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FDA Minutes Confirm Challenging Path Ahead for UniQure’s Huntington’s Gene Therapy

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.

The final minutes from uniQure’s pre–biologics license application meeting with the FDA are in, and they confirm the company’s and analysts’ suspicions—it’s going to be a longer regulatory road to market than anticipated for the company’s Huntington’s disease gene therapy.

The final minutes are “consistent” with a previous update on Nov. 3. At that time, five weeks after reporting highly positive three-year data from a pivotal trial of AMT-130, uniQure shared initial feedback from this meeting, revealing that the FDA “no longer agrees” that data from its Phase I/II trials would be “adequate to provide the primary evidence in support of a BLA submission.”

In uniQure’s press release on Thursday, the company added that the agency “conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.” UniQure is “carefully” evaluating this feedback and intends to “urgently” seek a follow-up meeting with the FDA, to take place during the first quarter of 2026.

UniQure’s stock, which fell to just over $26 on Nov. 3 from the previous closing price of $67.49, was down to just under $23 as of this writing.

Investors had not been expecting a speedy reversal of the November guidance, according to Stifel analysts.

“As time has passed since the 11/3 announcement, we think investors’ optimism has waned around a quick reversal at FDA,” they wrote in a note to investors Thursday morning.

“As we see it, there are still many scenarios on the table here,” the analysts continued, ranging from, “(1) an unfortunately challenging path forward for AMT-130–i.e. need an outright new study, to (2) some sort of compromise, i.e. more patients followed for longer, to (3) even a FDA reversal, perhaps helped by political/external pressure.”

The past four months have been a rollercoaster ride for uniQure—and for patients with Huntington’s, a genetic, neurodegenerative disease.

On Sept. 24, uniQure reported three-year data from a pivotal Phase I/II trial AMT-130, showing that the gene therapy slowed disease progression by 75%, beating analyst expectations. Plans were underway to file a biologics license application (BLA) in the first quarter of 2026, and uniQure’s shares soared 248%.

But thatwas followed by the FDA’s feedback in November—a seeming reversal of position as uniQure had previously aligned with the agency on the protocols and statistical analyses used in its trials—specifically, comparing AMT-130 to a natural history external control, according to uniQure’s announcement.

“We still view the agency’s change in guidance as very surprising given the numerous interactions uniQure has had with the FDA since its November 2024 meeting,” William Blair analysts wrote in a note to investors on Thursday. They added that uniQure’s trial design aligns with the FDA’s recent publication of a plausible mechanism pathway for personalized therapies, as well as draft guidance on gene therapies for small populations, “which specifically stipulates that external controls may be an appropriate option in certain contexts.”

If approved, AMT-130 would be the first genetic treatment for Huntington’s disease.

News Staff by — December 05, 2025 By News Staff in Bio Space
Former CDC, FDA Chiefs Decry Vaccine Policy Changes as ACIP Meeting Begins

Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials.
More than a dozen former FDA and CDC officials warned of potential changes to federal vaccine policy in dual editorials published in two prominent medical journals on Wednesday.

The warnings come as the final 2025 meeting of the CDC’s Advisory Committee on Immunization Practices gets underway, where a number of changes, including to the birth dose of the hepatitis B vaccine, are set to be debated.

In an editorial in the New England Journal of Medicine published Wednesday, a dozen former FDA commissioners wrote that they are “deeply concerned by sweeping new FDA assertions about vaccine safety and proposals that would undermine a regulatory model designed to ensure that vaccines are safe, effective, and available when the public needs them most.”

Led by former commissioners Robert Califf and Janet Woodcock, the officials warned that proposed changes in a memo sent by Center for Biologics and Evaluation Research chief Vinay Prasad would “upend” in a “unilateral” manner longstanding policies around vaccine development and updates.

Among a series of changes, Prasad’s memo, according to the writers, “rejects” the FDA’s use of immunobridging studies, which serve as “a reliable correlation with effectiveness” for well-understood vaccines with existing safety data, particularly for viruses that mutate frequently, like COVID-19 and influenza, and need seasonally updated vaccines.

Epidemiologists use immunobridging studies to compare updated vaccines to previous iterations, using immunological markers like neutralizing antibody titers to infer effectiveness.

Also on Wednesday, three infectious disease specialists and epidemiologists, led by former CDC chief Rochelle Walensky, published an editorial in the Journal of the American Medical Association, warning of the risks of changing the CDC’s policies around birth dose hepatitis B vaccination, the topic of the first day of ACIP’s December meeting.

In their JAMA piece, the authors, including Michael Abers, an infectious disease doctor at the Albert Einstein College of Medicine in New York and Angela Ulrich, an epidemiologist at the University of Minnesota, estimated the number of perinatal infections that occur under the revised guidelines.

Pushing the birth dose of hepatitis B, given to newborn infants, to one month post-birth would immediately increase hepatitis cases 8%, according to an analysis done by the authors. Further restricting the birth dose only to infants born to mothers testing positive for hepatitis B—another potential change under discussion at ACIP—would raise perinatal cases by 63%-76%.

While the authors said that they “applaud” revisiting routine vaccine recommendations as new data emerges, they warned that “delaying the first hepatitis B vaccine dose beyond the newborn period introduces risks that have lifelong detrimental consequences and no measurable health benefit.”

News Staff by — December 05, 2025 By News Staff in Bio Space
Pegcetacoplan—the ‘closest thing to a cure’ for rare, severe kidney disease

A rare and life-threatening kidney disease in children finally has an effective therapy, thanks in large part to pioneering research and clinical leadership from University of Iowa Health Care Stead Family Children’s Hospital.

The disease, known as C3 glomerulopathy (C3G), is an ultra-rare condition that primarily affects children and young adults. Only around 5,000 Americans have C3G, which causes progressive kidney damage, with more than half of patients reaching end-stage kidney failure within a decade of diagnosis.

Unlike previous treatments for C3G that aimed to alleviate the damaging inflammatory process of the disease, the new, first-of-its-kind drug directly targets the root cause of C3G dysfunction in the body’s complement system, a part of the immune response.

Carla Nester, MD, director of the Rare Renal Disease Clinic at UI Health Care, led the global pediatric clinical trial for the new drug known as pegcetacoplan. The results were striking: there was a 68% reduction in the amount of protein in the patients’ urine, and stabilization of kidney function. Up to 67% of children achieved complete remission, and 72% showed no disease activity on their kidney biopsies.

“This is the closest thing to a cure we’ve ever seen for this disease,” says Nester, a professor of pediatrics with UI Health Care Stead Family Children’s Hospital, who also is the senior author of the study, which was published in the New England Journal of Medicine.

“We still need to follow the long-term outcomes for these patients, but the data from this trial are absolutely amazing.”

The Phase III, randomized, double-blind, placebo-controlled trial was conducted at 122 centers in 19 countries and included 124 patients.

Based on the results, pegcetacoplan was approved earlier this year by the U.S. Food and Drug Administration (FDA) as the first treatment for patients 12 years and older with C3G and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), a closely related rare and severe kidney disease.

This latest clinical advance is grounded in foundational research on the complement system led by Richard Smith, MD, UI professor of pediatrics and otolaryngology, and an expert in the causes of complement-related kidney diseases, and hearing loss.

That research, combined with Nester’s clinical expertise in glomerular diseases, has made Iowa and the Stead Family Children’s Hospital one of a very few centers in the U.S. capable of providing complete care to C3G patients, including efficient diagnosis, state-of-the-art management, and the opportunity to participate in both basic science and clinical research like the VALIANT trial, which tested pegcetacoplan.

“It’s the combination of the research and the clinical unit that makes Iowa such a powerhouse,” Nester says.

Importantly, the UI team has also earned the trust of patients and families affected by C3G, with patients traveling from across the country and around the world to seek care at the UI Rare Renal Disease Clinic.

“One of our patients used to fly in from Qatar to be seen in our clinic, and another moved from Europe to be under our care,” Nester says. “That’s how well known and trusted our program has become.”

This strong reputation and connection with the patient community allowed UI Health Care to be the highest-enrolling center in this global study. Recruiting enough patients into these types of clinical trials for rare diseases is critical for demonstrating that a drug therapy is effective.

From bench to bedside—and beyond

The research done in Smith’s lab over many years has advanced the understanding of the underlying biology of C3G and led to a realization that inhibiting excessive activation of the complement pathway could be the key to an effective treatment. Determined to translate this knowledge into therapies for patients, the UI researchers collaborated with pharmaceutical companies to develop and test new drugs that could inhibit complement activation.

Unlike previous treatments, which relied on broad anti-inflammatory drugs like steroids, or medications that targeted the wrong aspect of the complement pathway, this new class of medication precisely blocks the malfunctioning part of the complement system.

Pegcetacoplan is administered via a twice-weekly injection, which many young patients prefer over daily oral medications. Another new drug called iptacopan, which also inhibits the complement system, was also approved earlier this year to treat adults with C3G. UI Health Care was the lead enroller globally for that clinical trial, as well, which led to iptacopan’s approval.

For patients, the impact of these new medicines is profound. Nester recalls one college student who had been struggling with the disease and is now in full remission and thriving in his career, as well as pediatric patients who no longer have to worry constantly about their health and can instead look forward to normal childhoods.

“This is the year we finally get to help patients,” Nester says. “It’s been a long road, but we’re here.”

News Staff by — December 04, 2025 By News Staff in Global News
Airplane and hospital air is cleaner than you might think

When it comes to the air in public places, germophobes can breathe a bit easier. According to a new Northwestern University study, the ambient air on airplanes and in hospitals mostly contains harmless microbes typically associated with human skin.

In the first study of its kind, scientists used an unexpected sampling tool—used face masks and an aircraft air filter—to uncover the invisible world of microbes floating in our shared air. Their results revealed that the same types of harmless, human-associated bacteria dominate both airplane and hospital air.

Across all samples, the team detected 407 distinct microbial species, including common skin bacteria and environmental microbes. While a few potentially pathogenic microbes did appear, they were in extremely low abundance and without signs of active infection.

Not only does the study help illuminate which microbes exist in shared air, but it also demonstrates that face masks and air filters can be repurposed as noninvasive, cost-effective tools to monitor confined, high-traffic environments.

The study is published in the journal Microbiome.

“We realized that we could use face masks as a cheap, easy air-sampling device for personal exposures and general exposures,” said Northwestern’s Erica M. Hartmann, who led the study. “We extracted DNA from those masks and examined the types of bacteria found there. Somewhat unsurprisingly, the bacteria were the types that we would typically associate with indoor air. Indoor air looks like indoor air, which also looks like human skin.”

An expert on indoor microbiomes, Hartmann is an associate professor of civil and environmental engineering at Northwestern’s McCormick School of Engineering.

A second life for used face masks

Hartmann and her team conceived the project in January 2022, amid the COVID-19 pandemic. At the time, travelers were increasingly concerned about how well airplane cabins filtered and circulated air. Hartmann planned to collect airplanes’ cabin filters to look for evidence of pathogens.

Although Hartmann did procure an aircraft’s high-efficiency particulate air (HEPA) filter, which had been used for more than 8,000 flight hours, she quickly realized the project might be impractical.

“At the time, there was a serious concern about COVID transmission on planes,” Hartmann said. “HEPA filters on planes filter the air with incredibly high efficiency, so we thought it would be a great way to capture everything in the air. But these filters are not like the filters in our cars or homes. They cost thousands of dollars, and in order to remove them, workers have to pull the airplane out of service for maintenance. This obviously costs an incredible amount of money, and that was eye-opening.”

Searching for another method that passively trapped microbes, Hartmann and her team pivoted to a much cheaper and much less disruptive tool: face masks. For the study, volunteers wore face masks on both domestic and international flights. After landing, they put the masks into sterile bags and sent them to Hartmann’s lab. For comparison, Hartmann also collected face masks that volunteers took on flights but never wore.

To understand how indoor environments differ, Hartmann and her team wanted to examine another high-traffic, enclosed environment with heavily filtered air. The team selected hospitals as the second testbed. After wearing a face mask during a shift, hospital workers submitted their masks to Hartmann’s lab.

“As a comparison group, we thought about another population of people who were likely wearing masks anyway,” Hartmann said. “We landed on health care providers.”

The sky is clear

After receiving masks from travelers and health care workers, Hartmann’s team collected DNA from the outsides of masks. They found the air in hospitals and on airplanes contains a diverse but mostly harmless mix of microbes, with only minimal traces of potentially pathogenic species.

In both environments, common human-associated bacteria—especially those found on skin and in indoor air—dominated the samples. Although the abundance of each microbe present was slightly different, the microbial communities from hospitals and airplanes were highly similar. The overlap suggests that people themselves—rather than the specific environment—are the main source of airborne microbes in both settings. And those microbes floating around indoor air come from people’s skin, not from illness.

Hartmann’s team also identified a handful of antibiotic resistance genes, linked to major classes of antibiotics. While these genes do not indicate the presence of dangerous microbes in the air, they highlight how widespread antibiotic resistance has become.

Although indoor air might not be as harmful as some people may have feared, Hartmann emphasizes that airborne spread is just one way infections can travel. For many common illnesses, other routes—such as direct contact with an infected individual or interacting with high-touch surfaces—are far more important.

“For this study, we solely looked at what’s in the air,” Hartmann said. “Hand hygiene remains an effective way to prevent disease transmission from surfaces. We were interested in what people are exposed to via air, even if they are washing their hands.”

News Staff by — December 04, 2025 By News Staff in Global News
AI-powered vision gives meaning to wildfire chaos

How wildfires spread is more variable and unpredictable than Canada’s standard models assume, new research from UBC Okanagan data scientists shows.

Ladan Tazik, lead author of a new study in Fire and UBC Okanagan doctoral student, used advanced computer vision tools to capture fire behavior with a level of detail that wasn’t possible even a few years ago.

Her work sheds light on the random elements of fire movement—information that could reshape how fire behavior is modeled and forecasted in an era of worsening wildfire seasons.

“Image processing techniques let us quantify fire behavior in real time, including the parts that don’t follow consistent patterns,” says Tazik. “By capturing the randomness in how fires spread, we can build models that better reflect reality and help improve decision-making during active fire events.”

Tazik led the design, analysis and modeling that form the backbone of the study.

She used the “Segment Anything Model,” a state-of-the-art AI tool, to extract fire perimeters from experimental burn videos frame by frame to study fire spread dynamics.

This allowed her to study directional fire spread on sloped terrain without assuming the fire behaves predictably or spreads in a simple line.

Her analysis confirmed something firefighters may know instinctively: fires race uphill. But when she compared her measurements with the values used in Canada’s official Fire Behavior Prediction System, the numbers didn’t always line up.

Real-world fire behavior challenges models

Real fires often moved faster, and the influence of slope wasn’t consistent from place to place.

She tested the method on ponderosa pine and Douglas fir fuels often used in fire research.

This highlights that small differences in fuel, wind and terrain can add to the unpredictability of fire and introduce important variations in how it spreads.

Even under nearly identical conditions, the flames didn’t behave the same way twice.

In practical terms, that means most fire spread is shaped by randomness—far more than today’s deterministic models capture.

“These results show that we need to pair every spread estimate with a measure of uncertainty,” Tazik explains. “Simply multiplying by a slope factor isn’t enough. Fire is dynamic, and our models should acknowledge that.”

Advancing wildfire science with new tools

Research supervisor Dr. W. John Braun says the project demonstrates how emerging computer vision tools can transform wildfire science.

“Tazik proposed innovative ways to tackle this difficult modeling problem,” he says. “Her work shows how high-resolution perimeter data and advanced modeling can help us understand the real variability in fire behavior. That’s essential if we want to move toward more probabilistic, data-driven prediction systems.”

The study also included contributions from Dr. John R.J. Thompson, Assistant Professor of Data Science, Mathematics and Statistics, as well as other partners who provided the experimental and field video datasets. While the fuel experiments supported the research, Tazik alone led the segmentation and modeling components.

Tazik says the next step is to expand the approach to more fuel types and fire conditions and use airborne or satellite imagery to study fire spread dynamics.

With more Earth observation and remote sensing tools available, she sees an opportunity to build models that better capture wildfire dynamics while embracing the inherent uncertainty of fire, rather than smoothing it away.

“Fires don’t behave perfectly,” she says. “Our tools shouldn’t pretend they do.”

News Staff by — December 04, 2025 By News Staff in Biotechnology Today
A new tunable cell-sorting device with potential biomedical applications

Poly(N-isopropylacrylamide) (PNIPAM) hydrogel undergoes significant but precise changes in size between 20°C and 40°C, making it an excellent candidate for use in variable-size deterministic lateral displacement (DLD) array devices. Researchers from Science Tokyo have built a tunable DLD cell-sorting platform and verified its ability to sort cancer cells of defined sizes from blood samples. This platform could offer high-resolution size-based cell sorting for a wide variety of biomedical applications.

Importance of cell sorting in diagnostics

Isolating specific kinds of cells from their surrounding tissue is a crucial step in many medical diagnostic processes. For instance, detecting cancer cells in the blood is necessary to identify whether cancer has metastasized. Size-based cell sorting techniques, such as deterministic lateral displacement (DLD), have grown popular in recent years, thanks to their high-throughput and preservation of metabolic activity in the isolated cells.

DLD uses arrays of precisely spaced micropillars. Cells smaller than a certain critical diameter (Dc) are diverted to one side of the array, while those larger than Dc are diverted to the opposite side. However, this means that a typical DLD device can only sort cells based on one specific Dc, which limits its utility. This also means that DLD devices are at risk of fouling and blockage, since there is no effective way to remove large-diameter objects that get caught within the array.

Development of the tunable DLD device

A team of researchers from the Institute of Science Tokyo (Science Tokyo), Japan, has created a tunable DLD device using poly(N-isopropylacrylamide) (PNIPAM) hydrogel micropillars. This project was led by Associate Professor Takasi Nisisako and Assistant Professor Yusuke Kanno of the Institute of Integrated Research, Science Tokyo, together with graduate student Ze Jiang from the Department of Mechanical Engineering, School of Engineering, Science Tokyo, Japan. Their work is published in the journal Lab on a Chip.

PNIPAM undergoes precise changes in size between 20°C and 40°C, making it an excellent candidate for a DLD with variable Dc. “In our previous work, we demonstrated a thermo-responsive DLD array on a glass substrate using hydrogel micropillars composed of PNIPAM within a poly(dimethylsiloxane) (PDMS) microchannel,” Nisisako says.

“The PNIPAM-based approach requires no complex external field-generating equipment, offers a simpler fabrication process, and facilitates size-based separation through direct temperature-driven modulation of pillar dimensions.”

The latest version of the team’s DLD device consists of a silicon base on a Peltier element. PDMS microchannels that are plasma-bonded to silicon carry the liquid sample and sheath fluid to the PNIPAM microarray. Two outlets—L and S—at the far end of the array allow sorted cells to be separated from the rest of the sample. “The use of silicon, chosen for its superior thermal conductivity, enabled more precise Dc control,” says Nisisako, listing the improvements in this version.

“Plasma bonding of the PDMS channel to the silicon substrate allowed stable, positive-pressure operation across a range of flow rates. Furthermore, by employing a new PNIPAM-based photoresist with higher polymer concentration, we fabricated micropillars up to 30 μm in height, suitable for processing diverse biological particles.”

Testing and future applications

The team verified the tunability of their device using blood samples spiked with Michigan Cancer Foundation-7 (MCF-7) breast adenocarcinoma cells. With an average diameter of 17 μm, MCF-7 cells are significantly larger than blood cells. The team passed the sample through the array at 25°C (Dc = 14.1 μm) and achieved 90% sorting efficiency of MCF-7 cells into outlet L. At 26 °C (Dc = 18.5 μm), similar numbers of MCF-7 cells were seen in both outlets, but those at outlet L were consistently larger than those at outlet S. At 37 °C (Dc = 29 μm), all MCF-7 cells were at outlet S.

Buoyed by the successful validation of their tunable DLD device, Nisisako aims to verify its performance when used with actual biological samples from patients. “The precision, versatility, and reliability of this platform underscore its potential for high-resolution size-based sorting, making it a promising tool for a wide range of biomedical applications.”

News Staff by — December 04, 2025 By News Staff in Biotechnology Today
New UK/EU Rules and AI Adoption Define CRO Priorities for 2026

With new UK clinical trial rules landing in 2026, the EU Biotech Act on the horizon and China and Australia gaining ground, CROs are zeroing in on study timelines, AI/ML and data privacy as the industry’s next pressure points.

Pending and proposed changes to UK and EU clinical trial regulations, especially concerning trial timelines, are among the key focus areas for CROs in 2026, said Kathy Noonan, senior vice president of global regulatory policy at the Association of Clinical Research Organizations (ACRO), in an interview with BioSpace.

Other topics that have engaged members include AI/ML and data protection and privacy.

The Washington, DC-based ACRO has 15 members, including global CROs such as ICON, IQVIA, Parexel and PPD, and technology and platform companies such as Medidata, Oracle and Zelta. In 2024, ACRO members employed more than 73,000 individuals across Europe.

Noonan said there has been increased recognition within life sciences of the need for Europe to bolster its clinical competitiveness versus the U.S., China and Australia. As BioSpace previously reported, in-China trial volume has risen rapidly compared to the U.S. and Europe, positioning the country as a major global player in clinical development. Australia has also become an attractive hub for clinical studies, especially early stage trials.

UK/EU Regulations at the Forefront

ACRO’s European regulatory committee analyzes regulatory policy and legislation impacting the clinical research industry at the pan-European and national levels in Europe. It has engaged in industry consultations to press for faster trial timelines in Europe, Noonan noted.

A key CRO industry interest is the revised UK clinical trial regulations which take effect on April 28, 2026, and aim to strengthen participant safety and accelerate research. Key changes include legally requiring trial registration on a public register and the publication of results within 12 months, streamlining the application and approval processes with a single application route and harmonizing with international standards.

Noonan said there is also much member attention on the EU Biotech Act, a proposed legislative package from the European Commission intended to boost the European biotechnology sector. Its primary goals are to simplify regulations, accelerate product approvals and streamline processes to help biotech innovations move from the lab to the market more quickly. The act also aims to improve access to funding for biotech companies and enhance the workforce’s skills.

In terms of regulatory guidelines, ACRO members are also revamping processes to implement the ICH Guideline for Good Clinical Practice E6 (R3), published in January 2025. In terms of key changes from previous guidance, they “reflect advancements in trial design, technology and the digital ecosystem for trials, with expanded content on data governance and computeri[z]ed systems.”

AI/ML Remain Priorities

In addition, to faster trial timelines, ACRO members are invested in AI/ML growth, Noonan said. The organization has a committee that convenes member company subject matter experts engaged in the development and use of AI/ML tools in the design, conduct, oversight and analysis of clinical trials.

In April, ACRO submitted comments to the FDA on its draft guidance “Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products.” Overall, the organization supported the guidance, as it found it “flexible, pragmatic and very risk-based” and provided industry with the confidence to develop these applications further, Noonan said. ACRO expects the European Medicines Agency to issue AI/ML- related guidance documents soon as well, with the hope the FDA document will be a good model.

ACRO plans to survey its members about how its members are using AI/ML internally, in terms of operational uses and how they are incorporating it into clinical trials.

U.S. lawmakers have a keen interest in incorporating AI into clinical research, with many conversations in the Senate, noted Fiona Lewis, ACRO’s advocacy associate. The aim is to drive U.S. leadership in the AI field, especially in terms of competition with China.

Another intersection with AI that ACRO members are increasingly interested in is sustainability, said Maddy Sever, the organization’s communications and member associate. “We’ve heard a lot about using AI and digital tools to create more sustainable trials, whether that’s decentralized trials or for internal operations,” she said. ACRO’s members are in turn seeing customers increasingly ask for sustainability metrics, she added. The organization created a sustainability committee this year to focused on advancing sustainable practices in clinical research.

Eye on Data Protection and Privacy

Data protection and privacy issues directly impact ACRO members, Noonan said. As CROs handle sensitive data across increasingly global trial sites, cross-border data security is a concern.

Pharmaceutical companies must share proprietary knowledge, patient information and intellectual property with their CRO partners when outsourcing highly sensitive research tasks. However, the methods used to transfer this information introduce security vulnerabilities that may result in data leaks, unauthorized access to protected assets or accidental exposure of private details, according to a Business Research Insights report. Another caveat is that data protection regulations vary widely among countries.

The topic is relevant as according to internal research, 50% of clinical trials have data capture issues, said Ali Pashazadeh, founder of TreeHill Partners.

An internal ACRO committee comprised of member company data protection and privacy professionals considers relevant legislation and regulation globally, including hot topics such as data localization and transfer, Noonan said. In addition, the committee engages with professional colleagues such as European Federation of Pharmaceutical Industries and Associations and the International Pharmaceutical and Medical Device Privacy Consortium, Noonan added.

News Staff by — December 04, 2025 By News Staff in Bio Space
Capricor Heads Back to FDA With Pivotal Results for DMD Cardiomyopathy Therapy

Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.

Christmas came early for Capricor Therapeutics, which announced Wednesday that its investigational cell therapy for Duchenne muscular dystrophy cardiomyopathy—which the FDA rejected in July—hit both the primary and second endpoints in a pivotal Phase III trial.

In the Phase III HOPE-3 trial, deramiocel showed statistically significant benefits in upper-limb function, the trial’s primary endpoint. The treatment also slowed a decline in cardiac function as measured by left ventricular ejection fraction, satisfying the key secondary endpoint.

Deramiocel elicited a 54% slowing of skeletal muscle disease progression, which principal investigator UC Davis Health professor Craig McDonald called “extraordinary in Duchenne” in Capricor’s statement on the results. Cardiac function decline was slowed by 91% vs. placebo.

In Wednesday’s announcement, McDonald noted that HOPE-3 study is the first-ever Phase III trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint.

“This call this morning represents the culmination of 20 years of work,” Capricor CEO Linda Marbán told analysts on a conference call Wednesday morning. “Through all the years of understanding the mechanism of action, trying to find the perfect way to deploy the powers of [deramiocel], we are now proud to be able to present today the data . . . that shows that the [HOPE-3 trial] has shown statistically significant improvement in both skeletal and cardiomyopathy.”

With these pivotal data in hand, Capricor plans to submit its response to the complete response letter it received from the FDA in July.

“We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 [open label extension] studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval,” Marbán said in a statement.

HOPE on the Horizon?

It’s been a long road for Capricor and deramiocel. In the July CRL, the FDA said Capricor’s data package “fell short of the “statutory requirement for substantial evidence of effectiveness.”

This was a surprise to Marbán. “The complete response letter was unexpected given the trajectory of positive interactions [with the FDA],” she told investors on the company’s second quarter earnings call on August 11.

Deramiocel and Capricor have been wrapped up in the FDA’s ongoing personnel and communications dramas this year. In June, Center for Biologics Evaluation and Research Vinay Prasad canceled a previously scheduled advisory committee meeting for deramiocel. Marbán was caught off guard when she learned that the meeting was no longer listed on the Federal Register.

“Investors watch these boards all day, every day, so my phone started blowing up with ‘your adcomm has been canceled. It’s been taken down. Why?’ I had no answers to give,” Marbán told BioSpace in August. “I couldn’t make an official statement.”

Deramiocel was also reportedly at the heart of the ouster of Nicole Verdun, the former director of the office that reviews cell and gene therapies, and her deputy, Rachael Anatol, who were pushed out of the agency in June. The two leaders were allegedly “put on administrative leave because of an argument about our file, and that Prasad wanted to CRL us and Nicole was fighting it,” Marbán recounted hearing from a STAT reporter who’d contacted her after receiving a tip from an agency insider.

While a significant amount of attention and R&D dollars are poured into developing treatments for the muscular effects of Duchenne muscular dystrophy, Marbán told BioSpace in August that cardiomyopathy is what typically takes a patient’s life.

“Duchenne takes typically between 25 and 30 years to take the life of a boy or a young man and every day during that progression of the disease, they are losing some aspect of their ability to function,” Marbán said on Wednesday’s conference call. “We hope to be able to attenuate the course of this disease with deramiocel moving forward.”

News Staff by — December 04, 2025 By News Staff in Bio Space
Exercise slows tumor growth in mice by shifting glucose uptake to muscles

It’s well known that exercise is good for health and helps to prevent serious diseases, like cancer and heart disease, along with simply making people feel better overall. However, the molecular mechanisms responsible for preventing cancer or slowing its progression are not well understood. But, a new study, published in the Proceedings of the National Academy of Sciences, reveals how exercise can increase glucose and oxygen uptake in the skeletal and cardiac muscles, instead of allowing it to “feed” tumors.

Reduced tumor growth in exercised mice

To study how exercise-induced metabolic changes affect tumor growth, the research team injected mice with breast cancer cells and fed some of the mice a high-fat diet (HFD), consisting of 60% calories from fat, while others were fed a normal diet as a control. The HFD mice were given running wheels for exercise, although exercise was voluntary. The team used stable isotope tracer studies [U-13C6] glucose and [U-13C5] glutamine to track metabolic changes.

After 4 weeks of wheel running, the team found a significant difference in tumor sizes between mice that chose to exercise, compared to those that did not—even when they were fed the same diet.

The study authors write, “Obese mice which underwent 4 weeks of voluntary wheel running after tumor injection exhibited nearly a 60% reduction in tumor size. The exercised mice had greater lean mass and lower fat mass than their nonexercised, obese counterparts, with plasma glucose and insulin concentrations comparable to the sedentary chow fed controls.

“After a 30-min bout of acute moderate intensity (15 m/min) treadmill exercise, the exercised obese mice had higher cardiac and skeletal muscle 2-deoxyglucose uptake and reduced tumor glucose uptake.”

The researchers also analyzed exercise-induced changes in mice with a type of melanoma that is typically not exacerbated by obesity (in contrast to breast cancer). Still, after four weeks of exercise, obese mice with melanoma had significantly smaller tumor sizes, along with reductions in tumor glucose uptake and oxidation compared to the sedentary controls. These results suggest that the shift in glucose (and thus the slowing of tumor growth) is not tumor-type specific.

Some mice also underwent “prehabilitation”—exercise undertaken before tumors were introduced. Similar beneficial results were found in these mice. The study authors explain, “These observations may be due to an earlier achievement and maintenance of body composition or overall fitness (VO2 peak) with early exercise exposure.”

Exercise-induced metabolic changes

The repartitioning of glucose to cardiac and skeletal muscles instead of tumors appears to play a major role in slowing tumor growth, but there are other changes taking place, as well. The team also found 417 genes related to energy metabolism and other metabolic pathways that were expressed differently between exercised and sedentary lean mice.

The team says that a downregulation in a protein referred to as mTOR, is seen in the exercised mice and this may be helping to slow tumor growth, along with processes like shifts in the use of amino acids, which tumors have been documented to utilize.

Does exercise slow tumor growth in humans?

The team also gathered gene expression data from another study, which analyzed exercise training in women with breast cancer and a metaanalysis of skeletal muscle responses to multiple types of acute and chronic exercise. The data revealed an upregulation in the glutamine and leucine channeling genes in the muscle tissue of humans who exercise. They say that no distinct differences were observed in the expression pattern of these genes when comparing exercise intensity, which may be due to the small sample size or the limited number of analyzed genes.

So, more research is certainly required, but since metabolic pathways are similar in humans, mice, and other mammals, there is likely a similar relationship between exercise and tumor growth in humans. This would be in line with other studies indicating tumor suppression with the help of exercise in humans.

Overall, researchers remain optimistic about exercise in the role of cancer treatment in humans. A better understanding of how glucose is utilized after exercise in humans with tumors can inform prehabilitation strategies for cancer patients, help doctors integrate fitness into cancer therapies, and potentially assist researchers in identifying new therapeutic targets.

“We anticipate that this work may lay the groundwork to reveal key insights into the role of systemic adaptations to exercise in broader antitumor therapies. Further, examination of the role of fitness on the molecular pathways altered by exercise may uncover new therapeutic targets in precision oncology, particularly in patients who cannot tolerate exercise,” the study authors explain.

News Staff by — December 03, 2025 By News Staff in Global News
Childhood instability accelerates women’s sexual strategies, study suggests

California State University, Sacramento, researchers traced how disordered childhood social worlds in women connected to faster life history traits and greater mating effort, with those traits explaining 22.2% of the association between childhood microsystems and adult sexual behavior.

Childhood environments and strategies

Life History theory treats childhood ecology as a starting point for strategies that govern survival, mating effort, and parental effort. Mating effort involves behavior that increases access to sexual opportunities, while parental effort involves investing time and resources so children survive to reproduce.

Faster strategies align with earlier sexual debut, more short-term mating, more lifetime partners, and more offspring at younger ages. Slower strategies align with later sexual debut, safer reproductive behavior such as monogamy and contraceptive use, fewer lifetime partners, and greater parental investment.

Childhood harshness and unpredictability show up in prior work through divorce, parental job loss, frequent moves, unsafe neighborhoods, and parental substance misuse or violence. Supportive and predictable childhood environments appear with nurturing parents, stable housing, and safer neighborhoods.

Bioecological theory describes environmental layers that interact with the child over time. Microsystems contain parents, extended family, teachers, and neighbors who interact frequently and reciprocally with the child. Exosystems include factors that affect the child indirectly, such as parental employment or frequent moves.

Prior studies linked parental disengagement, father absence, and neighborhood violence to short-term mating, early sexual debut, and sexually risky behavior in adolescence and adulthood. Harsh and unpredictable environments relate to present-oriented thinking and impulsivity, which then link to risky sexual behavior. Dark Triad traits, including psychopathy and Machiavellianism, support resource control, low self-control, and mating effort, along with sexually risky behavior.

Slower profiles are associated with traits such as long-term planning, agreeableness, conscientiousness, mental and physical health, well-being, self-esteem, positive affect, and emotional intelligence.

In the study, “Using SEM to test the associations among women’s childhood ecology, adult psychosocial life history traits, and mating effort,” published in Evolution and Human Behavior, researchers used structural equation modeling to investigate how childhood microsystem and exosystem experiences, adult psychosocial traits, and mating effort fit together in women.

Participants were 875 ethnically mixed, self-identified female undergraduate college students at a university in Northern California. Ages ranged from 18 to 46 years, with a mean age of 20.55. Questionnaires included childhood ecology, adult psychosocial traits, and mating effort along with Adverse Childhood Experiences (ACE) Inventory, a 20-item checklist of hardships in childhood.

Childhood trauma, control, and partners

Correlations between microsystem indicators, faster traits, and mating effort unpacked those paths. Higher microsystem scores related to higher ACE scores, paternal and maternal disengagement, more parental cohabitation, and higher neighborhood crime. Higher microsystem scores also related to higher, faster scores and Dark Triad psychopathy, higher coercive and power-seeking resource control, higher Machiavellianism, and lower self-control.

Psychopathy showed strong links to mating effort. Higher psychopathy correlated with more lifetime sexual partners, stronger short-term mating orientation, and greater future sexual risk intentions. Machiavellianism correlated with stronger short-term mating orientation and greater future sexual risk intentions.

Self-control scores related inversely to harsh childhood conditions. Higher ACE scores and more neighborhood crime correlated with lower self-control. Lower self-control correlated with more lifetime sexual partners, more openness to casual sex, and higher future sexual risk intentions.

Resource control strategies formed another bridge. Neighborhood crime correlated with higher coercive and power-seeking resource control. Higher coercive and power-seeking scores correlated with more partners, stronger short-term mating orientation, and higher future risk intentions.

Higher neuroticism correlated with higher ACE scores, more neighborhood crime, higher father disengagement, stronger short-term mating orientation, and greater future sexual risk intentions.

Sexual start patterns

Age at first sexual encounter could not be included in the structural equation model because a substantial number of participants reported no sexual experience. 284 women reported never having sex, leaving 591 with a reported age at sexual debut. A hierarchical multiple regression used microsystem, exosystem, slower traits, and faster traits as predictors of age at sexual debut in that subgroup.

Women who grew up in more chaotic or troubled family and neighborhood environments tended to start having sex at younger ages. Broader issues such as money instability and slower life history traits did not change that pattern much.

Women with higher adversity scores, more disengaged fathers, more non-relatives living in the home, higher psychopathy, a stronger drive to control others and resources, and higher neuroticism reported younger ages at first sex.

Mothers

A total of 33 participants reported having children. For this analysis, 37 participants without children were randomly sampled to form a comparison group of 70 women.

Microsystem and exosystem scores did not significantly predict having children. Slower and faster traits did. Women in this small subgroup who showed more of the slower life history traits (things like resilience, well-being, conscientiousness, secure attachment, and grit) were more likely to have children than women with lower scores on those traits.

Women in this small subgroup who had children differed from those without children mainly in their adult traits, not in their reported childhood environments. Mothers tended to score lower on fast life history traits such as psychopathy, low self-control, and power-seeking, and higher on slow traits, while the childhood microsystem and exosystem scores did not clearly separate mothers from non-mothers.

Life history patterns and implications

Psychosocial life history traits clustered into two partly independent sets, one faster and one slower. Faster traits were linked both to harsher childhood microsystems and to higher mating efforts, many of which are explicitly risky or short term. Slower traits were linked to kinder childhood microsystems, and showed statistically non-significant associations with mating effort.

Microsystem conditions before age 10 are linked more strongly to psychosocial traits and mating effort than exosystem conditions. Childhood trauma, parental disengagement, parental cohabitation with non-kin adults, and neighborhood crime all clustered within higher microsystem scores and connected to faster traits, more partners, shorter-term mating orientation, and higher future sexual risk intentions.

Perceived resource inconsistency in the exosystem added further associations with partner counts, short-term orientation, and risk intentions.

Conclusions, limitations and open questions

Results showed that the childhood microsystem had a far greater impact than the external environment on personality development and mating strategies. Good parenting and a protective early environment that allowed security and predictability showed a trajectory of positive associations.

The cohort included only college students, a major limitation for generalizing the findings, as college attendance is conceivably a measurable outcome of Life History theory. How the findings might compare to women who did not attend college, and how this socioeconomic cutoff might alter the strength of the associations is unclear.

News Staff by — December 03, 2025 By News Staff in Global News