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6 times when drug development got personal

While biopharma’s overarching mission is to develop innovative medicines to improve patient outcomes, for these six people, the motivation came from much closer to home.

In February, Doug Ingram dropped a bombshell on the industry: He would be stepping down from his post as CEO of Sarepta Therapeutics after a career spanning more than three decades.

Ingram’s difficult decision was driven by what he called a “fairly shocking and ironic twist of fate.”

“As you know, in late 2024 we entered into a partnership with Arrowhead, and we gained access to a number of very promising therapies, including SRP-1003, for a devastating disease, DM-1,” he said. Shortly after the deal, that same disease came knocking on Ingram’s door, afflicting two members of his immediate family.

Leaving Sarepta, Ingram said, will help him pour more of his focus at home.

By the end of the year, Sarepta will be under new management, though there has been no word on Ingram’s replacement yet.

Ingram’s situation is heart-breaking but by no means unheard of. In fact, biopharma is replete with examples of devastating diagnoses driving ardent drug development and entrepreneurship. It’s unclear yet where this personal bout with DM1 will take Ingram, but if he decides to return to biopharma and focus his efforts on this disease once again, there will be paths open to him, forged by many others who were steered to biopharma by personal motivations.

BioSpace looks at six such cases, where drug development was spurred by a mission far more individualized than profit and shareholder value or the benefit of entire patient communities.

‘Deeply personal’ investment adds to Quiver’s arrows

Around one in 4,000 children is born with a specific chromosomal aberration that will leave them stunted, both physically and developmentally. Babies with the disease, called Dup15q, may appear floppy and could have trouble feeding and be behind on key motor milestones. Kids with Dup15q also suffer from learning and intellectual disabilities.

In rare cases, Dup15q leads to sudden death. There is no approved treatment for this disease.

That’s where Quiver Bioscience comes in. Based in Massachusetts, the biotech is working on an antisense oligonucleotide that targets the UBE3A gene, heightened expression of which has been linked to Dup15q, the company said in a March 2025 news release. A precision approach to reduce UBE3A has “disease-modifying potential” for this condition, Quiver noted.

Last month, Quiver received a “targeted investment” from a family office in Argentina, specifically to help the company take its oligonucleotide program forward. But instead of wanting to see the money grow, this placement was much more personal: One of the family members also has Dup15q.

“As the father of a daughter living with this condition, this is not an ordinary investment — it is a deeply personal commitment to every family navigating this journey every single day,” José A. Porta, president of the family office, said in a prepared statement.

In an email to BioSpace, Quiver declined to provide additional details regarding the Porta family’s investment, including how much money the company received. The support, however, will allow the biotech to carry out preclinical activities for its antisense oligonucleotide, including candidate selection and safety assessments, opening the path toward clinical development, according to its April news announcement.

Parents launch Solid in search of cure for son

Solid Biosciences was founded in 2013 by husband-and-wife team Ilan and Annie Ganot. Their goal: Find a cure for their son Eytani, who had been diagnosed with Duchenne muscular dystrophy (DMD).

At the time of Solid’s establishment, there were no therapies for Duchenne on the market yet. The first Duchenne drug to win the FDA’s approval—Sarepta Therapeutics’ exon-skipper Exondys 51—was still three years away, and even then, it left a big portion of the patient population unserved. Exondys 51 is only indicated for patients who are amenable to exon 51 skipping, which represents around 13% of all patients with Duchenne.

Indeed, not long after their son was diagnosed, the Ganots realized that exon skipping therapies and those that boost dystrophin expression—a key deficiency in Duchenne—wouldn’t lead to meaningful and profound symptom improvements that parents like them would want for their kids, Ilan told Rare Disease Advisor in 2022.

The Ganots decided that only a gene therapy could give the relief they were looking for.

Solid is working toward this goal with SGT-003, which makes use of a genetic construct that expresses the microdystrophin protein, according to the company’s website. This could help compensate for the dystrophin deficiency in patients with Duchenne. To deliver the genetic payload, Solid is using a proprietary adeno-associated virus (AAV) vector that selectively targets cardiac and skeletal muscles while avoiding the liver—a profile that could lend the asset better efficacy and safety. Sarepta’s now-approved Duchenne gene therapy Elevidys was rocked last summer by patient deaths relating to liver complications from the AAV vector technology.

SGT-003 is being tested in two studies: the Phase 1/2 INSPIRE trial and the Phase 3 IMPACT study. INSPIRE has shown robust microdystrophin expression and early signs of muscle improvements. Solid is working with the FDA for a potential accelerated pathway for SGT-003. IMPACT is set to start this year.

Elpida born from parents’ quest to save son

As in the case of Solid, Elpida Therapeutics was born out of a couple’s desire to save their son.

In 2019, Terry and Georgia Pirovolakis were told that their son Michael had SPG50, a neurodegenerative and developmental disorder that, with fewer than 100 known cases worldwide, is classified as “ultra-rare.” There is no cure for SPG50, only treatments to manage its symptoms.

SPG50—also known as spastic paraplegia 50—is caused by mutations to the AP4M1 gene, which under healthy conditions encodes a part of a protein that helps move molecules along inside cells. Children must inherit an altered version of this gene from both of their parents for the disease to manifest, causing developmental delays, poorly formed muscles and lower-limb paralysis.

Searching for relief for their son Michael, the Pirovolakises decided that they needed to develop their own gene therapy, leading to the launch of Elpida in May 2023. The company’s lead program is MELPIDA, which delivers a functioning copy of the AP4M1 gene.

Michael became the first-ever patient to receive MELPIDA in 2022, around three years after he was diagnosed—and the results were promising. At about a year after receiving the gene therapy, Michael showed improvements in limb spasticity and achieved notable development and motor gains, according to a June 2024 publication in Nature Medicine.

MELPIDA was also largely safe for Michael, and the gene therapy was able to keep SPG50 at bay, preventing disease progression. The asset is in Phase 3 development, according to Elpida’s website.

CEO-Dad enters biotech to develop treatment for kids

Joining the roster of parents going into biopharma to save their kids is John Crowley—and of those that made it to this list, he might be the most well known and his fight the most documented. Crowley’s story even became the backbone for the 2010 film Extraordinary Measures, starring Harrison Ford and Brendan Fraser.

In 2000, Crowley helped establish Novazyme Pharmaceuticals with the goal of developing a treatment for Pompe disease, a rare metabolic disorder that afflicted two of his children. Patients with Pompe suffer from muscle weakness and have enlarged hearts and livers, often leading to other complications. Left unchecked, the condition can leave children debilitated or lead to dead.

Indeed, the prognosis for Crowley’s kids was dire, according to reporting by Rare Disease Advisor last month: They wouldn’t survive past two years.

Roughly a year into its operations, Novazyme got acquired by Genzyme for $225 million. Despite being a hefty vote of confidence for his mission, this deal posed a crucial conundrum for Crowley: Being an employee, he couldn’t enroll his kids into the company’s clinical trial, Fierce Pharma reported last year.

But Crowley would not be stopped and ultimately stepped down from Genzyme. His gamble worked—as did the investigational therapy. His children were enrolled in the trial and experienced improved muscle strength and normalized heart sizes.

Today, this therapy is marketed under the brand name Lumizyme. Sanofi owns the rights to it after a 2011 deal with Genzyme worth $20 billion.

Crowley, meanwhile, has gone on to establish Amicus Therapeutics, still keeping his sights set on advancing therapies for rare diseases. After a $4.8 billion acquisition in December, Amicus today operates as a subsidiary of BioMarin.

Crowley is also currently serving as CEO of the Biotechnology Innovation Organization (BIO) after rising as an executive leader in biotech.

Mom launches EveryONE Medicines to honor daughter’s life

A parent’s perseverance sometimes ends in heartbreak.

That’s the case for EveryONE Medicines, which closed up shop in March. After cofounding the company in early 2021 with the goal of developing individualized precision treatments for patients with rare diseases, Julia Vitarello publicly debuted EveryONE in 2024.

EveryONE’s mission is deeply personal to Vitarello. Her daughter, Mila, suffered from Batten disease, an umbrella term that refers to a group of genetic disorders involving the toxic buildup of waste in a child’s brain cells. Symptoms include vision loss, muscle weakness and seizures, alongside developmental and cognitive problems.

Batten disease is fatal and often leads to death early in a patient’s life.

Vitarello worked hard to avert this fate for her daughter and enlisted researchers at the Boston Children’s Hospital to come up with a cure, C&EN reported in 2024. This effort resulted in an antisense oligonucleotide which they called milasen, designed to address the specific genetic mutation driving Mila’s condition.

The treatment helped improve Mila’s condition, reducing her seizures—but it wasn’t enough to save her. Mila was diagnosed at age 6 and died at 10.

Today, despite EveryONE folding, Vitarello remains involved in rare disease development. She is the chairwoman and CEO of Mila’s Miracle Foundation, a non-profit that works with academics, government agencies and companies to foster the development of individualized medicines. Vitarello also serves as a steering committee member of the N=1 Collaborative, a network of organizations and experts advancing personalized treatments.

Cure Rare Disease honors founder’s late brother

Capping off this list is Cure Rare Disease, a non-profit company that works to develop therapies for patients who had been “previously deemed untreatable,” according to its website.

The group was established by Rich Horgan with the goal of developing a treatment for his brother, Terry, who had been diagnosed with Duchenne muscular dystrophy. The group got very close to their goal and in October 2022 they were able to dose Terry with a CRISPR-based gene therapy made specifically for him to address the rare genetic mutation that drove his condition.

A month later, however, Terry died. Cure Rare Disease suggested on its website that the team was just too late, noting that Terry’s death “would have been avoided had the treatment been accessible sooner.” Research since has however pointed the finger at the viral vector used to deliver the gene therapy, which triggered a powerful immune reaction.

Regardless of the cause of his brother’s death, Rich Horgan has continued his mission of developing therapies for other rare diseases. Alongside Duchenne, and in collaboration with researchers, policy experts and other foundations, Cure Rare Disease is also working on programs for limb-girdle muscular dystrophy, spinocerebellar ataxia and adenylosuccinate synthase 1-related myopathy.

News Staff by — June 16, 2026 By News Staff in Bio Space
Germany rethinking drug price reforms after Lilly, Boehringer withdraw investments: Reuters

Weeks after Boehringer Ingelheim and Eli Lilly retracted billions of dollar in German commitments, the nation’s government is reportedly changing a contentious element of its planned healthcare reforms.

The German government is dropping plans to introduce variable discounts on medicines after receiving pushback from industry, Reuters reported Monday.

An anonymous government source told the publication the government will replace variable discounts with fixed reductions to help drugmakers plan for the shift. The original proposal tied the discount rate to Germany’s overall spending on medicines and to healthcare revenues, limiting visibility into the figure. Details of the size of the new, fixed discounts have yet to emerge.

The report comes less than two weeks after Boehringer Ingelheim and Eli Lilly pulled back from planned investments in Germany in response to the government’s proposed healthcare reforms. BioSpace did not receive an immediate response from Lilly to questions about whether the company will reconsider its actions in light of the government’s proposed move to a fixed discount rate. Boehringer, meanwhile, replied that it has “no comment” to the same questions.

Hundreds of jobs and billions of euros rest on the outcomes of the companies’ decisions. At Boehringer, concerns over the proposed German reforms led management to ax plans to invest €900 million ($1 billion) from 2027 to 2030 to expand its infrastructure in the country.

On the day Boehringer disclosed its investment U-turn, Lilly CEO Dave Ricks revealed that his company was halving a planned investment in a German plant. Lilly originally intended to invest €2.3 billion ($2.7 billion) in a German injectable manufacturing site to support the supply of its GLP-1 drugs Zepbound and Mounjaro.

The healthcare reforms drove Lilly to scale back its investment. Under the revised plan, Lilly will open the site as planned next year but run the plant at half the intended capacity. The change will involve a halving of the site’s planned headcount, which was originally 1,000. Lilly’s revised plan reflects Ricks’ belief that Germany’s reforms would make it the least supportive country in Europe.

Last week, Pfizer CEO Albert Bourla applied further pressure to the German government. Bourla told the government that Pfizer was reviewing its “external engagements as well as the timing, scope and future prioritization ⁠of certain planned investments in Germany,” Reuters reported.

The series of actions by drugmakers echoes the industry’s response to a drug pricing and market access dispute in the U.K. The long-running argument escalated in September when Merck pulled out of a $1.3 billion project and AstraZeneca paused plans to invest around $270 million in an R&D site. The U.K. later acceded to some of the industry’s requests in its trade deal with the U.S.

News Staff by — June 16, 2026 By News Staff in Bio Space
This high-fat eating plan may offer a powerful way to shield the aging brain

The gut and brain are in constant conversation through a powerful biochemical signaling pathway. This two-way connection allows them to exchange signals that influence everything from digestion to emotional health, and studies suggest even the fate of neurodegenerative diseases.

A new review investigated whether a high-fat, low-carb way of eating, also known as the ketogenic or keto diet, could protect the brain from devastating diseases like Alzheimer’s, Parkinson’s, multiple sclerosis and ALS.

Screening findings published in the past 15 years revealed that the diet that has helped so many people lose weight also offers a promising way to prevent or treat diseases that damage the brain over time by improving how the body and brain use energy. The findings are published in Translational Neurodegeneration.

Cells typically rely on sugar, or glucose, for energy, but in people with neurodegenerative diseases, brain cells often struggle to use it effectively. A keto diet, which is rich in fat and low in carbohydrates, shifts the body from glucose metabolism to fat metabolism, prompting the liver to produce ketone bodies—including acetoacetate (AcAc), beta-hydroxybutyrate (BHB) and acetone—from fatty acids.

These ketone bodies serve as an alternative energy source for the brain, ensuring that protective and repair processes within nerve cells have enough fuel to function properly. The keto diet can also bring about noticeable changes in the gut microbiome. It reduces inflammation-associated bacteria while supporting microbes linked to better gut barrier health.

Ketones protecting the brain

Alzheimer’s, Parkinson’s, Huntington’s, ALS and multiple sclerosis are neurodegenerative diseases whose symptoms might be very different, but they share several underlying mechanisms in which nerve cells in the brain and spinal cord slowly break down and die.

Mitochondrial dysfunction, in which the cell’s energy-producing machinery fails and reduces energy output, can be a key driver of nerve cell death and the buildup of harmful molecules that damage cells. Inflammation in the brain adds to this problem.

In 1921, Russell Morse Wilder, an American physician, designed the keto diet for the treatment of drug-resistant epilepsy in children. Over the decades, it gradually gained the attention of researchers studying progressive brain-related diseases. While current treatments can ease the symptoms of neurodegenerative diseases, they rarely address the underlying loss of nerve cells.

The ketogenic diet has emerged as a promising area of research, yet much of the evidence comes from animal studies and small clinical trials. Drawing on both biological research and clinical findings, this review examines the potential of the diet in real-world settings.

The review found that the keto diet may do more than provide the brain with an alternative energy source. It appears to activate autophagy, the body’s natural cellular cleanup system, helping to remove damaged cellular components and toxic protein buildup linked to brain decline. It may also reduce oxidative stress and calm chronic inflammation, both of which contribute to nerve cell damage.

People with Alzheimer’s disease experienced improvements in memory, daily functioning and quality of life without serious adverse effects. On the keto diet, patients with Parkinson’s disease experienced higher energy levels, less fatigue and improved motor function. Positive benefits were also reported in other neurodegenerative diseases.

However, sticking to the diet can be challenging. Many people struggle to maintain it in the long term, and some drop out of their studies because of its restrictive nature. Some also experience headaches, fatigue, nausea and dizziness when starting out, also known as the keto flu.

The researchers highlight that the keto diet is gaining ground as a promising approach for neurodegenerative diseases, thanks to its wide-ranging effects on the biological processes that drive these conditions. While preclinical results show promise, major questions remain about its long-term effects, safety and practical utility for patients in clinical settings.

News Staff by — June 15, 2026 By News Staff in Global News
Hidden switch lets two of four receptor subunits open brain ion channel

To transmit excitatory signals, nerve cells mostly use glutamate as a neurotransmitter. To detect these transmitter signals, the cells can rely on a whole repertoire of receptors with different signaling properties. Researchers at the Chair of Cellular Neurobiology, led by Professor Andreas Reiner at Ruhr University Bochum, Germany, together with collaboration partners in New York (Department of Biochemistry and Biophysics, Weill Cornell Medicine), investigated the function of a specific glutamate receptor complex and made some surprising observations. Their findings were reported in the journal Nature Communications on April 24, 2026.

Composed of different subunits

The structure of ionotropic glutamate receptors (iGluRs), which function as glutamate-activated ion channels in the membrane of neurons, has been known for many years. All iGluRs consist of four subunits that form a shared ion channel pore. Each subunit has a glutamate binding site. However, how glutamate binding affects individual subunits and how the subunits act together to cause the opening and closing of their common pore were largely unknown. The research team investigated this mechanism in a special glutamate receptor complex, the so-called GluK2/GluK5 kainate receptor heteromer, which consists of two GluK2 and two GluK5 subunits.

One initial observation was that ligand binding at just the two GluK5 subunits is sufficient to cause receptor activation. Using fast patch-clamp measurements, Laura Moreno Wasielewski, one of the study’s first authors, was able to show that 5-iodowillardiine, an agonist that binds only at the two GluK5 subunits, puts the receptors into a permanently open state.

“This is remarkable,” explains Wasielewski, “since it had been assumed that only the GluK2 subunits may mediate activation, as they are more closely coupled to the ion channel pore.”

Structural biology studies show details

Cryo-electron microscopy studies conducted in the laboratory of Professor Joshua Levitz in the United States provided further insights into the gating mechanism of this receptor complex. The structures revealed that ligand binding at the GluK5 subunits causes movement in the adjacent GluK2 subunits.

“This was unexpected; however, it explains why the GluK5 subunits are able to open the channel pore, although they are structurally less favorably positioned to do so,” Reiner summarizes.

The structures also confirmed that partial occupancy of the four subunits, which is sufficient to cause receptor activation, does not yet elicit the extensive restructuring responsible for the subsequent inactivation (desensitization) of the receptors. The latter is observed only when all four subunits are occupied.

The structures also revealed another surprising detail: A close interaction between the opposing GluK5 subunits was observed, a unique feature not seen in other kainate or related AMPA receptor complexes. In accompanying patch-clamp measurements, the researchers found that this interaction also plays an important functional role.

“This interaction site appears to affect the unusually slow deactivation that is seen for GluK2/GluK5 receptors, which is around 10 times slower than in other kainate receptors,” states Wasielewski.

Function in the nervous system

How the receptor’s unusual properties contribute to neuronal function remains to be investigated. The GluK2/GluK5 receptor complex is known to primarily exert a modulatory influence on synapses. This may also make the receptor an interesting target for therapeutic purposes, especially since it appears to be the most common kainate receptor in the human brain.

Since GluK2 and GluK5 subunits have different affinities for glutamate, the partially occupied states investigated in this study could be of actual physiological significance, as they could cause long-lasting, non-desensitizing currents, which are rather unusual.

“So far, it is also unclear to which extent the slow deactivation of this receptor heteromer contributes to synaptic signals. The GluK5-GluK5 interactions we have identified here now give us the possibility to address this experimentally,” explains Reiner.

The obtained structural information could also enable the future development of specific drugs tailored to this particular receptor.

News Staff by — June 15, 2026 By News Staff in Global News
Engineering enzymes with potential against ALS and Parkinson’s disease

In an advance that could one day lead to new treatments for neurodegenerative diseases, Meredith Jackrel, an associate professor of chemistry in Arts & Sciences at Washington University in St. Louis, and her team have developed a method to rapidly produce and screen a class of disaggregase enzymes that can break down the misfolded proteins associated with ALS and Parkinson’s disease.

“Disaggregases have a lot of promise, but previous methods for producing and identifying them were extremely slow and tedious,” Jackrel said. “Our new method is a significant step forward.”

The study was published in the journal Molecular Cell. The lead author is Jeremy Ryan, Ph.D. ’23, a former graduate student in Jackrel’s lab who is now a scientist at Bayer. Other co-authors include postdoctoral researcher Anuradhika Puri; staff scientist Macy Sprunger, Ph.D. ’23; graduate students Karlie Miller, Madalyn Bochantine and April Lopez; and several former undergraduate students.

Why Hsp104 matters

Jackrel and her team focused on Hsp104, a disaggregase enzyme naturally found in yeast. The enzyme is known as a disaggregase because it can break apart protein aggregates. Yeast uses the enzyme for protection from heat and other stresses, but it also has the power to dissolve proteins, including TDP-43, a misfolded protein that clumps in the nervous systems of people with ALS, and α-synuclein, which accumulates in Parkinson’s patients.

“Not only does Hsp104 break down the misfolded proteins, but it can also help them refold, which can restore healthy cell functions,” Jackrel said.

Using an engineering strategy, the team produced a library in which they introduced many different mutations into a region of Hsp104 and then introduced this library into yeast. In this way, each yeast cell makes a different version of Hsp104. Some varieties are more potent than others, but previous methods for identifying versions of Hsp104 with optimal features were tedious and impractical.

A faster screening method

“You can grow yeast colonies on a plate and pick them up with a toothpick,” Jackrel said. Researchers can then use DNA sequencing to identify promising versions of Hsp104, but it’s a painstaking process that allows them to analyze at most only a few hundred versions at a time.

As described in the new paper, Jackrel and her team have found a way to rapidly produce, identify and sort the versions of Hsp104 that can break down misfolded TDP-43 or α-synuclein.

“We start with the wild-type Hsp104 gene, and then we introduce mutations to create tens of millions of variations,” Jackrel said. “We’re building a vast library of possibilities.”

The team then used deep sequencing, a highly sensitive process that sequences many fragments of DNA at the same time, to identify mutations.

“We can essentially look at the entire population at once and see which versions of Hsp104 work well in some situations and not in others,” she said.

Not every version of Hsp104 in the new library will be more effective than previously known varieties, but the new system allows researchers to analyze many more versions of Hsp104 at once, making it much easier to create and search for new and improved disaggregases, Jackrel said. The new versions that emerged from this process have several characteristics that make them more promising than earlier versions of Hsp104.

Potential impact on disease

TDP-43 is already a top target for scientists and pharmaceutical companies. In addition to playing a fundamental role in ALS, it is also associated with dementia, including some forms of Alzheimer’s disease. So far, efforts to develop drugs that clear the protein or slow disease progression have been unsuccessful.

It will likely take years of further experiments and fine-tuning before Hsp104 could be considered a potential therapy for ALS, Jackrel said.

“We know that buildups of misfolded TDP-43 and α-synuclein are important in the development of neurodegenerative disease, so anything that can reverse that buildup could be helpful,” Jackrel said. “Hsp104 could be part of the answer, so this is a major accomplishment for our team.”

News Staff by — June 15, 2026 By News Staff in Biotechnology Today
Could leaves help feed humanity after disaster?

UC researchers are investigating whether leaf protein and sugar extracted from plant fiber could help sustain people if major global shocks disrupt food production. Te Whare Wānanga o Waitaha | University of Canterbury (UC) Associate Professor David Denkenberger, who has spent more than a decade studying food resilience in extreme scenarios, says the work is part of a broader effort to identify practical ways to prevent mass starvation if global food systems are severely disrupted.

The research investigates how leaves from crops and forage plants can be processed into leaf protein concentrate for human consumption, while part of the remaining fiber can be converted into sugar. The idea is to make better use of plant material that is typically not eaten by people, potentially increasing the amount of food produced from existing land.

Associate Professor Denkenberger says the research matters because major global shocks, while rare, could disrupt the food systems people rely on every day. “For about 15 years, we’ve been looking at how to build resilience to global catastrophes.

“These catastrophes could include things like a large volcanic eruption that blocks sunlight, or an extreme solar storm that disrupts electricity. This leaf protein work is one of a suite of interventions that could help feed people in those kinds of scenarios.”

The study sits within a wider body of research into resilient food systems, including work on greenhouses, alternative proteins, seaweed, and microbes.

While the leaf protein research is designed with worst-case disasters in mind, Professor Denkenberger says it could also have more immediate benefits. “There is also potential for near-term applications to make food systems more sustainable,” he says.

One possible use is in conventional cropping systems. For example, a crop such as wheat could provide its usual grain harvest, while its leaves could also be processed to extract protein. The leftover fiber could then be turned into sugar, creating more food from the same amount of land already being farmed.

Another possible application is on grazing land. Plants such as alfalfa or red clover are not directly edible for humans, but they could potentially be processed to produce human food ingredients.

The research aligns with growing international interest in food security, disaster preparedness and how to build more resilient systems in the face of global shocks.

Associate Professor Denkenberger began working in this area in 2011 and later wrote the book “Feeding Everyone No Matter What,” which examines alternative food sources for global catastrophes. In 2017, he also helped establish the nonprofit Alliance to Feed the Earth in Disasters (ALLFED), whose work spans scientific research, policy engagement and practical implementation, to help build food system resilience and make food accessible to everyone in the event of global catastrophic food system failure. As the research progresses, there may also be opportunities for public involvement through citizen science.

For Associate Professor Denkenberger, the goal is both ambitious and practical: to find realistic ways to keep people fed when normal systems fail.

“We’re looking at different options that could be used if conventional food production were disrupted,” he says. “Leaf protein is one of many possibilities, but it could play an important role in improving resilience.”

News Staff by — June 15, 2026 By News Staff in Biotechnology Today
Amgen shores up Tavneos’ FDA defense with Duke data analysis

After the FDA flagged patient deaths linked to Amgen’s rare disease drug Tavneos and called for its voluntary removal, the pharma recruited an independent data analysis from Duke researchers to help build the case for the drug’s continued market approval.

Amgen has requested a hearing with the FDA over the agency’s insistence that Amgen pull its rare inflammatory disease drug Tavneos from the market—and this time, the company is bringing in help from the Duke Clinical Research Institute.

The pharma has asked Duke researchers to conduct an “independent and fully blinded re-adjudication” of data from the ADVOCATE trial, according to a June 1 letter to the FDA, which was posted online on Thursday. ADVOCATE is the Phase 3 study that Tavneos’ original developer ChemoCentryx used to support its approval in ANCA-associated vasculitis in October 2021. Amgen acquired ChemoCentryx for $4 billion just under a year later.

“Withdrawal of approval of TAVNEOS would be neither in the best interest of patients nor consistent with the statutory criteria,” Jay Bradner, Amgen’s executive vice president for R&D, wrote in the company’s letter to the FDA. Duke’s re-adjudication of ADVOCATE data began in February 2026 and the pharma plans to submit results, alongside other “detailed data,” to the FDA by June 29.

Alongside the Duke analysis, Amgen has listed in its letter more than a dozen ongoing or recently completed studies to support Tavneos’ overall benefit-risk profile.

The FDA first called for the voluntary withdrawal of Tavneos in January, raising concerns about “the process followed by ChemoCentryx” to evaluate the primary endpoints in ADVOCATE for nine of the 331 patients enrolled, according to an Amgen release in February. The regulator also pointed to liver toxicities in the context of Tavneos’ overall risk-benefit profile.

Amgen refused the regulator’s request, leaving Tavneos on the market.

The agency continued to apply pressure, however, and in late March put out a safety alert flagging eight deaths and 76 cases of drug-induced liver injury (DILI) that have “reasonable evidence of a causal association with Tavneos.” Seven of the 76 cases involved biopsy-confirmed vanishing bile duct syndrome (VBS), all of which necessitated hospitalization and three of which contributed to the total tally of eight deaths.

While the FDA at the time conceded that liver toxicities have been “identified in premarket clinical trials and described in product labelling,” potentially fatal DILI and VBS “represent new safety concerns.”

Last month, Japan’s Kissei Pharmaceutical, which is Amgen’s partner in charge of Tavneos’ Japanese distribution, said that 20 patients on the drug have died since the product launched in 2022. Most of the deaths were attributed to DILI and VBS, though Kissei took care to say that it remains unclear if Tavneos was the direct cause of the fatalities.

The European Medicines Agency has also launched a review of Tavneos, similarly flagging “questions regarding the data integrity” of ADVOCATE, according to a January release.

News Staff by — June 15, 2026 By News Staff in Bio Space
Novartis’ $12B Avidity buy pays dividends with Phase 1/2 muscular dystrophy win

The RNA-based medicine is one of a handful of antibody-oligonucleotide conjugates that Novartis acquired last October when it took over neuromuscular-focused Avidity Biosciences.

Novartis’ $12 billion gambit for Avidity Biosciences late last year is starting to pay off, with the pharma reporting promising biomarker findings for its investigational RNA medicine in patients with facioscapulohumeral muscular dystrophy.

In the Phase 1/2 FORTITUDE study, Novartis enrolled 90 patients to receive the antibody-oligonucleotide conjugate delpacibart braxlosiran (del-bax) or placebo. Aside from safety, the trial’s primary outcome is the treatment effect on KHDC1L, a plasma disease biomarker for facioscapulohumeral muscular dystrophy (FSHD).

Without disclosing specific data, Novartis in a Thursday news release said that FORTITUDE’s biomarker cohort met its primary and key secondary endpoints. KHDC1L levels were reduced in patients treated with del-brax, suggesting “strong target engagement,” the pharma noted.

Del-brax also elicited a drop in creatine kinase concentrations, which Novartis explained is indicative of “reduction in muscle damage” in treated patients.

These findings “replicate the target engagement and downstream muscle protection seen with del-brax in earlier dose-escalation cohorts,” Nazem Atassi, global head of neuroscience and gene therapy development at Novartis, said in a prepared statement.

A previous FORTITUDE readout covering earlier dosing cohorts showed that patients on del-brax improved on the 10-meter walk-run test at 12 months compared to placebo. Similarly, those given del-brax performed better on the timed up-and-go test as well as on quantitative muscle and upper limb function assessments at 12 months while placebo controls deteriorated.

“We are now evaluating the totality of the biomarker and clinical data and look forward to discussions with global regulatory agencies,” Atassi said on Thursday, though Novartis hasn’t yet specified filing plans for the asset. The pharma, meanwhile, has launched the Phase 3 FORTITUDE-3 trial of del-brax in FSHD, aiming to enroll around 200 patients and looking at the therapy’s effects on muscle performance.

FSHD is a rare and progressive neuromuscular disease that manifests as a life-long muscle wasting, pain, fatigue and disability. The condition, affecting some 45,000 to 87,000 people across the U.S. and Europe, is caused by the abnormal expression of the DUX4 protein, which in turn activates genes that are toxic to muscles. Del-brax addresses this disease mechanism by combining the specificity of antibodies and the precision of oligonucleotides to target and suppress the expression of DUX4, according to Avidity’s website.

The asset came to Novartis through the pharma’s $12 billion takeover of Avidity in October last year, alongside two other RNA-based therapies: del-zota, being proposed for Duchenne muscular dystrophy, and del-desiran, being trialed for myotonic dystrophy type 1. The pharma also won over a clutch of preclinical programs for other rare neuromuscular disorders.

Novartis closed the Avidity acquisition in February.

News Staff by — June 15, 2026 By News Staff in Bio Space
A higher-dose flu shot could spare millions of older adults a hospital stay

Influenza is a seasonal condition that causes coughing, sneezing, mild fever and aches in most cases. However, it can sometimes take a serious turn, leading to hospitalization, especially for young children, adults over 65 and pregnant people. A recent study published in JAMA Network Open examined whether the high-dose inactivated influenza vaccine (HD-IIV), which contains four times as much antigen as the standard dose, offers superior protection against hospitalization and death.

Researchers analyzed data from eight large-scale clinical trials involving more than 600,000 participants that compared a high-dose flu shot with the standard flu shot in older adults.

The high-dose vaccine provided substantially greater protection, reducing the risk of flu-related hospitalization by 38.5% and hospitalization for laboratory-confirmed influenza by 31.2%. While the high-dose shot kept more people out of the hospital, it did not show a significant difference in preventing deaths compared with the standard shot.

Higher dose better?

It takes most people less than two weeks to recover from the flu, but the illness does not always end there. For some, the flu can lead to complications that prolong recovery or become serious enough to require hospitalization. Moderate complications, such as sinus and ear infections, can extend the recovery period.

In contrast, severe complications, such as pneumonia, can trigger other health problems, including inflammation of the heart and brain, as well as respiratory and kidney failure.

A standard flu shot helps reduce the risk of getting sick and protects against many of the flu’s potentially serious complications, but some populations need an extra boost. This led to the development of a high-dose flu vaccine (HD-IIV) for older adults, which contains 60 μg per strain compared with 15 μg in the standard dose.

This higher dose was designed to generate a stronger immune response in older adults, who often respond less effectively to the standard flu shot and are at greater risk of severe flu-related complications.

Earlier work showed the high-dose shot prevents more lab-confirmed flu infections than the standard dose. To shape flu vaccination guidelines for older adults, policymakers need evidence on whether high-dose flu vaccines meaningfully reduce hospitalizations and severe illness, not just mild infections.

A large study, FLUNITY-HD, combined data from two major trials—DANFLU-2 and GALFLU—and found that the high-dose flu vaccine was more effective than the standard vaccine at preventing flu-related hospitalizations.

In this study, researchers brought together findings from FLUNITY-HD and other randomized clinical trials to provide a clear picture of how high-dose and standard-dose flu vaccines compare in reducing hospitalizations and deaths among older adults.

These studies provided data on a large group of 605,098 participants across North America and Western Europe, including older adults living in their own communities and in nursing homes, as well as those with heart conditions.

Older adults who received the high-dose vaccine were less likely to be hospitalized for the flu and to have a lab-confirmed case of the flu compared with those who received the standard dose. The high-dose shot reduced hospitalizations for pneumonia or the flu by 11.5%, heart and lung problems by 7.5%, and all-cause hospitalizations by about 3%. The vaccine’s benefits held up across all age groups, including people over 80, and in those both with and without heart disease.

The findings show that for older adults, the choice of flu shot can make a real difference in their risk of hospitalization due to influenza complications. Public health planners can factor these results into strategies to protect health systems from being overburdened during flu season.

News Staff by — June 13, 2026 By News Staff in Global News
Our brains may be automatically filtering out negative words

We tend to assume that emotionally charged words are more likely to grab our attention. An insult shouted across a crowded room or a disturbing phrase overheard on television can seem impossible to ignore. But a new study published in Psychological Science suggests the opposite may happen before words reach conscious awareness.

Researchers at the Hebrew University of Jerusalem found that when people were focused on a visual task, they were less likely to consciously notice negative spoken words than neutral ones. The findings offer new insight into how the brain determines which information enters conscious awareness and which remains outside it.

“This study is a nice example of how our conscious intuitions regarding what we notice are not always what our unconscious is doing,” said lead author Gal R. Chen, a doctoral candidate in psychology at the Hebrew University of Jerusalem.

What reaches conscious awareness

Although much of the brain’s processing occurs outside conscious awareness, scientists know little about how information is selected to enter consciousness, particularly in hearing. Insights into this process could explain how nonconscious information might influence an individual’s thoughts, feelings and behavior.

Much of what scientists know about nonconscious processing comes from studies of vision in which researchers briefly flash images that participants are unable to consciously report seeing. Speech, however, presents a different challenge because spoken words, unlike images, cannot be delivered in a split second. Researchers have therefore struggled to determine how much information the brain can process from spoken language before a person becomes aware of it.

Chen and his colleagues set out to examine whether the emotional meaning of spoken words influences their chances of reaching awareness when people are focused on another task.

How the experiment worked

In the study, 101 Hebrew-speaking adults were instructed to identify whether a figurine on a screen was identical to the one before it while listening to a stream of meaningless pseudowords. Occasionally, a real Hebrew word, either emotionally negative or emotionally neutral, was inserted into the audio stream. After hearing the word, participants were asked whether they had noticed it and completed additional tests designed to measure their awareness.

“We assumed initially that people would notice the negative stuff more because that is our conscious intuition,” Chen said. “There is a lot of data showing that when you see or hear something negative, you slow down or make more mistakes.”

Instead, the opposite happened: Participants were more likely to notice the neutral words over the negative words.

“We thought it was a mistake,” Chen said. “So we repeated the study while adding new words. The results gave us the same trend: People notice negative words less.”

The effect persisted when the researchers repeated the experiment with the same visual task but a larger set of words. To examine whether the observation was specific to conditions of high effort, the researchers conducted the experiment again, but this time replaced the demanding visual task with a much easier one. Again, participants were more likely to notice neutral words over negative ones.

Why negative words may fade

One possible explanation for this observation, the researchers said, is that consciously experiencing negative information is costly, and the cognitive system sometimes opts not to pay this price.

“It may be the default of the unconscious mind to suppress information that may be harmful to us,” Chen said. “If your primary task is to talk to me, random words popping up are not helpful. And if these words slow you down, the default unconscious bias might be, ‘don’t bring them around.'”

The findings may offer new avenues for studying mental health conditions. Chen speculates that future research could investigate whether the same unconscious filtering process operates differently in people with anxiety disorders, phobias or post-traumatic stress disorder.

“The normal population notices negative words less often compared to neutral words,” Chen said. “In a clinical population, they might not have this selection bias.”

“If you think of the unconscious as a gatekeeper guarding us against things that may harm us or influence our decisions, you might ask what happens if this gatekeeper screws up,” he added.

Limits and future questions

Chen noted that the study has limitations. For example, it examined single words rather than conversations or natural speech, and it did not test highly positive or taboo words, which could produce different results. He said future research could explore whether the same effects appear in sentences, stories and more realistic listening environments.

For now, he said, the findings suggest that the nonconscious mind may play a larger role in shaping our everyday experiences than we realize.

News Staff by — June 13, 2026 By News Staff in Global News