Tag: United States

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STAT+: U.K. advocacy groups threaten court action over a key provision in the pharma trade deal with the U.S.

Two advocacy groups are demanding the United Kingdom revoke regulations at the heart of a new trade agreement with the U.S. over concerns the deal will allow outsiders to influence official decisions about the cost-effectiveness of medicines. And if the government does not comply, the groups are readying legal action.

Under the trade agreement that covers pharmaceuticals, which was finalized last month, the Trump administration committed to impose zero tariffs on medicines exported from the U.K. for at least three years. The deal is significant because it would make the U.K. the only country with tariff-free access for medicines to the U.S. market.

In return, the U.K. government took steps to appease the pharmaceutical industry, which is a key part of its economy, by pledging to increase spending on medicines from 0.3% of GDP to 0.35% by 2028 and then to 0.6% by 2035. At the same time, the U.K.’s National Health Service will increase the prices paid for by medicines by 25% and slash the maximum rebate it can claw back from drugmakers to 15%.

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In Ebola outbreak, a number of Americans in the Congo believed to have had exposure to suspected cases

A number of Americans who are in the Democratic Republic of the Congo are believed to have had exposure to suspected cases in the country’s latest Ebola outbreak, with several deemed to have had high-risk exposures, sources have told STAT. At least one of these individuals may have developed symptoms.  

One source said that there are not yet test results for any of the individuals, but the U.S. government is reportedly trying to arrange to transport them out of the DRC to somewhere they can be safely quarantined, and cared for, if they prove to have been infected. It’s not clear if that would be in the United States; there is some discussion of perhaps taking the individuals to an American military base in Germany, a source said.

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Trump blasts ‘disloyal’ Sen. Cassidy while pushing challenger in Louisiana Republican primary

NEW ORLEANS — U.S. Sen. Bill Cassidy is fighting for his political life in Louisiana’s Republican primary on Saturday as he faces a challenge backed by President Donald Trump, the latest attempt by the president to purge the party of politicians he views as disloyal.

Trump endorsed U.S. Rep. Julia Letlow over Cassidy, in an unusual attempt to dislodge an incumbent senator. Cassidy voted to convict Trump in his second impeachment trial, stemming from the attack on the U.S. Capitol on Jan. 6, 2021. Cassidy, a doctor, has also clashed with Health Secretary Robert F. Kennedy Jr. over vaccine policy, even though he provided crucial support to help Kennedy get confirmed.

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Evaluating Crowdsourced Data Collection for Carceral Death Surveillance: Pilot Study Using Amazon Mechanical Turk

Background: People who are incarcerated face significantly higher health risks than the general population, yet deaths in custody remain underreported and poorly monitored by public health systems. Although the federal Death in Custody Reporting Act requires reporting of all deaths in correctional facilities to the US Department of Justice, reporting has been inconsistent, delayed, and often publicly inaccessible. Consequently, researchers have turned to press releases issued by correctional agencies as one of the few timely sources of information on deaths in custody. However, these press releases vary widely in content and structure, making standardized data extraction difficult. Crowdsourcing platforms such as Amazon Mechanical Turk (MTurk) may offer a faster, low-cost method for gathering data, but their utility in this setting remains untested. Objective: This pilot study evaluated whether MTurk could be used to extract structured information from press releases about deaths in custody. Methods: We selected 144 press releases describing deaths between 2000 and 2023 from state prison systems and Immigration and Customs Enforcement. Each press release was assigned to 3 MTurk crowd workers (who were required to be English speaking and located in the United States), resulting in 432 individual responses. Workers were informed in advance that the task involved reviewing sensitive content related to deaths in custody. Crowd workers completed a 16-question form aligned with Death in Custody Reporting Act variables, including age, race and ethnicity, date of death, and facility location. Data quality was assessed using strict concordance (all 3 responses matched), 2-way concordance (2 of 3 responses matched), and qualitative review of common errors. Task completion time was also recorded. Sampling included complete subsets of selected press releases and a stratified subset from systems with more complex reporting formats. Results: All 144 entries were completed within 48 hours. However, agreement across crowd workers was low: strict concordance was 14.2% (20/144) for age, 12.3% (18/144) for race or ethnicity, and 11.4% (16/144) for date of birth. Qualitative review identified frequent errors, missing data, and inattentive or automated responses. Crowd workers often misinterpreted system-specific terminology or copied placeholders instead of extracting information from the source. The low agreement indicated that this baseline MTurk configuration produced insufficient data quality for more resource-intensive use. Conclusions: MTurk enabled rapid task completion but produced low-quality results when applied to extracting structured data from carceral press releases. These findings suggest that general crowdsourcing platforms are poorly suited to complex data abstraction tasks without additional training or oversight. With improved task design or support from artificial intelligence tools, crowdsourcing may help address gaps in the surveillance of deaths in custody. Long-term improvements will require consistent, transparent, and standardized reporting practices across correctional institutions.
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Exchange Marketplace Launched to Help Stalled Cell and Gene Therapies

A type of exchange marketplace for cell and gene therapies has launched to try and reinvigorate and relaunch candidate therapies that are no longer being developed due to financial constraints, despite having good science behind them.

CGTxchange, an online platform enhanced by artificial intelligence, is the brainchild of the American Society of Gene and Cell Therapy (ASGCT) and the Orphan Therapeutics Accelerator. The latter was set up in 2024 as a patient‑centered, non‑profit biotech accelerator that acquires “shelved” clinical‑stage therapies for ultra‑rare diseases and completes their development and commercialization so patients can actually access them.

The new platform was launched at the ASGCT conference in Boston and is designed to help stalled assets to find a new home. This is designed to address funding pull‑backs, program terminations, and company exits, which are forcing hundreds of cell and gene therapy programs to be discontinued or indefinitely paused despite having good scientific promise.

CGTxchange will link up companies who want to develop their assets with funders and partners who are looking for new development opportunities in cell and gene therapy.

“The cell and gene therapy field has built an extraordinary base of clinical evidence, and yet too many of those programs sit on the shelf for reasons that have nothing to do with the science,” said Terry Flotte, MD, dean of University of Massachusetts Chan Medical School and president of the ASGCT, in a press statement.

“CGTxchange gives our community a structured way to bring those programs back into view, and to connect them with the partners and funders who can help reactivate them.”

The platform is built so that people who own assets they want to develop can securely upload information about them into a database that is then searchable by possible interested parties such as investors, nonprofits, biotechs, and academic groups. They will have to pay a fee for this service, but the press statement says, “up to 10 of the initial listings will be eligible for discounted onboarding.”

When a match is achieved then the asset owner and the interested party will come to some sort of agreement on further financing. This could include traditional or alternative development and financing models, including nonprofit and hybrid structures.

The platform can theoretically be used by people from around the world, but users must apply and be approved rather than simply signing up. According to the platform’s terms and conditions on its website, “access is restricted to authorized representatives of biotechnology companies, academic institutions, nonprofits, and accredited investors.”

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PPG-Derived Digital Biomarker Developed for Peripheral Artery Disease Detection

A research team at the University of California, San Diego has developed a machine learning-based screening approach for peripheral artery disease (PAD) that uses a light-based technology called photoplethysmography (PPG) that can measure changes in blood volume in tissue. The researchers reported that short-duration PPG recordings in a patient’s toe, analyzed by machine learning models, identified PAD with a high degree of accuracy and may provide the basis for a scalable digital screening tool that could eventually be deployed through smartphones, pulse oximeters, and wearable devices. The team’s findings are published in npj Digital Medicine.

“PPG works by shining a light into tissue, in our case, the toe,” said co-first author Ava J. Fascetti, a PhD student in the digital health technology lab of senior author Edward J. Wang, PhD. “A photosensor measures how much light is reflected back, allowing us to detect tiny changes in blood volume: what we call the PPG signal.”

PAD is caused by plaque buildup in arteries that restricts blood flow, particularly to the legs and lower extremities. The disease affects an estimated 12 million Americans and 200 million adults worldwide. PAD substantially increases the risk of limb loss and major cardiovascular events, yet many patients are not diagnosed until later stages of disease progression. The researchers noted that the condition disproportionately affects underserved populations and is underdiagnosed in part because the current standard diagnostic, ankle-brachial index (ABI), requires specialized equipment, staff, and clinic visits.

“There exists a glaring unmet clinical need to develop technology to meet the demands of modern practice,” the researchers wrote. Further, ABI testing, introduced about 60 years ago, has has remained largely unchanged and has long-standing barriers to widespread use in primary care and under-resourced settings.

The current study originated from discussions between co-first author Mattheus Ramsis, MD, and assistant professor of medicine and medical director of cardiology informatics, and co-author Elsie G. Ross, MD, an associate professor of surgery in vascular and endovascular surgery, who noted that vascular labs conducting ABI testing often also collected toe PPG waveforms.

“The light-bulb went off for me at that moment,” Ramsis said.

PPG works by shining light into tissue and measuring backscattered light associated with blood volume changes. PPG has previously been used to identify cardiovascular and metabolic conditions including diabetes and atrial fibrillation. Earlier research efforts to use PPG for PAD detection had relied on small datasets, long recordings and less interpretable deep-learning approaches.

For their approach, the UCSD team assembled a dataset containing more than 10,000 toe PPG recordings from more than 3,500 patients who underwent ABI testing at UC San Diego Health between 2020 and 2025. Using these data, the researchers extracted 78 waveform features from the PPG signals that correlated significantly with ABI measurements. Those features were then used to train an explainable support vector machine model designed to identify PAD from PPG data alone.

Ramsis said the model correctly distinguished PAD cases approximately 83% of the time using only PPG data, compared with roughly 60% to 65% performance typically achieved using clinical risk-factor assessments alone. Incorporating smoking status of the patients further improved the performance of the new method.

Importantly, the model performed consistently across Black, Hispanic, and White patient populations, and among patients with diabetes, coronary artery disease, and end-stage renal disease. The researchers also reported similar performance across two UC San Diego Health campuses that used different equipment and staff.

The investigators noted that the physiologic basis for their findings align with established vascular biology. In PAD, reduced blood flow and arterial stiffness alter the morphology of PPG waveforms. Healthier patients demonstrated steeper systolic upstrokes and narrower waveform widths, while patients with PAD showed more dampened signals.

“Our findings support the existence of a reproducible PPG-derived digital biomarker that captures peripheral vascular pathophysiology relevant to ABI-defined PAD,” the researchers wrote.

The researchers said they don’t think their new model should replace ABI testing. Instead, they envision PPG screening as a complementary tool that could serve to identify patients earlier that might need further vascular evaluation.

The team said prospective deployment studies are already underway to evaluate performance in clinical settings and across additional reference standards, including toe pressure measurements, ultrasound imaging, and angiography. Additional research will also gauge performance in consumer-grade environments, including smartphones and wearable devices, and assess how the screening tool functions in broader patient populations outside specialized vascular clinics.

“If we can catch PAD early enough to prevent a limb amputation, that would be the ultimate impact: preserving limb function, reducing mortality, and addressing barriers in underserved populations,” Ramsis said.

The post PPG-Derived Digital Biomarker Developed for Peripheral Artery Disease Detection appeared first on Inside Precision Medicine.

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Antibody-Drug Conjugate Shows Activity in Hard-to-Treat Uterine Cancer

A Phase II clinical trial led by researchers at Yale School of Medicine and Yale Cancer Center has found that the antibody-drug conjugate sacituzumab govitecan, also known as Trodelvy, demonstrated encouraging clinical activity in patients with recurrent uterine cancer who had already exhausted several standard treatment options.

The findings, published in Clinical Cancer Research, suggest the therapy could become an important new option for patients with advanced endometrial cancer after chemotherapy and immunotherapy stop working.

A difficult disease to treat after relapse

Endometrial cancer is the most common gynecologic cancer in the United States, and rates continue to rise worldwide. While recent advances in immunotherapy have improved treatment for some patients, options remain limited once the disease returns after platinum-based chemotherapy or checkpoint inhibitor therapy.

Patients with recurrent disease often face poor outcomes, particularly those with aggressive tumor types such as uterine serous carcinoma and carcinosarcoma. Standard second-line chemotherapies typically produce modest response rates and short-lived disease control.

Researchers therefore wanted to investigate whether sacituzumab govitecan could improve outcomes in this challenging setting.

How the drug works

Sacituzumab govitecan belongs to a newer class of targeted therapies known as antibody-drug conjugates, or ADCs. These drugs combine an antibody that recognizes cancer cells with a chemotherapy payload designed to destroy them.

In this case, the therapy targets Trop-2, a protein commonly overexpressed in several aggressive cancers, including many uterine tumors. Attached to the antibody is SN-38, the active metabolite of irinotecan, a well-known chemotherapy drug.

By delivering chemotherapy directly to Trop-2–expressing cancer cells, researchers hope to increase anti-tumor activity while limiting damage to healthy tissue.

The drug is already approved for metastatic breast cancer and urothelial cancer, but remains investigational in uterine cancer.

Trial included heavily pretreated patients

The study enrolled 50 patients with recurrent or persistent endometrial cancer between 2020 and 2024. All participants had previously received platinum-based chemotherapy, and many had also undergone treatment with immune checkpoint inhibitors such as pembrolizumab or dostarlimab.

The study population represented a particularly difficult-to-treat group. Most patients had aggressive tumor histologies, including serous carcinoma, carcinosarcoma, or grade 3 endometrioid disease. Patients had received a median of two prior treatment regimens, with some undergoing as many as four lines of therapy before entering the study.

Encouraging responses and survival data

The trial met its primary endpoint, achieving an objective response rate of 28%. Two patients experienced complete responses with no detectable cancer remaining, while another 12 patients achieved partial responses with substantial tumor shrinkage.

In total, more than 70% of evaluable patients experienced some degree of tumor reduction during treatment. The study also reported durable responses, with a median response duration of 9.3 months. Several patients were still responding at the time of analysis.

Median progression-free survival reached 5.5 months, while median overall survival was 17.5 months in this heavily pretreated population.

Researchers also noted that responses were observed across multiple tumor subtypes rather than being limited to one specific histology.

“The results of our Investigator Initiated Trial complement and extend the TROPiCS-03 Trial results by demonstrating significant clinical activity of SG not only against the most common histological types of uterine cancer (endometrioid tumors) but also in patients harboring biologically aggressive endometrial tumors such as uterine serous carcinoma and carcinosarcoma,” said Alessandro Santin, MD, the study’s lead author.

Side effects remained manageable

As expected with potent cancer therapies, treatment-related side effects were common. The most frequent severe toxicities included neutropenia, anemia, fatigue, diarrhea, and febrile neutropenia.

However, investigators reported that most adverse events were manageable using supportive care measures such as growth factor support, anti-diarrheal medications, hydration, and dose adjustments. No treatment-related deaths were reported during the trial.

Looking ahead

The researchers cautioned that the study was relatively small and lacked a randomized comparison arm. Nevertheless, the results add to growing evidence supporting sacituzumab govitecan in advanced endometrial cancer, particularly for patients who have limited options remaining after standard therapies fail.

A larger international Phase III study is already underway to compare the drug directly against standard chemotherapy in patients with recurrent endometrial cancer following platinum chemotherapy and immunotherapy.

The team also highlighted future possibilities for combining the therapy with immunotherapy approaches, particularly because the drug’s chemotherapy payload may help stimulate anti-tumor immune responses.

“This is a major bench-to-bedside accomplishment for patients with uterine cancer,” Santin said.

 

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STAT+: Takeda will pay $13.6 million to settle allegations it paid kickbacks to doctors

Takeda Pharmaceuticals agreed to pay $13.6 million to settle allegations that it paid kickbacks to doctors to prescribe an antidepressant and, consequently, violated federal law by causing Medicaid to pay false claims, the U.S. Department of Justice said in announcing the settlement.

From January 2014 to October 2020, the company allegedly offered speaking fees and paid for meals at “high-end” restaurants to persuade physicians to prescribe Trintellix. Moreover, certain doctors who attended multiple programs on the same topic and received meals and drinks did not gain any educational benefit from attending the programs.

“This settlement demonstrates the continued commitment of my office to ensure that patients’ best interests remain paramount,” Eric Grant, the U.S. attorney for the Eastern District of California, said in a statement. “Prescribing decisions should not be influenced by drug companies’ payments or side perks made available to physicians.”

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ASGCT Q1 Landscape Report Paints Positive Picture for Gene and RNA Therapy

BOSTON – The CEO of the American Society for Gene and Cell Therapy (ASGCT), David Barrett, JD, presented highlights from the Society’s latest Landscape Report on Cell, Gene and RNA Therapy for the first quarter (Q1) of 2026.

The ASGCT report is developed in conjunction with Citeline, a subsidiary of Norstella (a pharmaceutical intelligence provider covering drug development from preclinical to commercialization).

Barrett said there are currently 42 gene therapies approved worldwide, along with 38 RNA therapies and 76 (non-genetically modified) cell therapies, which are steadily growing the field. Two cell therapies were approved in Japan in Q1.

There was a small increase in deal-making, and a significant 30% increase in startup funding compared to the same period in 2025. “I think that signals and underscores a rebounding sector,” said Barrett.

Of the eight gene therapies approved over the past 12 months, half were in the United States, with three more in China. “The regulatory pace is starting to pick up, another strong indicator for the future of our field,” Barrett said. It is a similar picture in RNA therapies. “We see a steady uptick over the course of the last year,” he added.

Zooming out, Barrett estimated that there are more than 4,200 therapies currently in development, from preclinical through pre-registration. The vast majority of those (more than 4,130) are gene and genetically modified cell therapies, including about 1,300 RNA therapies.

In the field of gene-modified cell therapies, CAR T continues to lead the pipeline for ex vivo gene therapies, with natural killer (NK) and T-cell receptors gaining traction. Not surprisingly, genetically modified cell therapy overwhelmingly targets cancers, but Barrett noted growth in the percentage of these therapies targeting immunological diseases, including lupus, multiple sclerosis, and HIV.

Pipeline growth

Barrett also noted growth and “a promising future” in the clinical trials pipeline. There are currently 350 Phase I, 319 Phase II, and 41 Phase III trials in gene therapy (up from 35 a year ago). “Hopefully, we will see a number of completed trials and FDA decisions in the near term,” said Barrett. A growing proportion of gene therapy trials (exceeding 60 percent) is for non-oncology indications.

In the RNA space, “RNAi therapies are jumping,” said Barrett. The same cannot be said, however, for mRNA. “Unsurprisingly, mRNA therapies continue to slide quarter over quarter,” a symptom of “shaken confidence” in that space, he continued. RNA therapies are targeting primarily non-oncology indications, especially in rare diseases.

Upcoming catalysts

On the business front, Barrett noted there has been “a nice uptick” in Q1 in start-up funding compared to the same quarter last year, which he deemed “a really promising indication.” The number of start-ups historically has tended to hover between 5-20. For Q1, that number was 26.

The Q1 report tracks various business catalysts anticipated through the end of 2027, including increased interest and uptake in expedited review designations—fast track, RMAT, orphan drug breakthroughs and other accelerated approval pathways.

“FDA is getting a lot done… and hopefully we’ll see the same moving forward,” Barrett said.

 

The full Landscape Report is available online from the ASGCT website.

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ASGCT 2026: Beverly Davidson Offers Vehicle and Route for Huntington’s Disease Gene Therapy

BOSTON – Geneticist Beverly Davidson, PhD, received the 2026 Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT). Davidson is currently the chief scientific strategy officer at the Children’s Hospital of Philadelphia (CHOP) and a former president of ASGCT.

Some of the research Davidson presented was conducted at a new biotech company she co-founded called Latus Bio, which earlier this month announced it had raised $97 million in a Series A round. The company develops novel AAVs to specifically target central nervous system (CNS) disorders, with a lead program in Huntington’s disease (HD).

After thanking her mentors—Bill Kelly, MD, Michael Welsh, MD, and Kathy High, MD—Davidson turned her attention to presenting new advances in engineered gene therapies. Throughout her career, she has focused on improving adeno-associated viruses (AAVs) for CNS gene therapies, with a particular emphasis now on HD. Key elements include selecting the right cargo and developing the appropriate delivery vehicle. Her goal is to scale lab research in neurons, mouse models, and non-human primates (NHPs) to treat patients, including adults with HD.

Major hurdles to tackling genetic diseases of the brain include scalability and a lack of potency, Davidson said. The search for alternative AAV serotypes to AAV2 that could target neuronal cells began back in 2000. IV administration does not provide sufficient targeting to the brain. Even AAVs that have been engineered to enter the brain from the blood have high peripheral exposure and a high cost of goods per patient, which significantly lowers scalability and impact. (In one study, liver biodistribution of AAV was many orders of magnitude higher than in the CNS.)

Davidson focused on HD, the late-onset, dominantly inherited genetic disease. The identification of the gene harboring the HD mutation in the early 1990s by a consortium of researchers was one of the biggest success stories in human genetics. Even more remarkable was the underlying disease mechanism—the expansion in exon 1 of the gene of a triplet repeat sequence (CAG) producing an abnormally long string of glutamine residues in the huntingtin protein.

The right target

One of the major challenges in devising a gene therapy for HD is ensuring that the therapeutic reaches the right network—the deep brain and cortical areas. Therapies have to reach the right circuit, and the right cells in those circuits, Davidson said. Over the years, her group has tailored AAVs for delivery to the brain, inserting peptides into exposed loops of the virion to allow for targeting and unbiased diversity for blood-to-brain delivery. Nowadays, she said, machine learning approaches can be applied for further capsid improvements.

Davidson’s CHOP lab developed a method for screening AAVs with enhanced potency for CNS therapies. After generating huge libraries containing tens of millions of novel capsids, the group performed serial enrichments to identify the most attractive capsids. After screening pools of injected capsids into two species of monkeys, a winning capsid emerged: AAV-DB-3.

Davidson’s group infused AAV-DB-3 into NHPs, looking for targeting to the putamen (base of the forebrain) and caudate regions. Those results were published in Nature Communications in 2025.  “AAV-DB-3 really stood out for its ability to transduce deep layer cortical neurons that are important” in HD, Davidson said. Moreover, the results were achieved with relatively low doses and only required a single infusion per hemisphere, outperforming the widely used AAV5.

Somatic instability

With a promising delivery vehicle identified, Davidson next addressed the therapeutic strategy, which takes aim at the somatic expansion of the CAG repeat. This codon grows longer over time in certain cells in the brain, sometimes expanding to hundreds of repeats.

MSH3 is a DNA repair protein that is required for CAG repeat expansions, as seen in mouse models of HD and other triplet repeat disorders, including myotonic dystrophy. Research led by Paul Ranum, PhD, who is a co-founder of Latus Bio, posted in a preprint on bioRxiv earlier this year, modeled the impact of lowering levels of MSH3 on somatic instability.

Ranum and colleagues used an artificial microRNA showed to lower MSH3 levels in NHPs by 48-94 percent. Computational modeling suggests that this would reduce somatic instability and delay onset of HD symptoms by many years. Early studies using a well-known HD mouse model, the Q111 mouse, to assess biodistribution, quantify knockdowns, and assess the impact on somatic CAG repeat expansion. AAV-DB-3 expression is highest in the striatum and cortex at 16 weeks, dropping MSH3 levels by 50%.

Davidson closed by emphasizing the need to ensure scalability for treatment beyond ultra-rare disorders. Latus hopes to file an Investigational New Drug application for its HD therapy, LTS-201, in the second half of 2026. At least two other biotech companies are also targeting MSH3 by other means.

 

 

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