Drug Discovery at the Intersection of Biology and AI
BIOSECURE ACT
Biopharma's New Reality?
NEW HEALTHCARE MARKETING TRENDS
p. 5
BIOTECH TO WATCH: PROTAGONIST THERAPEUTICS p. 18
ENMEDIA IS A PUBLICATION OF THE ENTENTE NETWORK OF COMPANIES
Caroline Montojo, PhD President & CEO
The Dana Foundation
Inside the BioFuture Issue
ON OUR COVER
As breakthroughs in brain science reshape what’s possible, the Dana Foundation is leading the charge to address the ethical and social challenges that come with progress. In this story, ENMEDIA explores how bold efforts like the Dana Center Initiative are ensuring neuroscience and society evolve together.
04 Letter From the Publisher 05 Healthcare Marketing Trends
06 TechBio Emerges: Where Technology and Biology Meet
10 Advancing Biotechnologies for Unmet Medical Needs: Four Innovators Leading the Way
12 Biosecurity or Burden? New US Law Could Reshape the Global Biopharma Landscape
16 Opinion: Keep it Simple, Sustainable: Empowering Global Health with Grounded Solutions over Lofty Ambitions
18 From Platform to Partner: Protagonist Therapeutics Formed Strategic Collaborations with J&J and Takeda. What's Next?
20 Sustainable Food: Will Fermentation Take Hold in the Alt-Meat Industry?
22 Dana Foundation: Connecting Advances to Communities, Advocacy and Education
26 Opinion: Moving Beyond the Amyloid Hypothesis: A Paradigm Shift Is Underway
28 Blockchain Beyond Bitcoin: A Look at Decentralized Science
THE ENTENTE NETWORK OF COMPANIES delivers corporate communications and reputation services, technologies and media that meet the needs of companies operating in increasingly complex, dynamic and connected markets: Pharma, life sciences, diagnostics, digital health, hospital systems, food science, sustainable energy, and social impact.
Personal Perspectives, Global Insights
The BioFuture Issue: At the Crossroads
Born at the intersection of business, science, media and society, ENMEDIA (E-N-Media) is a new media company championing fresh perspectives on how science and innovation are shaping our world. The BioFuture issue focuses on the synthesis of disciplines, technologies and business models.
Developing the business and reputation solutions that bring together our years of experience and deep networks in life sciences, pharma, diagnostics, digital health and science foundations. Media relations, financial communications, fundraising support, design, executive visibility, thought leadership, communications planning.
Serving the unique reputational and marketing communications needs for health delivery companies including those in telemedicine, health and hospital systems, and emerging companies blending disciplines to meet the needs of patients.
Read on for more about organizations at the crossroads of technologic advancement, the coming together of fields in ways that will change how we eat and how we treat disease, and how the collision of national security priorities and healthcare innovation are leading to division.
A big thank you to the ENMEDIA Editorial Team and our contributors, who worked diligently to develop another thought provoking issue. I look forward to your commentary, and we welcome your story ideas for future issues.
Virginia Amann CEO & Founder of ENTENTE Network of Companies Publisher of ENMEDIA
Providing actionable insights via our integrated suite of behavior and data science-based research methodologies and AI-integrated technologies. We speed and diversify clinical trial recruitment, help change consumer health behaviors and provide the data you need to drive your business.
Editorial Description
ENMEDIA Magazine champions innovation stories through conversations with influential thought leaders in the science industries. We empower our readers with evidence-based insights and guidance to lead, operate, and create positive change in a world continually shaped by technology and large-scale societal and environmental forces.
For story ideas and advertising opportunities please contact us: info@ENTENTENetwork.com | ENMEDIA.Network
Disclaimer: The content provided by ENMEDIA is for informational purposes only and should not be construed as investment advice. Our articles, analysis, and perspectives focus on providing insights into various companies, some of which may be publicly traded. However, this information is not intended to be a recommendation for buying, selling, or holding securities. We encourage our readers to seek professional advice before making any investment decisions. ENMEDIA does not assume any responsibility for investment actions taken based on the information provided in our publications.
Any logos and trademarks of entities featured in this publication are the property of their respective owners. Their inclusion is for informational purposes only and does not imply endorsement or sponsorship by the entities mentioned. Some of the entities featured may be current or former clients of ENTENTE, LLC. However, the editorial content remains independent, and any opinions expressed are solely intended to inform readers about companies and organizations in the industry.
HEALTHCARE MARKETING TRENDS
Key Factors Shaping the Future of Healthcare Engagement
The healthcare industry is rapidly evolving as marketing strategies adapt to technological advancements and shifting customer expectations. This transformation has reshaped how healthcare organizations engage with their audiences, prioritize direct customer relationships, and navigate ongoing challenges such as converting leads into sales and maintaining regulatory compliance. As the landscape continues to evolve, insights into emerging trends, successful strategies, and persistent hurdles will be critical for healthcare marketers looking to stay ahead in this competitive field.
WHAT COMPANIES SHOULD KNOW
Direct Relationships and Attention-Grabbing Strategies
Converting Leads into Sales Remains a Challenge
In-Person Events Retain Value, but Their Role is Evolving
Marketing and Sales Budgets Remain Strong
Current Strategies Remain Important
Regulatory Uncertainty Looms with Upcoming Election
Rising Customer Expectations
Content Marketing Strategies Need Strengthening
Marketing and sales leaders prioritize direct customer relationships by focusing on high-quality long-form content, leveraging digital marketing (especially video), and utilizing customer reviews to influence purchasing decisions, particularly in healthcare.
Despite significant investments in marketing efforts, converting leads into actual sales continues to be a top challenge. Budget constraints compound the issue, making it critical for organizations to optimize their marketing efforts to drive conversions.
In-person events remain valuable for building relationships, but their limited engagement throughout the buying journey is a concern. Companies must increasingly diversify their engagement strategies.
Marketing and sales budgets are expected to remain steady or slightly increase in 2025, with digital ad spending growing due to economic recovery, making it vital for future strategies.
Lead nurturing, content marketing, social media, and in-person events will continue to play an integral role in connecting with and engaging potential customers.
With the 2024 election nearing, uncertainties around Medicare, Medicaid, and HIPAA regulations are top concerns. The industry is also focused on maintaining transparency as consumers question the credibility of healthcare content.
Healthcare marketers expect tougher competition and higher customer expectations. AI and machine learning will drive personalized care, while patients will demand seamless engagement across all touchpoints.
Many healthcare companies lack strong content strategies and rely on outsourcing, but high-quality, targeted content is essential for converting leads, especially in the pharmaceutical sector. Investing in this will drive future success.
EMERGES TECHBIO
TECHBIO
EMERGES
WHERE TECHNOLOGY AND BIOLOGY MEET
What is TechBio?
Put simply, biotech companies lead with science. TechBio players leverage cutting-edge advances such as artificial intelligence (AI), machine learning (ML) and computational biology to solve pressing challenges in healthcare, agriculture and sustainability. With the global biotechnology market size estimated to reach $727.1 billion by 2025, TechBio stands as a key driver in this growth trajectory, transforming
not only drug discovery but also entire healthcare systems.
Why Now?
First, biological data is more accessible than ever. The cost of DNA sequencing has plummeted, making it feasible to digitize biological processes and generate immense datasets. For instance, technologies like high-throughput sequencing, CRISPR and RNA-based platforms are accelerating biological research from months to days, digitizing vast amounts of biological data.
Second, advancements in AI and ML provide the analytical tools necessary to interpret these complex datasets. As the amount of biological data generated continues to grow exponentially, AI’s ability to detect patterns and correlations between biological markers, patient outcomes and drug efficacy is revolutionizing everything from drug discovery to personalized medicine. AI-driven discoveries that would have taken decades through traditional methods can now be achieved in months. Finally, laboratory automation is significantly increasing the speed at which biological discoveries are made. Gone are the days of manual pipetting. Today, robotic systems and cloud-based computational tools enable remote experimentation, reduce human error and allow for reproducible, scalable experiments.
The TechBio Era
Drug discovery and development have long been costly, with timeframes extending over a decade and costs reaching as much as $2.7 billion per drug. But TechBio companies are introducing platform models that reduce the financial and temporal constraints traditionally associated with drug development. AI-enhanced drug discovery platforms such as AlphaFold have redefined protein folding and modeling, drastically cutting the time required to develop new therapies.
A new crop of TechBio companies is integrating biology with technology to redefine drug discovery. Next, we spotlight three companies making waves.
Mammoth Biosciences: Unlocking CRISPR’s Potential: Co-founded by Nobel laureate Jennifer Doudna, Mammoth Biosciences is a TechBio company focused on discovering new CRISPR proteins. Initially targeting diagnostics, they’ve expanded into gene editing and therapeutics, developing tools to address genetic diseases. Collaborations with companies like Vertex and Bayer highlight the potential for treatments of previously incurable disorders. What sets Mammoth apart is its “toolbox” approach, offering a versatile platform that addresses multiple biological challenges, driving both diversification and revenue opportunities across industries.
NonExomics, Exploring the Uncharted 98% of the Genome: NonExomics is advancing genomic research by exploring the 98% of the genome outside the protein-coding exome, discovering functional proteins linked to diseases. The company has identified over 250,000 new proteins and around 3,000 novel drug targets. Their innovative work on amyotrophic lateral sclerosis (ALS) exemplifies their approach, using non-coding regions to develop therapies for previously untreatable diseases, opening new treatment possibilities for ALS and other complex genetic conditions.
Envisagenics: Envisagenics uses AI and RNA splicing data to accelerate RNA drug discovery. With over 95% of human genes undergoing alternative splicing, the company targets over 370 diseases, including cancer and neurodegenerative disorders. Their AIpowered SpliceCore® platform expands RNA sequence analysis 400-fold compared to conventional tools, mapping over 14 million splicing events. This technology enables the rapid discovery of disease-specific targets, paving the way for new immunotherapies and RNA therapeutics.
The Business Models
One of TechBio’s key advantages lies in its unique business models. Unlike traditional biotech companies, which often focus on single therapeutic products, TechBio companies often develop platforms. These platforms are not only capable of generating multiple drugs or products but also capturing additional revenue streams from partnerships, licensing and service models.
For example, Mammoth Biosciences and Envisagenics both license their platform technologies to pharmaceutical companies, generating revenue while helping their partners develop tailored treatments at a rapid pace. These models enable TechBio companies to generate revenue earlier in their life cycles compared to traditional biotechs—a valuable de-risking strategy.
The Future
Despite the excitement surrounding TechBio, the industry is not without its challenges. Regulatory frameworks have not kept pace with the rapid developments in AI and computational biology, and public concerns about data privacy remain a hurdle. And while AI-designed drugs remain promising, they face the same bottlenecks as traditional drugs in clinical trials.
But as technologies mature and companies like Mammoth Biosciences, NonExomics and Envisagenics demonstrate the power of these TechBio platforms, the potential to transform healthcare is clear. After all, the goal of the biotech industry is to get life-changing therapies to patients as quickly and cost effectively as possible. TechBio is driving a shift where biology, once seen as a complex natural process, is becoming something we can precisely engineer via the integration of data, AI, and biology. As these technologies converge, the boundary between biology and technology will dissolve, leading to a future where both fields advance together, reshaping the way medicines are developed and brought to patients.
THE TECHBIO SHOWCASE AT BIOFUTURE
BioFuture returns to New York City this year to explore the convergence of biopharma R&D, artificial intelligence, technology and healthcare delivery. This year’s conference will introduce the new TechBio Showcase— featuring innovators applying tech-first approaches to biopharma innovation.
TechBio is driving a seismic shift in the life sciences, combining biotechnology with artificial intelligence, machine learning, and cutting-edge computational tools to unlock new possibilities in precision medicine, genetic sequencing, and biomanufacturing.
At the TechBio Showcase, innovators will present groundbreaking solutions that accelerate drug development, reduce costs, and transform healthcare delivery. From AIpowered drug discovery to advanced geneediting technologies, this is where the future of healthcare is being shaped.
ADVANCING BIOTECHNOLOGIES FOR UNMET MEDICAL NEEDS:
Four Innovators Leading the Way
Acuamark Diagnostics, Igyxos Biotherapeutics, NewBiologix and Triovance are making significant strides in tackling complex health challenges. Each company is leveraging innovative technologies to offer new solutions across a variety of health conditions and therapeutic areas, including early cancer detection, diabetic foot disease, infertility, and gene therapy. Their work highlights important advancements in fields that have long faced treatment gaps.
Acuamark Diagnostics: Early Cancer Detection
Igyxos Biotherapeutics: Exploring New Treatments for Infertility
Acuamark Diagnostics is focused on improving early detection of cancers, particularly those with poor prognosis when diagnosed late. Acuamark's qPCR-based platform enables ultra-sensitive detection of early-stage cancer markers, offering high accuracy even at low abundance to potentially catch cancer before it becomes untreatable. This approach may provide patients with a better chance at timely intervention, addressing the longstanding challenges of late-stage cancer diagnosis.
Male infertility, which accounts for about half of infertility cases, has historically lacked targeted treatment options. Igyxos Biotherapeutics is working to fill this gap with IGX12, a monoclonal antibody designed to enhance the natural function of FSH, a hormone crucial for reproductive health in both men and women. IGX12 has shown promise in early studies, potentially reducing the number of treatment cycles needed for patients and offering a new approach to male infertility, an area that has seen limited innovation.
NewBiologix: Optimizing Gene Therapy Production
Gene therapy holds great promise for treating rare and chronic diseases, but the production of viral vectors used in these therapies has been a significant challenge. NewBiologix is developing technologies to improve the efficiency and scalability of vector production. The NBX Engineered HEK293 Cell Line aims to address common issues in gene therapy manufacturing, such as low yields and high costs, by providing a stable platform for producing recombinant adeno-associated virus (rAAV) vectors. This work could support faster and more reliable gene therapy development.
Triovance: Addressing Diabetic Foot Disease
Diabetic foot disease remains a difficult complication for millions of people living with diabetes. Triovance is addressing this issue with its lead product, TRV-01, a genetically engineered skin substitute that promotes healing from within. TRV-01 works by increasing blood flow and cell regeneration at the wound site, offering a new approach to managing foot ulcers. The product could help reduce the need for frequent hospital visits and lower the overall burden of care for patients with diabetic foot disease.
BIOSECURITY OR BURDEN?
New US Law Could Reshape the Global Biopharma Landscape
By Ryan Flinn
In the latest chapter of escalating U.S.-China tensions, recently passed legislation could dramatically reshape the global biopharma industry. The BIOSECURE Act, targeting Chinese-based manufacturers, threatens to increase drug costs and shift production to countries like India and South Korea.
The legislation, passed by the U.S. House of Representatives in September with broad bipartisan support, is awaiting Senate review. If enacted, the legislation would prohibit U.S. pharmaceutical and biotechnology companies from collaborating with certain Chinese firms, particularly contract development and manufacturing organizations (CDMOs). This move, aimed at addressing national security concerns, could affect more than 100 drugs and disrupt established supply chains.
The move comes amid growing concerns by U.S. lawmakers and others over broader national security, data privacy and economic competition. Legislators have debated and passed measures including tariffs on Chinese goods and restrictions on Chinese technology platforms like TikTok. For the biopharma industry, the BIOSECURE Act could force major shifts in supply chains and research partnerships, potentially impacting drug development timelines and costs.
John Crowley, President & CEO of the Biotechnology Innovation Organization (BIO), one of the largest industry trade groups, described the legislation as very targeted, focusing on U.S. government contracts with a limited number of biotech companies based in China. Still, it points to a broader issue with manufacturing in the U.S., he said.
"BIO applauds this work and supports this BIOSECURE legislation,” Crowley said in a May statement. More recently, he noted that the U.S. has “lost the resiliency of our entire manufacturing supply chain in the biotech, biopharmaceutical world,” Crowley said at the GEN State of Biotechnology 2024 held on Sept. 11
A BIO survey of 124 companies found 79% have at least one contract or product with a China-based or China-owned CDMO/CMO, and that these companies estimated they will need up to eight years to switch manufacturing partners.
Targeted Companies
The act targets five specific Chinese companies, including WuXi AppTec and WuXi Biologics, which have been deeply integrated into the U.S. drug development ecosystem. WuXi AppTec was involved in 27% of the small molecule drugs approved by the FDA in 2023, according to the company’s Chief Business Officer Yu Lu. The company generated 65% of its revenue last year from the U.S., and currently operates 10 facilities in eight U.S. states with approximately 1,900 employees and plans to create an additional 500 jobs by 2026.
“We are concerned by a misguided U.S. legislative initiative to target our company without a fair and transparent review of the facts,” WuXi AppTec wrote in an open letter published in February. “We want to be clear and set the record straight: WuXi AppTec does not pose a national security risk to any country.”
Complete Genomics, one of the companies named in the bill, put out a statement in May saying the bill would “stifle innovation, raise costs for scientists and labs and could even delay important research in areas like Alzheimer’s, oncology and food productivity.”
The bill's impact could be far-reaching. According to data from GlobalData, more than 120 U.S. biopharmaceutical drugs in development involve partnerships with the Chinese companies named in the legislation. About half of these are
in clinical-stage trials, while a third are in early-stage preclinical or discovery phases.
Opportunities at Home and Abroad
“This demonstrates long-standing relationships between significant players in the biopharmaceutical industry and those major Chinese companies,” said Alison Labya, Business Fundamentals Analyst at GlobalData, in a statement. “Indian CDMOs such as Cipla, Syngene, and Aurobindo may be well-positioned for increased growth due to their cost-effectiveness and highly skilled workforce. However, growing concerns of US dependency on offshore suppliers may also result in a shift towards US companies selecting domestic CDMOs.”
Companies like Mark Cuban’s Cost Plus Drug Company and Eli Lilly have already announced plans to expand their U.S.based production facilities. Thermo Fisher Scientific has also reported increased interest from clients looking to shift their manufacturing partnerships. KBI Biopharma, a global CDMO based in Durham, North Carolina, has seen an uptick in business, according to J.B. Angus, chief business officer.
“KBI has been seeing an increase in requests for proposals from all industries and regions due to this potential legislation,” Angus said in an article written by the company. “Several have indicated definitive directives from their respective boards to make the change immediately.”
Similarly, a trio of U.S. based CMDOs have teamed up to offer more services to prospective clients: Durham, North Carolina-based Alcami has partnered with San Diego, California’s Tanvex CDMO and Oklahoma City, Oklahomabased Cytovance Biologics to offer comprehensive drug development and manufacturing services.
Other beneficiaries of the shift will likely include manufacturers in India and South Korea. Indian firms currently hold less than 2% of the global CDMO market but could see additional growth. South Korea’s Samsung Biologics, which already manufactures 17 innovator and biosimilar drugs approved in the U.S., are also likely to benefit from the shift away from Chinese CDMOs.
Even rodents may feel the impact of the legislation. Research firms that have depended on Chinese CDMOs might
now seek more U.S.-based or alliedcountry suppliers for drug discovery and preclinical research involving animal models.
Drug Shortages
Shifting drug manufacturing from Chinese-based facilities to other countries can still lead to problems. In early 2023, a major producer of chemotherapy medications, India’s Intas Pharmaceuticals, suspended production following an FDA inspection. More than a year later, more than half a million cancer patients had been affected, according to Julie Gralow, M.D., chief medical officer at the American Society of Clinical Oncology (ASCO), in an April 2024 article.
“The underlying problems that led to the situation have not changed,” Gralow said. ”And we are very vulnerable to this happening again.”
The act wouldn’t require an immediate halt to business – the legislation includes a grace period until 2032 for existing contracts to mitigate immediate disruptions. Still, a study by L.E.K. Consulting found that 68% of global life sciences companies have already adjusted their operations in response to the bill, including increasing legal and compliance requirements, diversifying partnerships and adding background checks for existing partners.
Predicting China’s Response
The Chinese government is likely to respond to the legislation in-kind, according to law firm Foley & Lardner LLP, and biopharma companies as well as universities and other U.S. research institutes that receive federal funding should prepare to wind down business operations with named Chinese firms.
“With such bipartisan, bicameral support, this could just be the tip of the iceberg in limiting American businesses’ dealings with Chinese firms, both in the biotech space and beyond,” said John H. Strom, Special Counsel and Kate M. Kros, Senior Public Affairs Advisor, in a September blog post on the firm’s website. China’s response “could range from imposing its own export controls to using the country’s sweeping national security laws to harass United States businesses and their employees.”
Uncertainty Ahead
During a June 20 podcast discussion on the legislation, Steve Usdin, BioCentury’s Washington Editor, said Chinese companies may find ways to spin out parts of their activities into separate entities that are not covered by the BIOSECURE Act, to continue offering their services to U.S. companies.
But there’s still much uncertainty, he said.
“Big picture – it's clear that there's geopolitical tensions between the United States and China, that there's no sign of those easing in the foreseeable future, and companies are going to be looking for ways to reduce their reliance on Chinese companies,” Usdin said.
Joshua Berlin, BioCentury’s Head of Corporate Alliances & Business Development, said on the podcast he was hearing from many industry contacts that investors, executives and boards of directors were scrambling for alternative manufacturing plans.
“This is a tremendous wake-up call to biotech CEOs and particularly to boards and investors,” Berlin said. “Many of those boards and investors right now are pressuring their biotech CEOs about what
their plan B is for Wuxi and even broader, what's their plan B for geopolitics.”
Unfortunately for smaller biotech companies, there aren’t many easy answers. Wuxi has offered high quality, affordable manufacturing, but when that capacity is taken off the market for U.S. companies, they’ll need to find alternatives, and larger companies will have the advantage in snapping it up, Berlin said. Overall, the legislation will likely lead to disruption and higher drug development costs.
“I don't think that it's desirable from the standpoint of view of innovation, or certainly not from the standpoint of companies and patients in both countries,” Usdin said.
KEEP IT SIMPLE, SUSTAINABLE
Empowering Global Health with Grounded Solutions over Lofty Ambitions
By Meredith Leston
We have everything we need to make the intractable more tractable.
With a backdrop of escalating tensions in the Middle East, ongoing conflict in Europe and an upcoming Democratic Party rematch with Donald Trump, there was plenty of ground to cover at the 79th United Nations General Assembly (UNGA). It was heartening to see global health still prioritised in such a politically fractious landscape: addressing issues including medical inequity, antimicrobial resistance and worsening maternal health were positioned as the superordinate goals that could see international differences put aside. However, the complexity of these challenges–especially their interlocking with the diplomatic sticking points of commerce, climate and even culture –saw simple solutions and real-world action lose out once again.
This was not lost on attendees. Side events were sometimes confrontational, with brokered resolutions and reports on emergent technology's humanitarian potential met with short shrift from those at the coalface of care. These were midwives, forced to split incubators across multiple infants in critical condition.
These were community care workers, supporting their patients without personal protective equipment and without forewarning that they harboured resistant bacteria. These were surgeons, factoring regular power outages and bed shortages into their case lists. They spoke as one: today’s challenges cannot wait for tomorrow’s solutions. Grandiose rhetoric and the promise of new technology do nothing to make their lives any easier or their patients any safer.
Philanthropic dollars instead need to trust in the tools that are tried, tested and true–first backing what is available, affordable, sustainable and–in the case of community health–portable and easily fixable. For antimicrobial resistance, this ‘grounded innovation’ could look like distributing virological point of care testing in primary care or promoting smartphone-based medication reminders. Likewise, harmonising hospital data entry, inventories and ordering forms–something that really amounts to the plumbing of digital healthcare–can do far more for infant death than AIdriven pop-ups in patients’ medical charts. We have everything we need to make the intractable more tractable.
That said, grounded innovation and blue sky thinking (Silicon Valley slang for the transformative) are not always at odds with one another. It takes a real ‘out of the box’ mindset to repurpose the toolkit we already have. Empowering local entrepreneurs will be key in this–still a critically underutilised asset in the global development world. No other segment unlocks local knowledge, local networks and local trust as readily–all of which are needed for innovation to embed itself without collateral damage.
In conclusion, although UNGA 79 brought welcome relief and renewed commitments to global health, the airtime given to far-off ambitions and untested technologies was concerning. Without immediate action, these resolutions lack real resolve. But innovation comes in all shapes and sizes, and–with sufficient and sustained political will–there are tools and technologies ready to be scaled for impact. Vaccines, PPE, decontamination, remote monitoring, point of care testing, telephonic care, video-assisted education, social media campaigns, social prescription, peer support, public patient involvement and engagement–there are no quick-fixes in healthcare, but these are the low-hanging fruits of transformation that can really get the needle going. After all, every first step needs a grounding foot.
Meredith Leston is an award-winning public health and social impact professional committed to improving public services and population health outcomes. With a career spanning academia, advocacy, policymaking and corporate philanthropy, she champions cross-sector collaboration and evidence-based approaches. She is also an advisor to ENTENTE Network.
FROM PLATFORM TO PARTNER
Protagonist Therapeutics Formed Strategic Collaborations with J&J and Takeda. What's next?
Protagonist Therapeutics has steadily built a name for itself in the biotech space, not just through breakthroughs in peptide chemistry, but by aligning with industry powerhouses like Johnson & Johnson (J&J) and Takeda. These partnerships underscore the strength of Protagonist’s innovative peptide technology platform and have provided the company with more than $250 million in license and collaboration revenues as of the six months ended June 30th, 2024.
Protagonist’s stock has risen by more than 90% year-to-date, driven by positive clinical developments, investor confidence in its pipeline and an inclusion in the S&P Small Cap 600.
CEO Dinesh V. Patel has said the company is seizing on immense opportunity to differentiate oral peptides from small molecules and large biologics, especially in chronic indications where oral drugs offer greater convenience to patients.
Oral IL-23 Receptor Agonist
Protagonist has engineered some of the world’s first promising oral peptides.
Designed for stability and bioavailability, they may overcome the body’s natural mechanisms that break down larger molecules in the digestive tract—a challenge that has long stumped scientists.
JNJ-2113 (formerly PN-235), an oral IL-23 receptor antagonist jointly discovered by J&J and Protagonist scientists, is a crown jewel in I&I pipelines. Data from a Phase 2b trial of JNJ-2113 moderate-tosevere plaque psoriasis look promising: 78.6% of patients achieved at least a 75% reduction in the Psoriasis Area and Severity Index (PASI-75) after 16 weeks at a 100 mg twice-daily dose. Today the drug is being shepherded through Phase 2b and Phase 3 clinical trials in moderate-to-severe psoriasis and other types of psoriasis as well as ulcerative colitis. Data readouts are expected soon. If the drug is successful, J&J has suggested it could achieve more than $5 Billion in sales annually.
Rusfertide
While the J&J partnership focuses on inflammatory conditions, Protagonist’s collaboration with Takeda showcases the versatility of its peptide platform in other disease areas. Together, the companies are advancing rusfertide, an injectable
treatment for polycythemia vera (PV), a rare blood disorder. Rusfertide, designed to mimic the hormone hepcidin, is being evaluated in a pivotal Phase 3 trial, and preliminary results are expected later this year. The drug aims to regulate iron homeostasis, controlling the overproduction of red blood cells that causes serious complications like blood clots, stroke, and heart attacks.
The partnership with Takeda involves a $300 million upfront payment, with potential regulatory and sales milestone payments exceeding $1.38 billion, depending on the success of the trial and commercialization. Protagonist retains the option to co-commercialize rusfertide in the U.S. on a profit-sharing basis or opt-out for additional milestone payments and royalties of up to 29% on ex-U.S. sales.
What’s next? Protagonist is working to expand its discovery pipeline by focusing on new biological targets, including oral peptides that mimic natural hormones, as well as IL-17 pathway blockers, which have the potential to address unmet needs in inflammation, hematology, and obesity.
Peptide drugs are increasingly important in addressing conditions like diabetes and obesity. GLP-1 receptor agonists, a class of peptide drugs, have gained significant attention for their role in managing these conditions by mimicking the action of the body’s natural incretin hormones, which regulate insulin and appetite. Drugs like semaglutide and liraglutide, both GLP1 agonists, have shown substantial efficacy in improving glycemic control and supporting weight loss. Advances in peptide delivery systems, including oral formulations, are making these therapies more accessible. However, regulatory bodies like the FDA are carefully monitoring their safety due to concerns about long-term effects. Peptides have potential in a range of other diseases, including autoimmune disorders and cancer. Smaller than proteins and consisting of short amino acid chains, they are ideal for modulating biochemical processes in the body.
Could mushrooms and microbes change the future of food? As demand for plant-based and lab-grown alternatives rises, fermentation—both traditional and cutting-edge—emerges as a key player. This ancient process is now being used to create proteins that replicate the taste, texture, and nutrition of animal products while reducing the environmental costs of industrial farming.
Fermentation has a long history, from bread and beer to cheese and yogurt. Today, there are two types steering the alt-meat revolution:
• Traditional fermentation uses naturally occurring microorganisms like yeast, bacteria, or fungi to break down sugars and create protein-rich biomass. For example, mycelium, the root structure of mushrooms, is used by companies like Meati to create meat alternatives. This natural process doesn’t involve genetic modification and is seen as a sustainable, scalable way to produce clean-label products that appeal to health-conscious consumers.
• Precision fermentation takes things further by genetically modifying microorganisms to produce specific proteins found in animal products, like whey or casein. Companies like Perfect Day and The EVERY Company use this method to create animal-free dairy and egg proteins. This approach allows for precise control over protein structures, offering a more innovative alternative to traditional farming.
Despite its promise, fermentation faces hurdles. Critics question whether precision fermentation can scale costeffectively given its significant energy demands. Unless powered by renewable sources, its environmental benefits could be limited. On the other hand, traditional fermentation is less energy-intensive but faces challenges with consumer adoption, scalability, and regulatory barriers. However, both methods offer significant land and water savings compared to conventional animal agriculture.
Recent disease outbreaks highlight the fragility of traditional meat production. In 2019-20, China’s pork industry suffered a severe shock from African Swine Fever, leading to the culling of over 100 million pigs. This crisis, alongside outbreaks like H5N1 avian influenza, underscores the need for safer, more sustainable protein sources. China has to import hundreds of billions of pounds of soybeans annually (as feed) to sustain its pork industry, pointing to its food insecurity, not to mention the fuel usage involved.
Government support is helping fermentation gain momentum. In 2022, the Biden administration backed biotechnology and biomanufacturing through an Executive Order aimed at improving food security and sustainability. This support fosters public-private partnerships and encourages the development of both traditional and precision fermentation. With the potential to reduce resource consumption and tackle global challenges like biodiversity loss, fermentation is a key part of the government’s longterm food strategy.
Corporations are also investing heavily. Cargill, one of the world’s largest privately held companies, is scaling fermentation-derived proteins and sweeteners through partnerships with companies like Perfect Day and Avansya. However, with recent financial challenges and restructuring, it’s unclear if Cargill will fully commit to expanding its altprotein ventures.
Beyond Meat, a well-known player in plant-based meat, recently announced a launch of mycelium-based steak with a health-focused restaurant partner lined up. Its reception will be a litmus test of consumer sentiment and may reveal if the alt-meat market is setting up for a recovery after a difficult few years.
Mycelium, the root structure of mushrooms, is used by companies like Meati to create meat alternatives.
Picture a grocery store in the near future where myceliumbased steaks and animal-free milk, all produced through fermentation, are available alongside traditional products. While this scenario is becoming more realistic as fermentation technology evolves, significant questions remain about whether these products can be scaled affordably and gain widespread consumer acceptance.
Fermentation offers a glimpse into a world where clean-label, nutrient-dense foods could dominate the market, especially as consumers increasingly prioritize health and sustainability. With government support and growing industry interest, fermentation could become a cornerstone of the alternative protein industry, transforming how we produce and consume food globally.
By Wendy Diller
A DANA FOUNDATION Connecting Advances to Communities, Advocacy and Education
comparatively young field, neuroscience is entering a renaissance, with researchers producing breakthroughs that were once tagged largely as science fiction.
Witness the newfound interest in brain-computer interface (BCI) technology, which is rife with innovation. Companies like Neuralink, which has captured attention due in part to Elon Musk’s involvement, and Synchron, a lower-key player, for example, have grand ambitions to use thoughts to control digital devices. They are working on increasingly less invasive technologies that demonstrate surprising, albeit early, efficacy in helping people who have lost the ability to move or speak.
Their very effectiveness, however, has generated a range of significant questions around data privacy, consent, individual autonomy, and control, as well as safety and reliability of device use—issues that are relevant throughout medicine, but assume special dimensions when applied to the human brain.
The Dana Foundation, a philanthropic organization with longstanding interest in neuroscience research and education, is pioneering efforts to address these complex issues. In April 2024, it launched the Dana Center Initiative for Neuroscience & Society (DCI), with the goal of facilitating collaborations among multidisciplinary scholars and the communities they serve, so that these diverse groups together can tackle the complex ethical and practical implications of emerging neuroscience.
The initiative brings together “diverse voices that contribute to identifying research priorities at an early stage in the scientific process,” as well as train future leaders to shape the future of the field, says Caroline Montojo, PhD, the president and CEO of the Dana Foundation and a neuroscientist by training. Ultimately, the Foundation envisions that incorporating viewpoints from various disciplines and directly involving those who may be impacted by scientific advancements will enrich the research process and enhance neuroscience’s positive impact on society.
The DCI’s latest investment, announced in October 2024, underscores the strength of the foundation’s commitment to
these goals. It is a five-year, $8.6 million matching grant to underwrite a new Neurotech Justice Accelerator at Mass General Brigham, the largest hospital-based research enterprise in the US, which aims to improve equitable access to “beneficial neurotechnologies and to mitigate medical and legal risks.” The accelerator is the third major grant distributed so far, with an earlier Center at the University of California, Los Angeles (UCLA) with Charles R. Drew University of Medicine and Science (CDU) and a smaller program at Loyola University in Chicago.
The rapid advances of technological innovations that only a few years ago seemed far-fetched adds to the urgency of the Foundation’s mission, Montojo says, citing as examples advances in neural organoids, brain-computer interfaces, and psychedelic medicines that raise thorny questions and are in urgent need of ethical and legal frameworks.
Incorporating community values into scientific research is “another kind of innovation, a paradigm shift, which we hope to see in the neuroscience field,” she points out. The Foundation is acutely aware that “neuroscience today isn’t optimized to reflect the changing needs and challenges facing society or the complexity of neuroscience technology advancement and its potential impact,” she continues, emphasizing that its work will prepare future leaders “to embrace a more complete picture of how neuroscience advances here and abroad.”
Optimizing Clinical Access to Advanced Neurotechnologies
A series of “a-ha moments,” in which Montojo recognized the urgent need for new approaches to manage neurological and psychiatric conditions helped pave the way for her leadership of the Foundation’s laser-focused strategy.
As a post-doc, Montojo investigated early biomarkers for serious mental illnesses, such as psychotic symptoms of hallucinations, seeking to identify neurobiological risk factors for adolescent onset psychosis. At the time, “I would have loved to have dedicated education on the ethical and societal implications of my research to deepen my understanding of its impact on people and communities,” she says.
Prior to joining the Dana Foundation, she led the life sciences and brain initiatives portfolios for The Kavli Foundation, which supports basic research in four science fields, including neuroscience. While at Kavli she led convenings for the International Brain Initiative and had another “a-ha” moment when the scientific leaders of large-scale brain projects around the world called for increased attention to the ethical and societal dimensions of neuroscience.
More recently, as a member of the neuroethics working group for the NIH BRAIN (Brain Research Through Advancing Neurotechnologies) Initiative, a public-private partnership to develop tools and technologies to revolutionize our understanding of the brain, she has been privy to demonstrations of some of the most impressive advances in science today. At the group’s mid-August meeting, for example, the group discussed research from University of California, Davis on a 45-year-old man suffering from amyotrophic lateral sclerosis (ALS) who had lost his ability to speak. Through use of a novel, implanted BCI developed at the university, the researchers were able to record his brain activity and translate it into understandable text. They then took prior recordings of his voice and overlaid it on the text to convert the text to speech. The device is still in research stages, and this was a single case, but the room was “in tears,” she recalls.
research collaborations and training of multi-disciplinary neurosociety experts. It is supported by a five-year $9.8 million partnership between the Dana Foundation, UCLA, and CDU.
Community organizations in South LA have long been leading advocates for brain health and policies that improve the lives of people in the community, many of whom are underserved minorities. But until recently, UCLA did not have systemic opportunities to engage with them about community needs and values, despite its strong presence in traditional neuroscience research and its status as a public university. CDU, one of four historically Black medical schools in the US, on the other hand, founded as a response to the Watts riots of 1965 to address health inequities through community-based partnerships and training programs, has a much stronger relationship with local groups.
The device is not yet commercial, but it—and devices like it--raise ethical and practical considerations that have yet to be addressed in a holistic, systematic way. Questions arise such as who will have access to this technology and whether insurance will cover it following clinical trials, she continues, adding, “How do you make these devices into accessible clinical therapies?”
This reflects the kind of situation that prompted the Dana Foundation to establish the DCI, which has three geographically dispersed sites reflecting its priorities in education, training, and public engagement on neuroscience and society issues. The DCI investments are complemented by a broader portfolio of more modest sized grants to organizations for projects that align with the Foundation’s program pillars.
The UCLA-CDU Dana Center: Innovating CommunityPartnered Research
The first Dana Center, launched in April 2024 at UCLA-CDU), is re-thinking the relationship between academic researchers and the public. It serves as an incubator for community-partnered
With support from the Dana Center, UCLA and CDU are now collaborating to implement community-partnered neuroscience, a new research methodology, which brings to neuroscience participatory research techniques that are already wellintegrated in other areas of medicine.
Because CDU has such deep roots in the area, community organizers have responded with “remarkable enthusiasm” to the partnership’s outreach, says Helena Hansen, MD, PhD, a professor and interim chair of psychiatry at the UCLA David Geffen School of Medicine and the DCI partnership’s co-director. Using human centered design practices honed in engineering, the Dana Center has worked with community members to identify priorities for research.
For example, the partnership’s interest in the intergenerational effects of exposure to toxicogens, an enormous field, has led it to focus on a very specific issue: the impact on brain development and learning from noise generated by low-flying LA law enforcement helicopters, operating late at night and early in the morning. Officials use the helicopters to monitor gangs and street violence in parts of South LA and certain public housing projects. Fellows engaged in this project are working with rapidly reproducing models such as rodents and fruit flies to track the epigenetic effects of noise-related sleep disruption on brain development and learning.
The community also was interested in identifying strategies that enable people to thrive in disadvantaged environments like South LA. Through human-centered design, the partnership identified exposure to music and engagement with community as positive
influences on residents. “The creation of green space in South LA meets a lot of different needs, given the area is a food desert and populated by many migrants from agricultural cultures,” says Denese Shervington, MD, professor and department chair of psychiatry at CDU, who is also a co-director of the Center. “This makes it a really interesting place to study from the standpoint of gardening, green space, and interaction with plants and animals.”
The ultimate goal is to translate this work into evidence-based policies that will improve the local environment, she says. Community leaders, for example, used data from the helicopter project to push back against recent proposals by the LA government to increase spending on surveillance helicopters. The process has been a learning experience on how to incorporate community perspectives into research and increase evidence-based policy decisions. “Embedded in this work is ethics that enable neuroscientists to do science that is beneficial to everyone and gives standing to people who have traditionally been left out of the neuroscience enterprise,” says Hansen.
The Loyola University Chicago Program: Launching Careers in Nextgen NeuroSTEM
A second grant for $1 million over two years went to Loyola University Chicago to lead a program aimed at broadening how underserved secondary school and middle school students relate to neuroscience and appreciate its societal impact.
If UCLA and CDU are working on novel strategies for engaging underserved communities in academic research, the Loyola program (it is not formally designated as a center by the Foundation) aims to make a powerful impact much earlier, when students are first taking STEM courses in middle and high school.
Led by M. William Rochlin, PhD, an associate professor of biology and director of the university’s Molecular/Cellular Neuroscience Program, Loyola University Chicago is running a threecomponent educational program termed ETHOS (Ethics-based Teaching Helps Optimize STEM). The first component consists of after-school workshops in which neuroscience discoveries are introduced and then students discuss their ramifications. Discussions revolve around topics like whether people should take drugs that make them smarter, and do we need to change the way we play sports to avoid risks that arise from long-term impact injuries to the brain. Workshops currently are taught by professors, but eventually will be led also by pre- and postdoctoral scientists.
The goal is to invigorate learning STEM components and demonstrate that science and societal concerns are inextricably intertwined, thereby increasing interest in STEM research careers, particularly among underrepresented minorities. Individuals who have experienced injustice prefer careers which address inequities, and traditional approaches to STEM education by focusing on reductionist thinking, do not do that, Rochlin says.
Interested participants in the workshop can apply for the second component, summer internships in Loyola’s neuroscience laboratories. Students accepted into this program receive research training and also provide mentorship by developing neuroscience and society activities for middle schoolers. The idea is for middle school students to see themselves in their mentors. “We want the students to believe that neuroscience is something they can do and, for those concerned about neurojustice, they can still help.”
Currently, the program works with 15 to 20 students, with plans to scale by expanding the programs to a second high school next year and eventually making materials available internationally. Rochlin also wants to develop a version of ETHOS for conventional STEM courses, enlivening the classroom for students who have written off STEM as dry. (The Loyola team believes this will not only make STEM research careers more attractive to those who have experienced social injustices but also will foster development of socially conscientious scientists,
The Dana-MGB Neurotech Justice Accelerator: Tackling Neuroethics in the Medical and Legal Systems
The newest grantee, the Neurotech Justice Accelerator at MGB (NJAM), aims to act as a “national leader in addressing complex legal, ethical and societal challenges imposed by emerging neurotechnologies and to mitigate the risks associated with neurotechnologies, such as misuse in the legal system and ethical concerns about their deployment,” says director Theresa Williamson, MD, MPH assistant professor of neurosurgery and neuroethicist at MGB and Harvard Medical School, and codirector of the NJAM. “Neurotech will change the world in so
many ways, but neuroscience as a field today is not optimized to reflect the changing needs and challenges facing society or the complexity of neuroscience research and technology advances,” she adds.
Emerging technologies and new applications of technologies such as PET, functional MRI, and stimulus-based electroencephalograms provide researchers with access to brain activity, increasing understanding of consciousness and memory. The rapid advances in this field raise complex questions around access, clinical utility, and clinician training.
The accelerator will play a leading role nationally in tackling these issues through a wide net of collaborations led by clinical experts and leading researchers in neurosurgery, neurolaw and bioethics at Harvard Medical School, MGB, and the University of Minnesota. One top priority is to provide interdisciplinary training on the equitable application of technology and on the use of neuroscience as evidence in legal proceedings and criminal justice reforms. A second goal of the accelerator is to form partnerships with communities and advocacy groups to engage with them on the impact of these technology advances.
Its wide-ranging and ambitious goals are evident in the projects it already has underway, started via the pilot program submitted to obtain funding from the Dana Foundation.
NJAM has established the following programs:
• Interdisciplinary research training on equitable application of beneficial clinical neurotechnologies and on neuroscience as evidence in court and in criminal justice reform.
• Community partnership and advocacy training to facilitate bi-directional learning between NJAM fellows and members of communities likely to be impacted by neurotechnology advancement.
In traumatic brain injury, for example, with a large public health impact and opportunity for improvement in care with neurotechnology, the accelerator has projects to improve the design of clinical trials, explore our understanding of consciousness, and improve equitable access to promising new technologies outside of clinical trials. Another project involves surveying entrepreneurs to better understand barriers to developing therapies that are ethically just and find business solutions to overcoming those challenges, says Williamson.
In the legal field, the accelerator is training judges on how to interpret neurotechnology exams and avoid bias. Work in these areas is not new to jurisprudence, and advances in our understanding of brain development, for example, have shaped the way the judicial system deals with problematic adolescents for more than a decade, but it still has a long way to go, she
continues. The Suffolk County MA District Attorney’s office is a collaborator in this effort, led by Dr. Francis Shen, JD PHD at the University of Minnesota.
The Center’s fellowships also aim to understand how communities view these new technologies. Recently, MGB Dana Fellows offered visitors to the Museum of Science in Boston the opportunity to play a game they created called NeuroFutures, which immerses participants in using new technologies to make decisions. A set of cards depicting new technologies, such as mood sensing headphones, is put before participants, who are asked which ones they would invest in. The Fellows have played a variation of this game with community-based organizations including those empowering system-involved youth to learn who they would trust to make decisions.
A Hub and Spoke Model to Grow the Neuroscience & Society Field
The Foundation views the DCI grantees as the hub, the flagship investment, in a hub-and-spoke model to build the neuroscience and society field. The foundation awards smaller grants to research institutions and related science organizations to support education, training, and public engagement on neuroscience and society topics–these grantees comprise of the spokes. Grants here include support for neuroscience educational programs for professionals such as judges and medical professionals, neuroethics pedagogy for graduate students, and community engagement for neurotechnology development. For example, it has supported the establishment of the Implantable Brain Computer Interface Collaborative Community (iBCI-CC), a diverse community of researchers, clinicians, medical device manufacturers, and individuals with lived experience of neurological conditions who are collaborating to accelerate the development, safety, and accessibility of implantable BCIs.
Montojo envisions that the Foundation’s strategic investments will not only establish a new paradigm for neuroscience research but also inspire other funding organizations to innovate on their own funding approaches. Science funders can allocate a portion of their investments, even as little as 5%, to enable scientists to collaborate with experts from fields like ethics, law, humanities, and social sciences. This funding could also support engagement with local communities, potentially enriching the research and inspiring new questions or scientific approaches.
MOVING BEYOND THE AMYLOID HYPOTHESIS Opinion: A Paradigm Shift Is Underway
ENMEDIA Editorial Board
The Alzheimer's research community has been entrenched in the amyloid hypothesis for decades, with the belief that the buildup of amyloid plaques in the brain is the primary driver of Alzheimer's disease. While recent drug approvals like Biogen and Eisai's Leqembi and Eli Lilly's donanemab have provided some hope, these treatments target amyloid and offer only modest benefits, raising fundamental questions about the future direction of Alzheimer's research.
The Amyloid Hypothesis
Since its inception in the 1990s, the amyloid hypothesis has driven most Alzheimer's research. It posits that the accumulation of beta-amyloid plaques in the brain leads to neurodegeneration and cognitive decline. Billions of dollars have been invested in targeting this pathology, leading to the development of several drugs aimed at reducing amyloid plaques. However, the results have been underwhelming, with therapies showing limited efficacy in slowing the progression of cognitive decline.
As Jonathan Behr, Ph.D., of the Dementia Discovery Fund, noted during a 2023 panel discussion at the BioFuture Conference, while amyloid-targeting
therapies like Leqembi offer some benefits, they fall far short of a cure. “I think finally what we can say definitively is that it provides benefit for patients. Unfortunately, the benefit is not of the magnitude that we would hope.” This leaves a significant unmet need for effective treatments, underscoring the need for a shift in research priorities.
Tau and Other Pathologies
One of the major criticisms of the amyloid hypothesis is its narrow focus. Alzheimer’s disease is complex and multifactorial, with amyloid being just one piece of a much larger puzzle. Tau proteins, which form neurofibrillary tangles in the brains of Alzheimer's patients, are another critical marker of the disease. Like amyloid, tau tangles are associated with neuronal damage and cognitive decline, yet research into tau-targeting therapies has lagged behind.
Eli Lilly’s recent failure in a Phase 2 trial targeting tau pathology further underscores the complexity of Alzheimer's. Despite early excitement around tau as an alternative therapeutic target, developing effective treatments remains elusive. The focus on amyloid may have led to an underfunding of alternative approaches that could offer greater promise.
Precision Medicine and New Approaches
Recent discussions among experts highlight a growing consensus that Alzheimer's research must move beyond amyloid and tau to embrace a broader range of approaches. Precision medicine, which involves tailoring treatments to individual patients based on their unique genetic, molecular, and environmental profiles, is one area gaining traction. Companies like Altoida are developing technologies that assess cognitive and functional impairments arising from amyloid pathology, but the next step is identifying how different pathologies contribute to these deficits.
Marc Jones, CEO of Altoida, emphasized the need for personalized assessments: “There are plenty of technologies; blood biomarkers today are helping identify pathology. But the second shoe to fall needs to be their functional or cognitive impairment resulting from that pathology.” This approach, which involves understanding how various factors like amyloid, tau, and other markers interact to cause cognitive decline, could be key to developing more effective therapies.
Limitations of Amyloid Therapies and Systemic Challenges
Despite the promise of recent drug
approvals, the real-world effectiveness of amyloid-targeting therapies remains limited. While therapies targeting amyloid, such as Leqembi, have shown some ability to slow cognitive decline, their effects are modest and not curative. Targeting amyloid, which is present late in the disease course, fails to address the complex, multifactorial processes underlying Alzheimer’s disease, such as tau pathology and inflammation, which may begin earlier and drive cognitive decline more profoundly. By the time amyloid plaques are targeted, much of the irreversible neurodegeneration has already set in. As a result, while therapies like Leqembi may slow the disease's progression, their overall impact remains limited, leaving the underlying neurodegeneration largely unchecked.
What’s more, the U.S. healthcare system is ill-equipped to manage the rollout of these new therapies. Medicare’s current cautious stance on covering Alzheimer’s drugs further complicates access. According to Tamara Syrek Jensen, a top official at the Centers for Medicare and Medicaid Services (CMS), Medicare’s coverage decisions for fully approved Alzheimer’s
drugs are tied to ongoing data collection from patient registries. Full evaluations of these treatments' performance could take years, prolonging the time before patients gain widespread access. These systemic challenges highlight the need for a more holistic approach to treating Alzheimer’s, one that goes beyond simply targeting amyloid plaques and addresses the broader context in which care is delivered.
The Future: A Multi-Faceted Approach to Alzheimer's
As the failures of amyloid-targeting therapies pile up, it has become increasingly clear that a multi-faceted approach is necessary to make meaningful progress in Alzheimer's research. Moving forward, the field must focus on understanding how various pathologies— amyloid, tau, inflammation, vascular factors, and more—interact to cause cognitive decline. This shift will require greater investment in alternative research avenues, including tau-targeting therapies, precision medicine, and digital biomarkers.
Moreover, public advocacy and policy changes will be critical to ensuring that new discoveries are
translated into effective treatments. UsAgainstAlzheimer’s and other advocacy groups must continue to push for increased funding, better access to care, and policies that support earlier detection and intervention.
Alzheimer's research is at a crucial turning point. While the amyloid hypothesis has shaped the field for decades, the efficacy of recently approved amyloid-targeting drugs demonstrates that this narrow focus may no longer be sufficient. To truly turn the corner on this intractable disease, the field as a whole must broaden its focus and embrace a wider, more comprehensive range of research approaches that address the multiple factors driving Alzheimer's disease. Only then can we hope to develop truly effective treatments and significantly impact the lives of those affected by this devastating condition.
BEYOND BLOCKCHAIN BEYOND BITCOIN BEYOND BEYOND
A Look at Decentralized Science
Blockchain technology was once a niche interest among tech enthusiasts. But since it hit the mainstream consciousness in the early 2010’s it has continued to garner attention from diverse corners–speculators, techies, people all across the political spectrum and of course, regulators. Blockchain underpins not just cryptocurrencies like the well-known Bitcoin and Ethereum, but increasingly a wide array of applications from supply chain management to secure voting systems. Multi-trillion-dollar asset manager Blackrock recently made headlines with its entry into the space.
At its core, blockchain provides a decentralized ledger, ensuring data integrity through a network consensus rather than a central authority. Although it has plenty of skeptics, this architecture promises transparency and immutability; once data is encoded, altering it without network consensus is virtually impossible, as long as the chain boasts strong security via a diversified and robust network of transaction validators or “miners.”
Blockchain provides a decentralized ledger, ensuring data integrity through a network consensus rather than a central authority.
And now, blockchain has made its way into the realm of scientific research through the Decentralized Science (DeSci) movement. You’ve likely heard of its cousin, decentralized finance or “DeFi.” Just as DeFi looks to disintermediate financial services, DeSci hopes to remodel the landscape of scientific research, publishing, and funding, thereby addressing systemic barriers that have long stifled innovation. At the forefront of DeSci is the promise to dismantle the paywalls that have restricted access to scientific knowledge, positioning scientific discovery as a public good accessible to all.
These types of projects are often organized under a structure called a DAO (decentralized autonomous organization). A DAO is an entity that operates through the use of smart contracts on a blockchain. These rules-based contracts automate organizational governance, allowing DAOs to operate without centralized control. Members typically govern the DAO collectively, making decisions through a consensus mechanism, commonly involving token-based voting.
Tokens can be listed on exchanges (both centralized and decentralized), resulting in a market mechanism where anybody can buy and sell the tokens. If you’re optimistic on the potential of a project, you can buy and hold the token, anticipating it will rise in value as the project grows.
The enthusiasm for DeSci is tempered by some inherent challenges. Questions arise around maintaining the rigor and integrity of scientific research when traditional peer review mechanisms are replaced by community-driven evaluations and token-based incentives. Could the allure of commercial gains skew the focus away from foundational science?
The advent of blockchain applications has led to an explosion of tokens and cryptocurrencies, each competing for investor attention and market liquidity. For a project to thrive, it may need to attract enough trading volume to ensure liquidity while also demonstrating clear value and utility beyond speculation. The emergence of new tokens can dilute investment from existing projects, potentially undermining their development and stability.
Balancing innovation with scientific integrity is key. But whatever the potential drawbacks, movements like DeSci do have one significant edge: impeccable timing. They’re coming along at a time when trust in traditional institutions is eroding at an alarming rate.
SOME NOTABLE DESCI PROJECTS
An online collective enhancing computational life science research with open-source tools, including BioML models, for global collaboration.
Utilizes blockchain and AI to revolutionize healthcare data, evolving from a telemedicine startup to a DAO focused on transparent, patient-centric care.
Targets aging research through advanced AI, analyzing network data to offer breakthrough insights, aiming for accessible and affordable outcomes. A community-driven collective funding longevity science, with over $4.2 million invested in 22 research projects to combat age-related diseases.
Greater Accuracy Equals Greater Confidence in Your Studies
FcResolv® Mouse Models
Murine Fc gamma receptors (FcγRs) can confound preclinical antibody-based therapy study results, leading to incorrect conclusions and derailing drug discovery. By knocking out these receptors, FcResolv® NOG models provide clarity in these studies, offering more translatable data while utilizing fewer resources.
Which FcResolv® Model is Right for You?
FcResolv® NOG mouse models expand the utility of the Taconic super-immunodeficient NOG platform. They are ideal for investigating any indication which utilizes a therapy incorporating an Fc domain, including immuno-oncology, autoimmune disease, and more.
Suitable for tumor xenografts, cell or tissue engraftments, and immune system humanization studies. CD34+ HSC engraftment available.
NEW! hIL-3 and hGM-CSF expression for exceptional support of human myeloid and lymphoid cells. CD34+ HSC engraftment available.
Expresses human IL-15 cytokine to support engraftment of human NK cells.