle manure into natural gas. It’s become wildly popular because the subsidies are extremely lucrative. But research suggests the program exposes the shortcomings of carbon offsetting and trading schemes.

Instead of forcing industries to directly cut their pollution or pay for it as a cost of doing business, legislators have opted for incentives that swap climate responsibilities between parties and regions. The system could ultimately lock in more warming.

Read the full story on California’s dubious carbon calculations.

—James Temple

This story is from The Spark, our weekly climate tech newsletter. Sign up to receive it in your inbox every Wednesday.

Watch now: longevity’s next frontier—“reprogramming” your body

Billions of dollars are pouring into efforts to reverse aging as scientists investigate ways to return cells to a younger state. But how close are these experimental treatments? And are they likely to work? 

At a recent virtual Roundtables event, MIT Technology Review explored the answers with science editor Mary Beth Griggs and senior biotechnology reporter Jessica Hamzelou. Subscribers can now watch the full recording of the fascinating discussion.

MIT Technology Review Narrated: the search for dark matter has been blown wide open

For decades, physicists have hunted for weakly interacting massive particles (WIMPs), a leading candidate for dark matter. But their search has run into a new problem: neutrinos. 

These tiny particles from the sun and other stars can create a “neutrino fog” that drowns out any signal of dark matter. Hitting the neutrino fog does not, however, mean an end to the search. Researchers just have to shift the focus of their hunt.

They’re now casting a much wider net. New proposals include quantum sensors, liquid-helium detectors, and even searches in Jupiter’s atmosphere.

—Dan Garisto


This is our latest story to be turned into an MIT Technology Review Narrated podcast, which we publish each week on Spotify and Apple Podcasts. Just navigate to MIT Technology Review Narrated on either platform, and follow us to get all our new content as it’s released.

The must-reads

I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.

1 The US has lifted restrictions on Anthropic’s Mythos and Fable models
Anthropic said it would begin restoring access today. (NYT $)
+ The US had imposed controls over security concerns. (Bloomberg $)
+ It lifted the restrictions after lengthy talks with Anthropic. (BBC)
+ But the crackdown has already opened doors for Chinese AI rivals. (CNBC)

2 The most detailed survey of the universe ever is now underway
It’s using the largest digital camera on Earth. (New Scientist $) 
+ The project is based at the Vera C. Rubin Observatory in Chile. (NYT $)
+ It aims to transform our view of the cosmos. (MIT Technology Review)
 
3 Tech talent is fleeing the US due to H1-B visa chaos
They’re eyeing relocation to Canada, the UK, or the Gulf. (Rest of World)
+ While China is poaching AI talent from the US. (CNBC)
+ Visa rules are also affecting young scientists. (MIT Technology Review)
 
4 Trump raked in more than $1 billion from crypto businesses in 2025
He reported $635 million in royalties from a Trump meme coin. (BBC)
+ The rest largely came from his World Liberty Financial venture. (The Hill)
 
5 The UN warns that the rapid spread of AI may worsen global inequality
It’s proposed a shared framework for responsible AI development. (Guardian)

6 Companies are making LLMs talk like a caveman to curb AI spending
A senior OpenAI employee contributed to the “caveman” project. (404 Media)
 
7 Babies are born with the neural foundations for math
Brain recordings have identified the mechanisms. (New Scientist $)

8 An independent studio has bought the OpenAI movie Amazon dropped
Neon has purchased “Artificial,” which focuses on Sam Altman. (NYT $)
+ Amazon had dumped it after investing in OpenAI. (Gizmodo)
+ The depiction of Altman is reportedly unsympathetic. (Variety)

9 AI has re-created Gene Wilder’s voice for a new “Willy Wonka” series
Wilder’s wife said his estate is “delighted” with the new show. (NBC News)
+ Netflix partnered with AI company ElevenLabs on the project. (The Verge)

10 NASA aims to send a spare Mars rover—and soccer ball—to the moon
The nuclear-powered “Promise” may help establish a lunar base. (NYT $)

Quote of the day

“Caveman save you token, save you money.” 

—The GitHub repository for the “caveman” plugin explains how the project curbs AI spending by turning verbose LLM outputs into concise text.

One More Thing

white pill tablet with a meter etched onto the surface

SELMAN DESIGN


AI is dreaming up drugs that no one has ever seen. Now we’ve got to see if they work.

On average, it takes more than 10 years and billions of dollars to develop a new drug. A growing number of startups are betting that AI can make the process faster and cheaper. 

By predicting how potential drugs might behave in the body and discarding dead-end compounds before they leave the computer, machine-learning models can cut down on the need for painstaking lab work. 

Yet it is still early days for AI drug discovery. A lot of AI companies are making claims they can’t back up—and the technology is not a panacea. But the technology is beginning to move from promise to practice.

Find out how AI is speeding up drug discovery.

—Will Douglas Heaven

We can still have nice things

A place for comfort, fun, and distraction to brighten up your day. (Got any ideas? Drop me a line.)

+ Explore the surprisingly diverse world of regional dartboards from across the UK.
+ Judge a book’s beauty by its cover with this collection of the best designs of the last decade.
+ This John Wick parody with almost no dialogue understands what audiences really came for.
+ Focus your mind or unwind with over 80 custom albums of ambient instrumental electronic music on Caught In Joy.

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Agriculture is ready for AI, but its data isn’t

Artificial intelligence is transforming what is possible in agriculture, but industry leaders should be wary of investing in AI without first laying the groundwork. 

The use cases are promising, especially for an industry navigating volatile fertilizer costs, unpredictable weather, and margins that leave little room for error. Research shows AI-enabled predictive models can improve crop yield by 26%, reduce water use by 41%, and cut chemical usage by 33%. 

However, what AI vendors usually won’t tell you is that these solutions are only effective if you have a clean, solid data foundation. However, at Reltio, we have experience in this area, including leading technology strategy at a major agricultural distributor and building a data platform used by enterprises worldwide–we’ve seen it first hand.

What AI vendors won’t tell you 

Vendor conversations in agriculture tend to follow a familiar pattern. The pitch leads with grand promises around using AI to monitor crop health in real time, optimize irrigation, and squeeze more yield from every acre. 

The promise is compelling, but what rarely comes up is the question of whether the data foundation underneath those promises is accurate and complete. If not, there is a real and significant risk that AI will generate misleading outputs that seem authoritative but inspire action that is, at best, counterproductive. 

For instance, a yield prediction model fed inconsistent historical data will generate imprecise forecasts. Similarly, a precision irrigation system drawing on fragmented sensor data will make watering decisions that waste resources instead of saving them. 

In each case, the AI is failing because the data it was trained on was not sufficient to produce trustworthy outputs. In agriculture, every AI hallucination is a liability, and the likelihood of error is high.

Why agriculture is a uniquely challenging test case

The data landscape across a modern agricultural operation or a large distributor serving thousands of growers is extraordinarily complex.

Modern farming environments make extensive use of IoT devices and machinery. Irrigation systems are automated, tractors navigate fields autonomously, and drones capture field imagery at scale. 

However, machine data is disparate by nature. Add in external sources, including weather feeds, U.S. Department of Agriculture data, and third-party market information, and the question of how you bring all of it together into something coherent becomes a significant undertaking. 

Agricultural AI also needs to understand more than just customer attributes; it needs to understand the land: GPS coordinates, farm boundaries, field blocks, and soil variation across a single property. Where do you apply fertilizer, and at what rate, and in which specific area of the farm? Not all parts of a field are the same, and an AI system that treats them as if they are will produce recommendations that are at best imprecise and at worst damaging.

There is also a compliance dimension due to the chemicals and the responsibility involved. Operational AI in agriculture needs significantly more checks and governance than it might in a lower-stakes environment. When a flawed recommendation gets acted upon in the field, the consequences can be severe. 

What data readiness means in practice 

Data readiness is the difference between AI delivering on its promise vs. a “garbage in, garbage out” scenario. Fundamentally, being ready for AI means having a data model that accurately reflects how the business operates. 

For a company like Wilbur-Ellis, a 104-year-old, family-owned agricultural distributor, that means understanding who your customers are, which fields they farm, which inputs they need, which suppliers those inputs come from, what they paid last season, and how all of that connects to margin. That information needs to be current, consistent, and accessible across the organization, rather than locked in separate systems that were never designed to talk to each other.

Similarly, for farming operations themselves, data readiness means having a reliable, connected picture of what is happening across every field: soil health records, input application histories, yield data from previous seasons, equipment performance, and real-time sensor readings from irrigation systems.

Governance matters just as much as structure. Prices change, relationships evolve, and suppliers come and go. An AI system drawing on data that was accurate six months ago but has not been maintained will make recommendations based on a version of the business that no longer exists. 

Building the foundation that makes AI trustworthy

The good news is that the path to data readiness is feasible. It starts with a strong data model: a single, governed source of truth that connects customers, suppliers, products, pricing, orders, and margins in a way that reflects how the organization operates. 

From there, it requires data pipelines fast enough to deliver insights when decisions need to be made, governance frameworks that keep that data trustworthy over time, and security controls that ensure sensitive commercial information is accessible to the right people under the right conditions.

This is precisely the challenge that Reltio, an SAP company, was built to solve. Reltio enables companies to unify their fragmented data so AI agents and systems can operate from a complete picture of the business. Reltio builds a trusted system of context, known as the context intelligence layer, that brings all entities, relationships, rules together under one roof and makes business data easy to access and interpret.

For Wilbur-Ellis, building that trustworthy data foundation has meant being able to ask more complex questions and trust the answers, which is the precondition for any AI system to be genuinely useful.

How agriculture can drive real value from AI

The question worth asking before the next AI conversation is not whether the use case is promising. It almost certainly is. The question is whether the underlying data foundation is strong enough to make the output trustworthy. 

Agriculture has always required its leaders to make high-stakes decisions under uncertainty, and AI offers the genuine prospect of making those decisions faster and better informed. That prospect is only achievable for organizations that have done the foundational work first, and the businesses that will get the most from AI are the ones investing in that foundation now.

This content was produced by Reltio. It was not written by MIT Technology Review’s editorial staff.

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Cytiva Completes Doubling of Utah Site’s Liquid Media Production Capacity

Cytiva has completed an expansion of its Logan, UT, facility that effectively doubles its liquid media production capacity, a project designed to support supply chain continuity for customers relying on the company for their cell culture needs.

The company has completed its animal-derived component-free (ADCF) liquid media expansion facility (A1X), Pierre-Alain Ruffieux, Cytiva group executive, bioprocess, told GEN in an interview conducted from the company’s booth during the Biotechnology Innovation Organization (BIO) International Convention recently held in San Diego. He said the completion was celebrated with a ceremony on the site.

Cytiva detailed the expansion project in a May 12 post on its website: The ADCF liquid media expansion facility (A1X) has larger mixing tanks than the existing facility, supporting batch sizes from 700 L up to 13,000 L—compared with batch sizes of 100 L to 10,000 L supported by Cytiva’s existing facility.

Also, the A1X facility uses mixing tanks and liquid media transfer lines comprised of AL6XN and 316 L stainless steel. This differs from the existing facility equipment, which is comprised solely of 316 L stainless steel. AL6XN is a low-carbon, high-purity stainless-steel alloy that is more resistant to wear and corrosion than 316 L, representing an upgrade to the product contact layer versus the existing facility equipment.

The expanded site’s added liquid capacity comes from the addition of three manifold fill lines, three filling manifolds, six mixing tanks, six formulation booths, and a utility building to support large volume liquid media production. Housed in the utility building are a 45,000 L tank and process water system, a 55,000 L tank and water for injection system, a clean steam generator, and additional supporting utilities.

“In addition to the added capacity, Cytiva has updated several aspects of the manufacturing floor layout and equipment, improvements designed to shorten production cycle time, improve safety, and minimize product risk,” the company explained. “The updates also establish closed systems for cleaning and a controlled environment for the transport and handling of raw materials and finished goods.”

Previously, Cytiva completed expanding its dry powder and liquid media manufacturing capacity for large-volume customers and added high-speed bottle filling for smaller-volume users. The company also opened an expanded staging area for finished goods, as well as a new centralized 10,000-square-foot quality control lab to support increased manufacturing.

AI’s “two major impacts”

Pierre-Alain Ruffieux, Cytiva group executive, bioprocess

During a wide-ranging interview, Ruffieux discussed Cytiva’s approach to AI and several recent Cytiva announcements.

“We see two major impacts from AI on what we are doing,” Ruffieux explained. “The first one, and I always like to start with the customers because it’s really our focus: We see our customers accelerating and increasing the number of targets they are doing. AI is helping them to have more targets and in a faster time,” Ruffieux said. “It’s putting pressure on the CMC folks, and I think it’s where we play: They ask us to provide innovative solutions to go faster.”

Cytiva’s focus on AI is two-fold, he continued.

“One, we are developing intelligent equipment which is using AI to be easier for customers to use and which are more functional; that is one aspect. It’s also delivering more experience in a shorter time frame,” Ruffieux said. “It’s a kind of next level of DoE [design of experiments], but it’s also delivering a productivity aspect because the goal is to have equipment which requires either fewer people or fewer people with less specific knowledge of the equipment.”

Like a growing number of companies in and outside biopharma, Ruffieux said, Cytiva has fully embraced AI “to make our product better, to make the customer experience better, but also to improve our internal processes.”

“Faster and better”

“We see AI helping us to develop software, writing new software to go faster and better. AI is very powerful for reviewing documents and doing things,” he explained. “It’s amazing what we can do both in writing code, but also perhaps as importantly, as we validate the code and we test everything, the use of AI is allowing our people to work in a much more comprehensive way, in a much faster way.”

AI also adds a layer, he said, to the continuous improvement ethos that Cytiva and other Danaher-owned companies practice through the Danaher Business System (DBS). Since the mid-1980s, Danaher has carried out an ongoing company-wide Kaizen or continuous improvement effort based on lean manufacturing and anchored on DBS, a common culture and operating system focused on people, plans, processes, and performance.

“AI is an additional pillar to this system, really helping the company to be more efficient and to drive business,” Ruffieux said.

Cytiva’s customers, he continued, have not specifically asked about AI. So what are customers telling the company that they want?

“What customers want is Cytiva delivering solutions which help them to innovate, produce drugs, and accelerate these processes. And AI is one of the attributes, but they don’t have a specific task on AI,” Ruffieux replied. “In discussing with senior customers, people are interested in the outcome, not in the product itself. So it’s not AI for AI, it’s AI for a business outcome. And in life science, the business outcome is quality. It’s reliability. It’s speed. It’s customers asking, can we help them to be better?”

AMT designation

Last month, Cytiva hailed the FDA’s granting its Advanced Manufacturing Technology (AMT) designation to the company for its Elevecta™ transient cell line for adeno-associated virus (AAV) manufacturing, one of the first gene therapy manufacturing technologies to receive the designation. Customers using the Elevecta transient cell line will benefit, according to Cytiva, from a clear, predictable regulatory and quality framework for gene therapy development.

Through its AMT designation, the FDA recognizes drug manufacturing technologies that it deems to have elevated the reliability, quality, and robustness of advanced therapeutics manufacturing. By enabling a streamlined Chemistry, Manufacturing, and Controls (CMC) review and frequent communication with the FDA, designees count on the AMT designation to help accelerate their manufacturing-related development timelines and create a meaningful advantage through faster time to market.

“This recognition by the FDA is giving confidence and trust for our customers: If they use this cell line to produce AAV, they know that the agency has seen the technical advantage and it’s confidence on the regulatory pathway,” Ruffieux said. “This recognition by that regulatory body is giving trust to the work of the company in helping customers develop drugs, which is really where we position ourselves as true partners.”

Elevecta is designed to significantly reduce the formation and encapsidation of host cell DNA (hcDNA).

“What is beautiful with that is, we get a reduction of 99% of the host cell DNA. You don’t have to worry any more about the host cell DNA which is coming with your product. Again, that is a huge advantage for the customer using that,” Ruffieux said. “This is the kind of innovation we are really proud to bring to our customers.”

Operating from hubs in Marlborough, MA, Amersham, U.K., Uppsala, Sweden, and Shanghai, Cytiva is a unit of Danaher that was re-launched in 2020 after Danaher spent $21.4 billion for the former biopharma business of GE Healthcare Life Sciences. Danaher oversees a global family of more than 20 operating companies focused on biotech and life sciences, as well as diagnostics, water quality, and product identification.

Bringing “the entire workflow”

Earlier this month, the company said that eight of its 2,000 L single-use Xcellerex bioreactors were among equipment contained in the new GMP-2 manufacturing facility inaugurated in Wuhan, China, by Chime Biologics, a decade-long customer that has used equipment made by Cytiva and its predecessor company.

“I want to put that in a larger context: At Cytiva, we really bring to the customers the entire workflow, which is really exciting for small to mid-sized customers. Coming to us, they really get a full facility that is working, really, from A−Z,” Ruffieux said. “It’s starting from an expansion of the cell line, to freezing the drug substance. It’s about a fully integrated solution that helps the customer to have that. And we have multiple facilities like that, that we are building every year for customers across the world.”

“We make significant investments to be able to supply our customers with what they need into different regions, in-region-for-region,” Ruffieux said.

In-region-for-region refers to Cytiva’s ongoing effort to satisfy customer demand for manufacturing tools and services usable within their regions of the world.

“This is really helping us and the customer to secure supply independent of any disruption,” he added. “Since COVID-19, we have seen multiple disruptions worldwide. And really, our original presence is giving confidence to customers that they will get what they need, independent of whatever crisis is happening across the world.”

Worldwide, the United States and European Union have championed “reshoring” efforts by drug developers and tools/technology providers across biopharma to manufacture more of their products within their regions rather than in China or elsewhere in Asia.

“When there is investment, it’s definitely always a tailwind,” Ruffieux said. “We welcome investment, and we are happy to support all customers to put up new facilities, and for the opportunity these facilities offer to position our equipment.”

The post Cytiva Completes Doubling of Utah Site’s Liquid Media Production Capacity appeared first on GEN – Genetic Engineering and Biotechnology News.

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Expedited Transition to Digital Delivery of Recovery Support Services Due to the COVID-19 Pandemic: Mixed Methods Needs Assessment

Background: Recovery support services (RSS) are an evidence-based approach to support recovery from substance use disorders, most often composed of peer-to-peer support, referrals to housing, job training, and other forms of prosocial engagement and activities. During the COVID-19 pandemic, RSS providers quickly converted in-person services to digital delivery to avoid disruption. It is unclear if this rapid conversion impacted the delivery of services or if this delivery model could enhance RSS reach and uptake more generally by extending the reach of RSS providers and offering an alternative delivery method and access point. Objective: The goal of this study was to identify how RSS providers in Texas adapted their services for digital delivery and to what extent, if at all, technology limitations (eg, lack of digital infrastructure) were present. Methods: We conducted an electronic survey of 85 RSS providers, assessing their current capacity and methods for the digital recovery support service (D-RSS), followed by semistructured online interviews with a subset of 20 respondents. Results: Most survey respondents (74/85, 87.1%) used D-RSS, though they used many dated technologies, devices, and platforms for service delivery. Many respondents indicated that they use Zoom (Zoom Video Communications) videoconferencing to communicate with participants; however, providers also indicated that they must use several different technology platforms to accomplish their service delivery goals. Four main themes emerged from the interviews: (1) the impact of the COVID-19 pandemic on RSS, (2) barriers and facilitators to technology-delivered D-RSS, (3) awareness and expectations regarding the use of D-RSS, and (4) training needs to deliver D-RSS. Conclusions: RSS organizations have access to technology for D-RSS; however, the technology is often outdated. Because the pandemic required a rapid and unexpected shift to D-RSS to maintain and potentially expand access during a public health emergency, providers desire guidance for training staff and participants on how to best use technology. A subset of providers endorsed the potential of a unified platform for D-RSS delivery, especially for data capture. Most barriers to D-RSS identified by our respondents may be addressable through the streamlined deployment of technology resources, rigorous training and onboarding programs in best practices for providers and participants, and tailored implementation strategies for varying local contexts.
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/3a2240fcd24e97753a368fb6d403d6fb" />

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Institutional Member Updates: Summer 2026

Institutional Members are clinics and programs in the US and around the globe that offer residential and/or intensive treatment for OCD and related disorders, are specialty outpatient clinics with a large staff dedicated to treating OCD, or provide low-cost treatment options through research studies.

Below are quarterly updates from our Institutional Members organized alphabetically. Click the (+) to open each menu and read updates and find contact information for clinics near you:

Do you work at a residential program, intensive outpatient program (ITP), or specialty outpatient clinic and looking to advertise your services? Learn more about becoming an Institutional Member and having your program updates included below!

Anxiety Experts – Santa Barbara, CA

Anxiety Institute – Greenwich, Connecticut

Anxiety Specialists of Atlanta

Atlanta CBT – Atlanta, Georgia

Bull City Anxiety & OCD Treatment Center – Chapel Hill, North Carolina

Cambridge Psychology Group – Camrbidge, Massachusetts

Cascade Programs – Midway, Utah

Center for OCD & Anxiety-Related Disorders

Center for OCD and Anxiety

Center for OCD and Related Disorders at Massachusetts General Hospital

Compassionate Healing Institute

Hopewell Therapeutic Community

Manhattan Center for CBT

Moment, Center for Anxiety Disorders – South Carolina

Nashville OCD & Anxiety Treatment Center – Nashville, Tennessee

Northwell Health OCD Center – Glen Oaks, New York

OCD Anxiety Centers

OCD Institute of Texas – Houston, Texas

OCD North

OCD Wellness

Palo Alto Therapy – Palo Alto, California

Polaris Family Behavioral Health

Psychology Houston, PC: The Center for Cognitive Behavioral Treatment

SkinPick (Online Treatment for Skin Picking)

Specialists in OCD & Anxiety Recovery (SOAR), PLLC

NOCD – Teletherapy Only

Stanford OCD Research Lab – Palo Alto, CA

StopOCD (Online Therapy for OCD)

Stress and Anxiety Services of NJ – East Brunswick, New Jersey

The Anxiety Center

The Anxiety Treatment Center of Greater Chicago

The Center for OCD and Anxiety at Sheppard Pratt

The Concord Center – Concord, Massachusetts

TrichStop (Online Treatment for Hair-Pulling)

UPMC Western Behavioral Health

The post Institutional Member Updates: Summer 2026 appeared first on International OCD Foundation.

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Claude Science is Here, Antibiotics Designed by Text Prompt Among Applications

Anthropic has released Claude Science, an AI workbench for scientists that consolidates fragmented research tools, including over 60 scientific databases and connectors pre-configured for genomics, proteomics, structural biology, and more, into a single reasoning layer. The platform joins an increasingly crowded ecosystem of tech platforms specialized for biology and aims to accelerate scientific discovery by making domain expertise more accessible.

Anthropic’s life science partners are delivering applications. Basecamp Research is targeting global public health, where drug-resistant infections play a role in nearly five million deaths per year. The London-based team has announced that its antibiotic design and vaccine target prediction EDEN models will now be available through Claude Science.

A metagenomic foundation model, EDEN demonstrated a 97% success rate when designing functional peptides with high potency against World Health Organization (WHO) critical-priority and multidrug-resistant pathogens. The work was done in collaboration with César de la Fuente, PhD, presidential associate professor at the University of Pennsylvania.

In a Claude Science demo, Oliver Vince, PhD, co-founder at Basecamp, uploaded a sample patient microbiology report. When given a simple natural language prompt, the platform designed peptides, predicted their efficacy, and provided a shortlist of candidates most likely to succeed in experiments in minutes.

While generating human-ready antibiotics at the click of a button is still a step away, Vince said democratizing these tools is a powerful first step, particularly for researchers in regions where accelerated computing infrastructure is not readily accessible.

“Most models require you to be a computational scientist,” Vince told GEN Edge. “Now, potentially any clinician in the world can chat with Claude and design an antibiotic that may work.”

“From a strategic perspective, you want the people with the most agency to solve the problem,” added Phil Lorenz, PhD, CTO at Basecamp. “Not the model builders who are two or three steps removed.”

Full stack

Founded in 2019, Basecamp has spent its initial years building a full computational stack spanning data, models, and therapeutic assets.

In addition to antibiotics and vaccines, the company’s U.S. office, based in Cambridge and led by Jonathan Finn, PhD, Basecamp CSO and former CSO of Tome Biosciences, has fine-tuned EDEN for programmable gene insertion. The approach places large therapeutic DNA sequences at precise locations in the human genome, expanding upon CRISPR-based approaches that use small edits to address a limited number of indications.

EDEN’s generalizability is enabled by training on BaseData, the company’s proprietary dataset composed of 9.8 billion protein sequences collected over 200 diverse and extreme locations, including thermal springs, polar ice, and high-altitude plateaus, across more than 30 countries. The database provides a 10-fold expansion of known protein diversity when compared to all public databases combined.

In March, the team published the compounding advantages of BaseData on model performance in a technical report on scaling laws for metagenomics. Basecamp is steadily pushing forward that data diversity through the Trillion Gene Atlas, a partnership with Anthropic, NVIDIA, PacBio, and Ultima Genomics that aims to scale BaseData 100-fold over the next two years.

Vince emphasizes that model deployment and integration into real-world workflows will be critical for these models to reach their full potential. Basecamp anticipates releasing more applications over the next year.

“I think it will surprise people what these models can do,” he said.

The post Claude Science is Here, Antibiotics Designed by Text Prompt Among Applications appeared first on GEN – Genetic Engineering and Biotechnology News.

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iPSC-Derived Retinal Endothelial Cells Offer Platform for Studying Diseases

Biomedical engineers at Duke University have for the first time used induced pluripotent stem cells (iPSCs) to grow specialized blood vessel cells critical to retinal health.

When injected into mouse models of retinal disease, these “retinal endothelial cells” (iRECs) integrated into the damaged tissue to regenerate blood vessels and restore retinal function. The team also demonstrated these cells’ ability to form functional retinal vascular tissue in a lab-grown environment, providing a pathway to model and research various eye diseases.

The results point toward the potential of using these retinal cells and models to develop new methods of impactful vision loss treatments and eye disorder research. “Retinal vascular diseases affect millions of people in the U.S., but our understanding remains limited, hindering our ability to discover and develop new therapeutics,” said Sharon Gerecht, PhD, the Paul M. Gross Distinguished professor and chair of Biomedical Engineering at Duke. “Using human stem cells, we generated the cells found in retinal blood vessels, paving the way for new therapeutic approaches.”

Gerecht is senior and corresponding author of the researchers’ published paper in Nature Biomedical Engineering, titled “Derivation of functional retinal endothelial cells from human pluripotent stem cells for therapeutics and modeling.” In their report the authors suggested that their iREC differentiation strategy will “… advance cell therapy and disease modeling, accelerating the discovery of treatments for retinal microvascular diseases.”

The old saying that the eyes are windows into the soul is more accurate than one might think. Neurons from the retina—the back part of the eye that detects light—extend directly to the brain, technically making the eyes part of the central nervous system.

Also like the brain, the retina has a blood barrier that strictly controls what gets in and out including oxygen, nutrients, water and pharmaceuticals. While this barrier keeps the retina healthy and relatively protected from disease-causing agents, it also makes treating the retina difficult. “Retinal tissue has the highest energy and oxygen usage in the body due to the retina’s intense and continuous neuronal activity,” the authors further explained. “This demand leads to a crucial reliance on the inner blood–retina barrier (iBRB) to maintain ocular homeostasis.”

The barrier is formed by blood vessel tissue comprising a tight network of retinal endothelial cells, which form the inner layer of blood vessels, in concert with other specialized cells called pericytes and astrocytes. “Retinal endothelial cells (RECs) in the iBRB are continuous endothelial cells (ECs) that form tight junctions to regulate the diffusion of small molecules, such as ions and water, across their cell–cell interface,” the investigators continued. The specificity of these cells and the fact that they do not form in other areas of the body make the complex tissue difficult to heal or to grow from scratch.

This image depicts both healthy (right) and deteriorated (left) human retinal endothelial cells, which are essential for maintaining eye sight. The deterioration is caused by low oxygen and high glucose levels, mimicking conditions found in diabetic retinopathy, the leading cause of vision loss in working-age people in the United States. [Duke University]
This image depicts both healthy (right) and deteriorated (left) human retinal endothelial cells, which are essential for maintaining eye sight. The deterioration is caused by low oxygen and high glucose levels, mimicking conditions found in diabetic retinopathy, the leading cause of vision loss in working-age people in the United States. [Duke University]

“When this specialized blood vessel tissue begins to break down, it can cause a lot of different diseases that lead to vision loss,” said Parker Esswein, a PhD student working in the Gerecht laboratory and co-first author of the paper. “While there are sources of retinal endothelial cells, being able to grow a continuous supply from scratch could offer many advantages for those working in the field.”

These retinal endothelial cells are currently collected and grown from real patients, making them relatively expensive with a limited supply. “A renewable source of human iBRB endothelium is thus vital for advancing eye research and treatment development,” the team noted in their paper.

To expand access, reduce cost and control variability, the Gerecht lab wanted to see if they could grow them from iPSCs. These are essentially mature adult cells reprogrammed to become primal versions of themselves that can then grow into a wide variety of other cell types.

To do this, Esswein and Ying-Yu Lin, PhD, a former PhD student in Gerecht’s lab, took commercial iPSCs and used a well-established procedure to get them to grow into common endothelial cells that form the inner layer of most of the body’s blood vessels. The researchers then used a specialized cocktail of growth factors to coax the cells into becoming the specific type of endothelial cells found in the retina. “… we differentiated human induced pluripotent stem cells into retinal endothelial cells (iRECs) via the Wnt–β-catenin pathway, namely Norrin–Frizzled4 signaling,” they explained.

Once successful, the researchers put their development to the test. In benchtop experiments, the team was able to get the iRECs to form the same networks and structures that they do within the body. The team then subjected these lab-grown tissues to low oxygen and high glucose levels, which are detrimental conditions often seen within real people. These conditions are fundamental causes of diabetic retinopathy (DR), the leading cause of vision loss in working-age people in the United States, and caused the tissue barrier to break down just like it does in patients. They wrote in summary, “Overall, we were able to robustly recapitulate the DR phenotype in 2D and 3D with our iRECs, exemplifying their ability to be utilized for in vitro disease modeling and to elucidate aberrant pathways and therapeutic targets.”

The researchers then tried their lab-grown cells as a therapy for mouse models with weak, unstructured retinal blood vessels. When injected into the mice before any actual vision loss occurred, these cells successfully integrated into the existing tissue and helped develop strong blood vessels with strong barriers. “When injected into oxygen-induced retinopathy mice, iRECs integrated into the host vascular network and revascularized the ischemic eye, rescuing the tissue,” they stated.

“The tests showed that these lab-grown cells have promise for preventative treatments, especially since they should be easier and cheaper to obtain using our technique,” Esswein said. “And while our benchtop experiments did not attempt to model a wide variety of specific eye diseases in these studies, we’re confident we can create excellent human tissue models in the lab to help better understand these diseases and uncover therapies.”

Moving forward, the researchers are planning to explore these potential uses for their retinal endothelial cells both in their laboratory and through emerging industry partnerships. The group also has a patent pending that covers both the stem cell-based therapeutics and in vitro modeling for drug discovery and testing. In their paper they concluded “Our study establishes functional human iRECs and microphysiological iBRB models that facilitate mechanistic studies aimed at identifying therapeutic targets and promoting the revascularization of injured retinas, thereby supporting treatment advancement.”

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First Huntington’s Disease Patient Dosed with Neural Stem Cell Therapy

Huntington’s disease therapeutics have reached a historic milestone—the first patient has successfully received an experimental neural stem cell therapy at UCI Health. This groundbreaking dose marks the world’s first human trial of embryonic stem cell-derived neural stem cells for the devastating neurodegenerative disorder.

The treatment, performed in May at University of California Irvine (UCI) Health, represents the culmination of more than 12 years of laboratory research and eight years of clinical planning led by scientists and physicians at the University of California, Irvine. Researchers hope the treatment, known as hNSC-01, could eventually slow disease progression, protect vulnerable brain cells and potentially restore damaged neural circuits.

To date, the first participant has not reported any serious adverse effects, according to the clinical team. A second patient is expected to receive the therapy in July.

Leslies Thompson - Huntington's
Leslie M. Thompson, PhD, Donald Bren Professor of psychiatry and human behavior, as well as neurobiology and behavior, at the University of California, Irvine [UC Irvine]

“This clinical trial highlights the important role that an interdisciplinary academic and clinical team, together with the HD families, plays in advancing medicine,” Leslie M. Thompson, PhD, clinical trial sponsor as well as the Donald Bren Professor of psychiatry and human behavior UC Irvine, told Inside Precision Medicine. “We are grateful to our patients and their incredible families for their bravery to provide hope for others with very few options.”

hNSC-01

Huntington’s disease, caused by a mutation in the huntingtin gene, destroys brain cells, causing involuntary movements, cognitive decline, and psychiatric symptoms that begin between 35 and 50 and worsen over time. Without a cure, the fatal disorder burdens patients and families emotionally, physically, and financially, often requiring daily and long-term care.

Current treatments for Huntington’s disease primarily focus on managing symptoms rather than altering the underlying disease process. Drugs such as tetrabenazine and deutetrabenazine can reduce involuntary movements known as chorea, while antidepressants, antipsychotics and mood stabilizers help address psychiatric symptoms. Physical therapy, speech therapy and occupational therapy can also improve quality of life. However, none of these approaches has been shown to slow or stop the progressive loss of neurons that drives the disease.

Over the past decade, researchers have pursued several experimental disease-modifying strategies. Among the most advanced are gene-targeting therapies designed to reduce production of the mutant huntingtin protein. These include antisense oligonucleotides (ASOs), which are delivered through repeated spinal injections, as well as RNA-targeting and gene-editing approaches intended to suppress or correct the faulty gene. While these strategies directly target the genetic cause of Huntington’s disease, clinical results have been mixed, and questions remain about long-term effectiveness, safety and the need for lifelong treatment.

The hNSC-01 neural stem cell therapy being tested at UCI Health takes a different approach. Rather than targeting the mutant gene itself, the therapy aims to protect vulnerable neurons, replace lost cells, rebuild damaged neural circuits and provide supportive factors that promote brain health. 

The UCI researchers believe stem cell-based therapies may offer a new approach by addressing multiple aspects of the disease simultaneously. The experimental treatment, hNSC-01, consists of pluripotent neural stem cells derived from embryonic stem cells and manufactured through the UC Davis Good Manufacturing Practice facility.

Preclinical studies in animal models suggested the cells could perform several functions relevant to Huntington’s disease, including protecting existing neurons, replacing cells that have been lost, rebuilding damaged neural networks and releasing beneficial proteins such as brain-derived neurotrophic factor (BDNF). The cells were also shown to reduce harmful protein accumulations associated with neurodegeneration and demonstrated long-term safety in mice.

Unlike conventional drug therapies, the stem cells are delivered directly into the brain. During the approximately six-hour procedure, performed under general anesthesia, patients lie face down within an MRI scanner while neurosurgeons use a specialized stereotactic navigation and delivery system to implant the cells into the striatum, a deep brain structure heavily affected by Huntington’s disease.

The striatum plays a central role in motor control, decision-making, motivation and learning. Degeneration of this region contributes significantly to the hallmark symptoms of the disorder. The first intervention was delivered by UCI Health neurosurgeon Jefferson W. Chen, MD, and a multidisciplinary surgical team.

Tracking treatment impact

As a Phase Ib/IIa study, the trial’s primary objective is to evaluate safety. However, researchers will also track biomarkers and clinical indicators that may provide early clues about whether the treatment is affecting disease progression.

When asked which biomarkers would help identify how the therapy is working in patients, Thompson emphasized that current measurements are focused more on assessing treatment impact than revealing biological mechanisms. “We will be including HD relevant clinical endpoints and biomarkers, including NfL in plasma and NfL and PENK in CSF; however, these are geared to understanding whether the treatment is having a benefit to these outcome measures versus informing the mechanism of action,” Thompson said.

One of the most important early indicators will be whether disease-related biomarkers remain stable rather than continuing their expected decline. “The earliest sign first and foremost is safety in this initial trial,” Thompson said. “Initial signs that the therapy could be meaningfully altering disease progression would be if the blood-based or CSF-based biomarkers do not show progression.”

Reaching the point of treating the first patient required overcoming a series of scientific, manufacturing and logistical hurdles. According to Thompson, selecting the optimal cell line was among the most significant challenges, testing multiple cell lines in vitro and in vivo.

Researchers also had to establish quality-control standards for the final therapeutic product and create Good Manufacturing Practice cell banks following extensive testing in Huntington’s disease mouse models. The COVID-19 pandemic introduced additional delays. “Disruptions caused by COVID-19, in particular the safety and tumorigenicity studies, delayed the timeline,” Thompson explained.

Another major undertaking involved creating the clinical infrastructure necessary for a first-of-its-kind procedure. Thompson said that it’s not really a challenge, but getting the overall procedural pipeline in place is the first study of this kind at the UCI Health–Irvine hospital in the MRI suite.

Despite the complexity of the project, Thompson said interactions with regulators proceeded smoothly. “We actually had a very good experience in terms of regulatory activities. A very helpful pre-pre-IND, pre-IND and relevant feedback from the FDA on the clinical trial.”

Scalability and competitive landscape

Whether hNSC-01 will ultimately compete with or complement emerging gene-targeting therapies remains unclear. Gene-silencing approaches may be easier to distribute because they do not require brain surgery, but repeated administrations over many years could result in substantial cumulative costs. In contrast, hNSC-01 involves a specialized MRI-guided neurosurgical procedure that may initially be limited to major medical centers, but it is designed as a one-time treatment whose long-term costs could compare favorably with chronic therapies if benefits prove durable.

Thompson believes the infrastructure requirements may be less of a barrier than many assume. “Yes, major medical centers can eventually offer it, and several medical centers are now using this system for other indications,” she said. “The other aspect is this would be a one-time administration so an individual could even travel to a medical center that offers the procedure.”

The REGEN4HD trial plans to enroll 21 adults aged 18 to 65 with early-stage Huntington’s disease. Twelve participants will be included in a Phase Ib dose-escalation cohort, while nine additional participants will be enrolled in a Phase IIa expansion group. The study is funded through a $12 million grant from the California Institute for Regenerative Medicine and coordinated through the UC Irvine Alpha Clinic, one of nine state-supported regenerative medicine clinical research centers.

Even if the therapy proves safe and beneficial, researchers caution that it remains unclear whether stem cell transplantation alone will be sufficient to combat Huntington’s disease over the long term. “At this point we do not know whether this will be sufficient alone or will need to be delivered with other disease-modifying therapies,” said Thompson. “For example, ones that specifically target an HD mechanism such as somatic repeat instability,” Thompson said. “However, these cells also have the potential to exert therapeutic effects directly while serving as vehicles for the delivery of additional interventions.”

For families affected by Huntington’s disease, the first successful treatment in the REGEN4HD trial represents more than a scientific milestone. It marks the beginning of a new chapter in regenerative medicine—one that researchers hope could eventually transform the outlook for a disease that has long remained untreatable.

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Treating ADHD With Methylphenidate (Ritalin, Concerta)

Methylphenidate is a stimulant medication used to treat symptoms of ADHD. It helps the brain regulate attention, focus, and impulsive behaviors.

It’s one of the two stimulants widely used in ADHD medications. Methylphenidate is the active ingredient in Ritalin and Concerta, among others. The other commonly used stimulant, amphetamine, is the active ingredient in Adderall and Vyvanse, among others. Both stimulants work by increasing levels of dopamine and norepinephrine, chemicals in the brain that control attention, focus, and impulsivity. If a child doesn’t do well on the first stimulant medication they try, they may respond better to a different formulation of that type or the other type of stimulant.

How is methylphenidate different from amphetamine?

Methylphenidate is somewhat less powerful than amphetamine and tends to have milder side effects.

If your child is under 12 and has just been diagnosed with ADHD, a doctor is likely to prescribe a methylphenidate medication first, to see how well the medication reduces their ADHD symptoms, and whether the side effects are problematic.

Methylphenidate is also many doctors’ first choice for younger children because it has been used to treat ADHD much longer than amphetamine. Ritalin (methylphenidate-based) was FDA approved in 1955, while Adderall (amphetamine-based) wasn’t approved until 1996. In countries outside the United States, amphetamine-based ADHD medications are less widely approved than those based on methylphenidate.

How methylphenidate works vs amphetamine

The two stimulants target the same brain chemicals but work slightly differently, says Paul Mitrani, MD, PhD, a child and adolescent psychiatrist at the Child Mind Institute. Methylphenidate increases the levels of dopamine and norepinephrine by blocking what’s called reuptake — the process by which nerve cells reabsorb these chemicals after they’ve been released. As Dr. Mitrani describes it, methylphenidate “enhances” the norepinephrine and dopamine the brain naturally releases by making the chemicals stay around longer. It boosts the stimulation the brain is already getting from whatever activity the child is engaged in.

Amphetamine, on the other hand, not only blocks reuptake but stimulates the release of more dopamine and norepinephrine, which is why it’s considered stronger. “Adding stimulation with amphetamine sometimes helps,” he notes. “But sometimes that added stimulation is too much, and it increases side effects the child experiences.”

Kids vary in how they respond to methylphenidate vs amphetamine

There is individual variation in how children respond to the two stimulants. So if methylphenidate doesn’t give the desired symptom relief or produces problematic side effects, it’s recommended practice to try amphetamine, or vice versa. Research shows that 70 percent of children with ADHD respond to a trial of methylphenidate. More than 90 percent will have a beneficial response to one of the stimulants if both methylphenidate and amphetamine are tried. Studies also show that approximately 41 percent respond equally well to both types of stimulant.

Children can also vary in their response to different formulations of the same stimulant, which affect the rate at which the medication goes into the bloodstream.  For instance, a short-acting form of Ritalin will kick in quickly and last for 3-4 hours, while Concerta, a delayed-release formula, lasts as long as 10-12 hours. It’s very common for kids to try several before finding the best fit.

What are the side effects of stimulant medications?

Methylphenidate and amphetamine have the same side effects, though they may be less intense with the former.

Appetite suppression

The most common side effect of stimulants is appetite suppression. It can be especially concerning with long-acting forms of the medication, which are often preferred to get better coverage through the school day. Kids who take a long-acting stimulant in the morning tend to lose their appetite for lunch and may not be interested in eating until after dinnertime.

When this is a problem, Dr. Mitrani notes that taking a shorter-acting form of the medication can help. “For instance, Concerta is a methylphenidate medication that lasts for a long time and can suppress appetite for 10–12 hours.” An alternative might be a medication that lasts for 6–8 hours, such as Metadate CD or Ritalin LA. Some children with more pronounced problems with appetite will do better on a short-acting dose in the morning and then another after lunch, he adds, since it gives them a break during the day where they can eat better.

Sleep issues

Kids who take stimulant medication can have trouble falling asleep. This can happen when a long-acting medication or an afternoon dose of a short-acting medication wears off and they get restless or hyperactive around bedtime. Difficulty falling asleep can get better after a few weeks, but if it doesn’t, it may be helpful to change either the timing or the type of the medication that is given. It’s also important to explore whether there are other contributors to sleep challenges, such as worry, screen time too close to bedtime, or lack of a consistent evening routine that helps kids calm down.

Irritability

Stimulant medications can generate agitation and irritability, which can be especially problematic in kids who are already anxious. For children with anxiety, this can be another reason to start treatment with methylphenidate, because amphetamines can feel more activating.

But Dr. Mitrani notes that treating ADHD can also reduce anxiety: “Some kids are so stressed about school — because they can’t pay attention or arealways getting in trouble — that when you treat the ADHD, they are better able to manage the demands of school and become less anxious.”

That reduction in school anxiety can also affect what happens when they get home from school. “When there is anxiety, it’s like kids are holding it together at school, and then they come home after a stressful day and just let it out,” he says. “So if the school day is less stressful, you may also see that come down at the end of the day.”

Mood changes

Some children report that stimulant medications seem to dull their personality. Dr. Mitrani suggests that this may be connected to the medication stimulating the prefrontal cortex, the part of the brain that not only manages attention and focus, but also helps regulate emotions and impulse control in other brain areas. “Enhanced control of the emotional part of the brain can cause this feeling of dullness,” he notes. “Some people will even say they feel depressed, that they’re just not like themselves because they don’t have the same energy or personality.”

If this happens to a child on methylphenidate, Dr. Mitrani will recommend trying an amphetamine or a non-stimulant medication.

Rebound effects

Some families report that their child is irritable or emotional after school or at the end of the day, when the stimulant medication is wearing off. Dr. Mitrani notes that this can coincide with the child being hungry after missing lunch. It can also be connected to the medication level dropping too quickly, and strategies that create a more gradual decrease may help take it away. For example, he might suggest adding a small dose of  short-acting form of the stimulant a half hour before the morning medication wears off.

Starting children on methylphenidate

Dr. Mitrani usually starts a child on a short-acting form of methylphenidate for two reasons: as a quick test to see if the child will experience side effects and to have an opportunity to try it twice in a day, to have more chances to assess for positive changes.

He recommends starting the medication on a weekend or a break from school and giving the child some tasks that are challenging for them because of their ADHD, like reading or something else that requires concentration, such as cleaning their room or doing household chores. “After lunch you want to try it again, to have another time point to check on. Because if you only give one dose of the medication, you don’t know if the child’s behavior was a result of the medication or some other factor. The more data points that we have, or more trials, the more information we get.”

He recommends keeping the child on short-acting doses for at least several days before trying a longer-acting formula.

Starting children on a low dose

Practice guidelines for psychiatrists recommend starting children on a low dose to assess any side effects the child might experience and gradually increasing it over 1-2 weeks with careful monitoring of response until you reach the minimum dose that will give the best symptom relief.

There is a great deal of variation in how children respond to these medications, so starting with an “average” effective dose, even adjusted by body weight, would be under-medicating some kids and overmedicating others.

For instance, for a 6- or 7-year-old child, a common starting dose of a short-acting medication might be about 2.5 mg, going up to 5 mg if more is needed for symptom relief and side effects are not an issue, Dr. Mitrani says. 

Liquid versions of either stimulant have an advantage when it comes to getting exactly the right dose, he notes: “You can do, 1 milliliter, 1.5, 1.6, depending on the syringe.”

Long-acting formulations that come in capsules can be especially frustrating, he adds — since they come in set doses and can’t be opened and divided effectively, because the beads inside are made to be triggered at different time periods.

Trying different formulations

Dr. Mitrani stresses that small differences in the formulation of a medication can make a difference in a child’s reaction.

For instance, Focalin (dexmethylphenidate) is a refined form of methylphenidate. Standard methylphenidate medications contain two mirror-image forms, or isomers, but most of the benefit comes from one of them. Focalin contains only this more active isomer. For some children, it works better, causes fewer side effects, or feels smoother.

He also notes that variations in the release patterns among long-acting formulations can affect a child’s experience. “Take Concerta, which has a unique mechanism for the extended release,” he explains. “There are three phases: a really immediate phase, then a regular Ritalin kind of phase and, then a slow extrusion of the remaining methylphenidate throughout the day that helps it last as long as 12 hours.”

By contrast, he describes Ritalin LA, which tends to last for 6-8 hours, as “50-50” — 50 percent of the dose is immediate released and the other half is delayed release. Other formulations are “40-60” or “30-70.” “These subtle differences can result in some kids responding better to one than the other, while other kids can do well on any of them.”

So even within the methylphenidate group, there may be reason to try a child on number of different formulations to get the best fit. And, of course, other reasons for trying different versions are limits on what insurance covers —which can change suddenly — and what’s available because of shortages. “And that can be really frustrating for families,” he says. “What I hear is, ‘My child was on Concerta or on Metadate CD and they made me switch to this one and now my kid’s not doing as well.’ “


When families cannot get a medication that has been working, finding another medication that’s available, that’s effective, and that insurance will approve can be a lot of hoops to jump through, he adds.

The post Treating ADHD With Methylphenidate (Ritalin, Concerta) appeared first on Child Mind Institute.

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Schistosomiasis Vaccine Shows Strong Immune Memory in Early Clinical Trials

Helminth parasites of the Schistosoma genus cause roughly 290,000 deaths annually, primarily in tropical and subtropical regions. In addition, an estimated 250 million people are currently chronically infected with Schistosoma parasites—with an additional 800 million people at risk of getting the infection—making schistosomiasis second only to malaria among the world’s deadliest tropical parasitic diseases.

The larvae, which live in fresh water, penetrate the skin and develop into adults. Schistosomiasis can be found in nearly 80 countries and is common in sub-Saharan Africa.

Now, new research shows promise for a vaccine being tested to prevent and treat schistosomiasis. SchistoShield® (Sm-p80 + GLA-SE) is a leading vaccine candidate for schistosomiasis that has successfully completed Phase I (USA) and Phase Ib (Africa) safety and immunogenicity clinical trials. Findings in a new report suggest that the vaccine triggered an adaptive immune effector and memory responses.

This work is published in npj Vaccines in the paper, “Schistosomiasis vaccine SchistoShield® induces functional immune memory responses in U.S. and African populations.”

Afzal Siddiqui, PhD, director of the Center for Tropical Medicine and Infectious Diseases and chair of the Department of Immunology and Molecular Microbiology at the TTUHSC School of Medicine has devoted decades to creating SchistoShield.

In this study, samples taken from people who’ve received trial doses of the vaccine in both the United States and Africa now demonstrate the vaccine’s effectiveness. Using Peripheral Blood Mononuclear Cells (PBMCs) obtained from intercontinental Phase I and Phase Ib trial participants, the team analyzed adaptive immune effector and memory responses to SchistoShield.

“The SchistoShield vaccine,” Siddiqui notes, “induced robust cell-mediated effector and memory responses, hallmarks of a potentially efficacious vaccine against schistosome/helminth parasites.”

More specifically, the paper reports results demonstrating that “the vaccine induced pronounced effector and memory T-cell responses. Upon recall with Sm-p80 antigen, cytokines including IFN-γ, TNF-α, IL-17A, IL-9, and granzyme B were produced, indicating the generation of functionally heterogeneous CD4 T-helper and cytotoxic lymphocyte responses. Consistent with T-helper responses that promote humoral immunity, Sm-p80 antigen-specific antibody-secreting plasmablasts were detected in vaccinated volunteers who were tracked longitudinally.”

“The people we have vaccinated, in both the U.S. and in Africa, have the memory response, both B-cell and T-cell-based,” Siddiqui said. “The vaccine is doing what it is supposed to. But always remember that these trials are very small 50 to 100 people. Now it has to go to thousands of people. So that’s where we are moving into.”

Schistosomiasis is considered a “neglected disease” because it predominantly affects impoverished communities in tropical and subtropical regions. There’s only one drug available to treat people, but it does not prevent re-infection. Through his efforts, and the support of TTUHSC, federal grants and national and international charitable and non-profit groups, Siddiqui has been able to develop SchistoShield as a humanitarian effort, rather than making it for profit.

“Our purpose from the beginning has been to expand access to care,” Lori Rice-Spearman, PhD, president of TTUHSC said. “Dr. Siddiqui’s work reflects that commitment through research that could help address a disease affecting millions of people around the world.”

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