Tag: Biotech

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Oral Small-Molecule GLP-1s Linked to Deep Brain Activity and Reduced Cravings in Mice

Interest in glucagon-like peptide 1 receptor agonists (GLP-1s) continues to surge due to their effectiveness in reducing body weight and improving metabolic outcomes. This includes interest in small molecule oral GLP-1s which are more bioavailable and more easily manufactured than their injectable counterparts.

Now data from a new study in mice performed by scientists at the University of Virginia shows that this emerging class of weight-loss drugs suppress hedonic eating by modulating a reward circuit deep in the brain that is separate from previously described mechanisms that broadly affect appetite. The scientists believe that this pathway could be an avenue by which GLP-1s treat other dysfunctions in reward processing such as substance use disorders.

Details of the National Institutes of Health-funded study were published this week in a Nature paper titled “A brain reward circuit inhibited by next-generation weight-loss drugs in mice.” In it, the team reported that they investigated the small-molecule GLP-1s including Eli Lilly’s recently approved drug orforglipron, also known by the brand name Foundayo, as well as danuglipron, an oral GLP-1 that was being developed by Pfizer until the company decided to discontinue its development in 2025. 

Previous studies that explored the effects of larger peptide GLP-1s such as semaglutide in the brain have found that they suppress hunger-driven eating by engaging networks in the hypothalamus and hindbrain. What has been less clear is the mechanism by which small-molecule GLP-1s work. “As the accessibility of these medications continues to rise and patient uptake increases, it’s crucial that we understand the neural mechanisms underlying the effects we’re seeing,” said Lorenzo Leggio, MD, PhD, clinical director of NIH’s National Institute on Drug Abuse.

The current study gets scientists one step closer to that goal. According to the paper, the scientists first used gene editing to modify the GLP-1 receptors of mice to make them more humanlike. They then administered orforglipron or danuglipron to the mice, and identified brain regions where the drugs induced activity. The results showed that in addition to inducing activity in familiar pathways, the drugs also triggered the central amygdala, a region associated with desire that is deeper in the brain than scientists previously thought GLP-1s could directly reach. Further testing showed that once activated, the central amygdala reduced the release of dopamine into key hubs of the brain’s reward circuitry during hedonic feeding. 

“We’ve known that GLP-1 drugs suppress feeding behavior driven by energy demand,” said co-corresponding author Ali Guler, PhD, a professor of biology at the University of Virginia. “Now it seems oral small-molecule GLP-1s also dial back eating for pleasure by engaging a brain reward circuit.”

Given the effect of these drugs on eating for pleasure, future studies could explore whether small-molecule GLP-1s can also suppress cravings for other addictive substances. It is a question that the team hopes to explore in follow up studies focused specifically on substance use disorder. 

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Seaport’s IPO adventure, obesity pill battles, and Makary’s troubles

On this week’s episode of “The Readout LOUD,” we chat with Seaport Therapeutics CEO Daphne Zohar, fresh off the biotech’s successful IPO. Plus, Elaine, Allison, and Adam chat about this week’s notable news, including the obesity pill battle between Eli Lilly and Novo Nordisk, a Phase 3 study win for Cytokinetics, and FDA Commissioner Marty Makary’s White House troubles. 

Oh, by the way, this is the 400th episode of your favorite biotech podcast. 

Read the rest…

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STAT+: FDA revisits a rare cancer treatment it rejected a few months ago

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

The Trump administration is touting massive projected savings from its still-secret drug pricing deals, though outside experts say there’s little way to independently verify the math.

Also, FDA leadership is facing fresh accusations of political interference in drug reviews, AI oncology startups are trying to make cancer care virtual, and scientists are opening up an entirely new frontier of biology hidden inside the “dark proteome.”

Continue to STAT+ to read the full story…

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Bayer to Acquire Perfuse for up to $2.45B, Seeing Ophthalmology Opportunity

Bayer has agreed to acquire Perfuse Therapeutics for up to $2.45 billion, the companies said, in a deal designed to broaden the buyer’s ophthalmology pipeline with Perfuse’s sole pipeline drug and two clinical phase programs for eye disorders.

Perfuse’s PER-001 is a small molecule endothelin receptor antagonist being developed for the treatment of ophthalmic diseases. Two of PER-001’s four programs are in Phase II development: One designed to treat open-angle glaucoma by improving the visual field for patients, and the other designed to treat diabetic retinopathy (DR) by improving contrast sensitivity and reducing ischemia in patients with the disorder.

Last year, Perfuse announced positive results from two Phase II clinical trials evaluating PER-001.

One was a Phase IIa trial (NCT05822245) assessing PER-001 in glaucoma, which showed that six months after a single intravitreal administration of PER-00, added to existing standard-of-care intraocular pressure (IOP)-reducing therapies, 22.2% of low-dose and 37.5% of high-dose patients experienced ≥7 decibel (dB) improvement in a pre-defined retina region of minimal five test points compared to 0% in control in six months.

The improvement was 8–14x better than the natural history of disease (2.7%) with currently available treatments, Perfuse said at the time.

In the other Phase IIa trial (NCT06003751), which focused on DR, patients showed a mean of +0.9 dB improvement in low luminance contrast sensitivity in the high-dose group and +0.65 dB in the low-dose group across multiple frequencies measured at week 20. In contrast, a mean of -2.1 dB worsening occurred in the control group over the same period.

The low luminance, low contrast visual acuity was better by a mean difference of 5.5 and 5.1 letters from baseline in low- and high-dose groups compared to control measured at week 20, Perfuse said at the time.

PER-001 is also in preclinical development for dry age-related macular degeneration (AMD)/geographic atrophy, as well as for retinal vein occlusion.

“We are excited by the work of the team at Perfuse Therapeutics and encouraged by the potential of PER-001,” Juergen Eckhardt, MD, head of business development and licensing at Bayer Pharmaceuticals, said in a statement. “With this acquisition, we are complementing our expertise in ophthalmology and our pipeline, reinforcing our commitment to developing urgently needed therapies for patients.”

Looking beyond Eylea®

Bayer’s ophthalmology pipeline has long been dominated by the blockbuster drug Eylea® (aflibercept), co-marketed with Regeneron Pharmaceuticals and initially approved in 2011. However, Eylea is close to losing exclusivity for key U.S. patents: According to Regeneron’s Form 10-K annual report for 2024, patents for Eylea expire between 2027 and 2039, starting with four formulation patents expiring on June 14, 2027. Patents for the higher-dose version, Eylea HD®, expire between 2027 and 2032, starting with two formulation patents expiring on June 14, 2027.

Last year, Eylea and Eylea HD saw their sales slip in the mid-teens, generating a total combined $8.04 billion in revenue, consisting of $4.385 billion in U.S. net sales for Regeneron and €3.11 billion in ex-U.S. sales for Bayer (about $3.655 billion today, up from the $3.506 billion reported in January).

During the first quarter of this year, Regeneron reported $941 million in U.S. sales, down 10% from a year ago; Bayer plans to report Q1 sales on May 12.

PER-001 is an intravitreal bio-erodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide a sustained release of the drug, allowing for a convenient dosing regimen, according to Perfuse and Bayer.

Bayer has agreed to pay $300 million upfront for Perfuse, which is headquartered in San Francisco with R&D facilities in Durham, NC. The remaining up to $2.15 billion in deal value hinges on Bayer achieving development, regulatory, and commercial milestones.

The acquisition deal is subject to approval by Perfuse shareholders and antitrust clearances.

“I’m incredibly proud of what the Perfuse team has accomplished and deeply thankful to all our investors and collaborators,” stated Sevgi Gurkan, MD, Perfuse’s founder and CEO. “Bayer’s vision aligns closely with ours, and they have the scale and global resources to unlock the full potential of PER-001 to change the trajectory of human blindness. We are very excited to see our mission continue with even greater momentum.”

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From Discovery to GMP: Building Scalable Cell Therapy Manufacturing

From Discovery to GMP: Building Scalable Cell Therapy Manufacturing eBook

Over the past decade, our industry has witnessed the promise of cell and gene therapies. Patients with rare diseases or hard-to-treat diagnoses now have treatment options harnessing human cells and genes to alter disease. The accessibility of these therapies remains constrained not by what’s biologically possible, but how they are designed and manufactured.

The field has reached an inflection point. We’ve demonstrated the scientific foundation and its curative potential. To make advanced therapies sustainable as a pillar of medicine, we must make them more accessible. The companies that will define cell and gene therapy’s future will be those who can eliminate the distance between top science and efficient manufacturing.

This eBook brings together perspectives from Genetic Engineering News and ElevateBio to examine both the technical and operational realities shaping cell therapy today. From emerging innovations to persistent manufacturing challenges, the goal is to connect scientific progress with the systems required to scale it.

Traditional drug development has relied on siloed pathways, where therapeutics are designed and developed by one team and then manufactured by another. This approach is especially challenging in cell therapy, often leading to delays, setbacks, or outright failures. ElevateBio was built differently. Therapeutic design, development, and manufacturing operate as an integrated ecosystem, enabling tighter coordination and faster iteration.

The future of cell therapy depends on therapies designed with manufacturability in mind from the start. Process development, analytical strategy, and quality considerations must be embedded early, allowing manufacturing insights to inform development decisions in real time. This includes optimizing constructs, delivery systems, and processes to ensure scalability, reproducibility, and readiness for GMP production.

Looking beyond the science, a sustainable cell therapy ecosystem requires more than better therapeutics. It requires expanded treatment infrastructure, new commercial models, and systems capable of supporting broader patient access. But that ecosystem cannot scale on unreliable manufacturing.

As cell therapies expand into larger patient populations and new indications, the need for manufacturing designed for reliability and scale from day one becomes more urgent. The therapies being developed today have the potential to transform millions of lives—but only if the systems supporting them are built to deliver at scale.

Michael Paglia, Chief Technology Officer, ElevateBio

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What’s next for cervical spine tech and startup Synergy Spine Solutions

Synergy Spine Solutions designed its Synergy Disc cervical implant like a ball and socket hip joint to improve alignment while preserving range of motion for patients with degenerative disc disease. Synergy Spine engineers created Synergy Disc using a new patented geometry paired with time-tested materials. “One of the secrets to great design is to just…

The post What’s next for cervical spine tech and startup Synergy Spine Solutions appeared first on Medical Design and Outsourcing.

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May 2026 issue: Fighting diabetes with next-gen sensors and drug delivery devices

 Fighting diabetes with next-gen sensors and drug delivery devices We’ve come a long way from fingersticks for diabetes patients. Though the diabetes patient population continues to grow, I’ve never been more optimistic about medtech’s ability to take on this global epidemic. Medical devices have traditionally played a more passive role, allowing patients and physicians…

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Heavy-Chain BsAbs More Manufacturable than Light-Chains

Bispecific antibodies (BsAb) were first approved in 2014. Since then, a total of 19 have been approved globally, and approximately 250 BsAbs are being developed by some 180 companies, according to a report from Research and Markets. As this therapeutic class moves out of the lab and into clinical and commercial sectors, manufacturing may be its biggest challenge.

These dual-targeting compounds are considered difficult to produce, especially at scale. With low yields, potential chain mispairing, stability and aggregation issues, and analytical characterization challenges, biomanufacturers are eager to find expedient solutions. “There is still significant work to be done in bioprocess engineering to substantially improve the efficiency of bispecific antibody design and manufacturing,” Laura A. Palomares, PhD, senior researcher, Universidad Nacional Autónoma de Mexico (UNAM), tells GEN.

To that point, rather than relying upon transient expression systems or evaluating various antibody sequences, Palomares and her team are identifying manufacturable BsAbs by correlating their architectures to growth, productivity, downstream recovery, production of product-related variants, and in vitro binding to Zika virus and transferrin receptor (TfR). They found BsAb architecture is crucial in terms of those manufacturing criteria.

Structure governs performance

Specifically, as much as a 70% difference in productivity was found between symmetric heavy-chain scFv fusion BsAbs—which performed like the parental antibody—and BsAbs that were designed as light-chain scFv fusion, dual-variable domain immunoglobulin (DVD), or asymmetric antibodies. That’s according to a recent study by Palomares, doctoral student Juan Carlos Rivera-Castro, and senior researcher Octavio T. Ramirez, PhD.

Asymmetric BsAbs had the worst culture performance and productivity of the BsAb architectures tested. Asymmetry created imbalanced chain expression and formed homodimeric and half-antibody by-products, they reported, which dropped purity to approximately 68% after protein A purification. They found that binding to the LC-scFV diminished the binding to Zika virus, and that DVD increases it. Also, binding to TfR varied according to BsAb valency and configuration.

In contrast, “Bivalent heavy-chain scFV formats show[ed] stronger apparent binding than monovalent formats,” they report.

For the best manufacturability, the team says, “Avoid the modification of the light chain and preserve symmetric assembly.” This strategy resulted in higher cell viability, productivity, and final purity.

“The construction and head-to-head comparison of various formats, including the effect of the formats on antigen binding, can guide those planning the design and production of BsAbs,” Palomares says, by understanding the relative tradeoffs of various architectures as they design and clone BsAbs for specific functionalities.

“Format selection should prioritize manufacturability, with complex designs reserved for cases with particular functional requirements,” the scientists conclude.

Next steps, Palomares says, are to “determine the in vivo functionality of the constructed formats to neutralize Zika virus after traversing the blood-brain barrier. The results of those experiments will also be useful to scientists interested in BsAb design.”

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Researchers’ Spinout Focuses on Simplifying Viral Vector Purification

U.S.-based researchers have developed a portfolio of peptide ligands for purifying viral vectors for gene therapies and have launched a company that develops affinity technologies for biopharmaceutical manufacturers.

ChromaGenix, a spinout from North Carolina State University (NC State), commercializes peptide ligands as an alternative to the traditional protein ligands used in affinity chromatography.

According to Stefano Menegatti, PhD, CSO at ChromaGenix and a professor in the Department of Chemical and Biomolecular Engineering at NC State, the ligands are cheaper and less likely to trigger an immune response in patients than the protein ligands traditionally used.

“Protein ligands have been a fantastic enabler of advanced biological therapies over the past two decades, but they do have shortcomings,” he explains. “Proteins can denature or degrade, potentially releasing immunogenic fragments, which poses a certain level of risk.” As such, Menegatti says, protein ligands have a short lifetime and must be replaced frequently, adding further to production costs. They can also bind too strongly to the product, making it harder to recover it from the chromatographic step.

By contrast, peptide ligands, which are very small proteins, overcome these issues, he says. They can be produced synthetically, making them cheaper than proteins. As they don’t have a complex structure, they can be cleaned under harsh conditions without becoming inactive. They have a longer lifespan and a much lower immunogenicity risk, Menegatti says. Also, as they’re small, he explains,they can be cleared during final product filtration.

This “represents a new frontier of gene therapy manufacturing, says Menegatti, “as it boosts the efficiency of the viral vector manufacturing pipeline.”

Having developed peptide ligands for a wide variety of viral vectors, ChromaGenix is already selling to many companies, Menegatti says. The researchers and the company are now hoping to move beyond gene therapies.

“Our next chapter is going to be developing ligands for the purification of therapeutic cells, starting with CAR [chimeric antigen receptor] T cells,” he says.

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